Jeff Wren, Vice President, Biotherapies

Oct. 8 | 10:45am | FLW Ballroom F

Bethesda, MD

(Private)

AABB is a global, nonprofit organization dedicated to advancing the quality and safety of cell and gene therapies (CGTs) and other biotherapies. Through internationally recognized standards, accreditation, and education programs, AABB helps ensure that patients receive safe, effective therapies—from cellular starting material collection through advanced therapy handling in pharmacy settings. We collaborate closely with therapy developers, healthcare providers, and competent authorities to foster innovation, support regulatory readiness, and promote trusted partnerships across the CGT ecosystem. AABB’s offerings include accreditation for collection and processing facilities, standards for pharmacy-based handling of advanced therapies, and credentialing programs that help companies build qualified teams and accelerate market readiness. With 6,000+ members, 1,300+ accredited facilities, and a network of engaged stakeholders, AABB provides the infrastructure and community to drive progress and expand access to transformative therapies worldwide.

www.aabb.org

Michael Banigan, President & CEO 

Oct. 7 | 3:00pm | FLW Ballroom G

Frisco, TX 

(Private) 

Aileron is a technology company providing the most advanced and only solution for unified data connectivity for advanced therapies.  Our state-of-the-art platform allows manufacturers, institutions, and third parties to connect and share data through a single, secure, P2P connection that transforms operational efficiency for advanced therapies. Our technology utilizes the highest standards in data integrity, blockchain, and AI to deliver a world-class solution that only allows the sender and receiver to access data ensuring data sovereignty. Using our unified calendar, clinics can schedule a drug manufacturing date online in real time avoiding the issues associated with multiple portals and 800 numbers to simply schedule a patient.  Aileron accelerates patient onboarding, streamlines operations, and provides the most advanced unified data connection in pharmaceuticals and healthcare.  

Aileron-AI.com

Brandon McNaughton, Ph.D., CEO & Co-founder

Oct. 7 | 5:15pm | FLW Ballroom G

Ann Arbor, MI

(Private)

Akadeum Life Sciences’ next generation cell isolation platform solves long-standing problems in cell therapy and research: time, cost, cell health, scalability. The first new GMP cell separation modality in 40 years, Akadeum’s buoyant microbubble technology represents one of the first meaningful innovations in a critical early step of the cell therapy manufacturing process. Kits are readily incorporated into workflows without the need to purchase equipment or overhaul processes, delivering consistently high performance while enabling up to a 42% reduction in workflow footprint, reducing GMP suite time by 80% and training requirements down to one hour, all via a simple closed system in-bag cell separation done at any scale, with any starting material. Microbubbles are compatible with both centralized and decentralized manufacturing models, enabling increased access to life changing therapies at reduced costs. Together, we deliver consistent, high-purity cells that set the foundation for better therapies — and real clinical success. 

www.akadeum.com 

Tom Foti, Vice President & General Manager

Oct. 6 | 5:30pm | FLW Ballroom F

Fargo, ND

(NASDAQ: DHR)

Aldevron is a premier manufacturing partner, producing high-quality plasmid DNA, mRNA, proteins, and other key components for the development of vaccines, gene and cell therapies, immunotherapies and other treatments. As a part of the Danaher Corporation family of global science and technology companies, Aldevron supports thousands of scientists who are developing revolutionary, lifesaving treatments for millions of people. To learn more about how Aldevron is advancing biological science, visit Aldevron.com and follow the company on LinkedIn, Facebook and YouTube.    

www.aldevron.com 

Rajesh Ambasudhan, Ph.D., Chief Strategist

Oct. 8 | 11:30am | FLW Ballroom F

San Diego, California

(Private)

Allele Biotechnology & Pharmaceuticals is a science-driven establishment focused on developing mRNA and stem cell-based technologies and products. Allele holds a patent-portfolio for mRNA-based iPSC reprogramming, differentiation, and gene-editing. As the first commercial provider of GMP-grade iPSCs that maintain the full chain of custody from tissue acquisition to qualification we focus on providing best-in-class, footprint-free, and robust iPSC lines to therapeutic developers. Our technologies and therapeutic assets, and/or off-the-shelf/custom iPSC lines have been partnered/licensed by industry pioneers such as Astellas and KPharma. On the service side, our FDA-registered, purpose-built, cGMP facility offers end-to-end services supporting cell & gene industry, with multiple entry points for partnering from early-stage research, custom analytical services, process-development and technology transfer through commercial manufacture of drugs. We employ creativity and niche-experience to develop win-win business models with our partners to best utilize our combined assets in therapeutic programs, GMP-cells and facility, and mRNA-based platform technologies.

www.allelebiotech.com

Reagan Jarvis, Ph.D., CEO

Oct. 7 | 5:30pm | FLW Ballroom F

Södertälje, Sweden

(Private)

Anocca leverages its deep-tech cell-based platform and in-house cGMP capability to discover, develop and manufacture novel TCR-T product libraries at scale for the treatment of solid tumor cancers and other serious diseases, including autoimmune and infectious disease. 
The company’s oncology pipeline comprises over 40 high value TCR-T assets that span a wide range of cancer targets and HLA restrictions. The most advanced TCR-T progam, targeting KRAS G12V in pancreatic cancer is starting a phase 1/2a trial in Europe, following CTA approval in March 2025. 
Based near Stockholm in Sweden, Anocca is privately held and employs a team of over 130 talented individuals. 

www.anocca.com 

Devyn Smith, Ph.D., CEO

Oct. 7 | 2:30pm | FLW Ballroom F

Cambridge, MA

(Private)

Arbor Biotechnologies™, a next-generation gene editing company based in Cambridge, MA, is advancing a pipeline of novel gene editing therapeutics to address a wide range of genetic conditions – from the ultra-rare to the most common genetic diseases. The company’s unique suite of optimized gene editors goes beyond the limitations of early editing technologies to unlock access to new gene targets and has fueled a robust pipeline of first-in-class assets focused on diseases of high unmet need. With Arbor’s lead program, ABO-101 for the treatment of primary hyperoxaluria type 1, progressing into clinical trials, the company continues to focus their research and development efforts on genomic diseases of the liver and CNS for which there are no existing functional cures.  

www.arbor.bio 

Jennifer MacDonald, M.D., Chief Operating Officer 

Oct. 6 | 11:15am | FLW Ballroom G

Manchester, NH

(Private)

The Advanced Regenerative Manufacturing Institute (ARMI), through its BioFabUSA program, is accelerating the commercial readiness of regenerative medicine by bridging the gap between early innovation and large-scale manufacturing. ARMI supports more than 175 member organizations—from startups to global biotech leaders—by providing access to enabling technologies, bioprocessing expertise, and regulatory and commercialization support. Through its BioFab Startup Lab, ARMI helps early-stage companies de-risk manufacturing and prepare for clinical and market milestones. By addressing core challenges such as automation, standardization, and talent development, ARMI is building the advanced manufacturing capacity needed to bring cell-based therapies, engineered tissues, and regenerative technologies to patients. With strong national partnerships and infrastructure in place, ARMI plays a critical role in strengthening domestic biomanufacturing capabilities and reducing time to market for advanced therapies. 

www.armiusa.org

Nicholas Veomett, Ph.D., Vice President, Corporate Development 

Oct. 8 | 9:15am | FLW Ballroom F

San Diego, CA

(NASDAQ: ARTV) 

Artiva is a clinical-stage biotechnology company whose mission is to develop effective, safe and accessible cell therapies for patients with autoimmune diseases and cancers. Artiva’s robust manufacturing platform produces thousands of 1 billion-cell vials of NK cells from a single umbilical cord blood unit. Artiva’s lead program, AlloNK®, is an allogeneic, off-the-shelf, non-genetically modified NK cell therapy candidate designed to enhance the antibody-dependent cellular cytotoxicity effect of monoclonal antibodies. AlloNK is delivered in the outpatient setting with no required hospitalization. AlloNK is currently being evaluated in three ongoing clinical trials for the treatment of B-cell driven autoimmune diseases: two company-sponsored trials across multiple autoimmune diseases including RA, SLE, LN and Sjögren’s disease, as well as an investigator-initiated basket trial. Artiva’s pipeline also includes CAR-NK candidates targeting both solid and hematologic cancers. 

www.artivabio.com

Daniel Rosan, Chief Financial Officer, Chief Business Officer 

Oct. 6 | 4:30pm | FLW Ballroom G

Boston, MA 

Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With discovery, preclinical, and clinical programs in retinal, neurological, neuromuscular, and genetically defined diseases, Ascidian’s approach has the potential to treat patients with one dose of an RNA exon editor, opening new therapeutic possibilities for patients and their families who are seeking breakthroughs.

www.ascidian-tx.com 

Koji Ushioda, CEO

Oct. 7 | 2:00pm | FLW Ballroom F

Gembloux, Belgium

(Private)

Aseptic Technologies, part of the SKAN Group, is a global leader in aseptic processing solutions, ensuring the highest sterility standards for pharmaceutical, biotech, and healthcare applications. We specialize in advanced fill and finish systems and isolator technologies. As a trusted partner to pharmaceutical manufacturers, we integrate aseptic processes from R&D to production, offering cutting-edge filling equipment, barrier technologies, and custom single use solutions to enhance efficiency and safety. With decades of expertise, we provide tailored solutions for parenteral drugs, ATMPs, and high-potency compounds. Our mission is to drive aseptic manufacturing excellence through innovation, customer-centric solutions, and industry-leading standards.

www.aseptictech.com/en

Tamer Mohamed, Founder and CEO

Oct. 7 | 2:30pm | FLW Ballroom G

Vancouver, BC, Canada

(Private)

Aspect Biosystems is a biotechnology company pioneering the development of Bioprinted Tissue Therapeutics to transform how we treat some of the most elusive diseases. Aspect’s Bioprinted Tissue Therapeutics are designed to replace, repair, or supplement biological function inside the body. Aspect is creating these next-generation cell therapies by applying its full-stack tissue therapeutic platform, which integrates proprietary AI-powered bioprinting technology, computational design tools, therapeutic cells, and advanced biomaterials. Aspect is advancing a pipeline of Bioprinted Tissue Therapeutics across multiple disease areas in the endocrine and metabolic space including diabetes, obesity, rare endocrine disorders, and liver disease. Aspect’s pipeline development strategy involves both proprietary programs as well as strategic partnerships, including a collaboration with Novo Nordisk focused on diabetes and obesity. 

www.aspectbiosystems.com

Ana Sousa, Chief Regulatory Officer

Oct. 6 | 4:15pm | FLW Ballroom G

San Diego, CA 

(Private) 

Aspen Neuroscience, Inc. is a clinical development-stage, private company focused on autologous regenerative medicine. The company’s patient-derived iPSC platform is used to create personalized therapies to address diseases with high unmet medical needs, beginning with autologous neuron replacement for PD. 

www.aspenneuroscience.com 

Erin Kimbrel, Ph.D., Head, Cell and Gene Therapy Research

Oct. 6 | 12:15pm | FLW Ballroom F

Tokyo, Japan

(NASDAQ: ALPMY)

Astellas is a global life sciences company committed to turning innovative science into VALUE for patients. Through our R&D programs, we are pioneering new healthcare solutions for diseases with high unmet medical need. This includes our focus on delivering breakthrough gene therapies with life-changing potential for patients across many disease areas. We are building one of the world’s most robust pipelines of gene therapies for genetic disorders and an end-to-end gene therapy infrastructure with industry-leading discovery, development, manufacturing and commercial capabilities. With our partners, we have several candidates in clinical development including for rare neuromuscular diseases: KT430, in partnership with Kate Therapeutics, approaching First Subject Dosed for XLMTM by end of year, and AT845 in Phase 1/2 for Pompe disease. Alongside partners, clinicians, regulatory authorities and patient communities, we strive to overcome complex challenges across the value chain so we can deliver the promise of ground-breaking science to patients.

www.astellas.com

Jeremie Laurent, Ph.D., CEO

Oct. 7 | 3:15pm | FLW Ballroom G

Paris, France

(Private)

Founded 2016 in Paris by Jérémie Laurent (CEO), Astraveus aspires to radically reduce complexity and cost of cell therapy manufacturing, thereby making cell therapies more accessible to patients. Astraveus develops the Lakhesys Benchtop Cell Factory, a scalable, fully automated microfluidic platform for end-to-end cell therapy process development, clinical and commercial manufacturing. This revolutionary platform delivers unprecedented reduction of manufacturing footprint, labor and use of expensive process materials, leveraging proprietary microfluidic technology. The Lakhesys Benchtop Cell Therapy Factory enables parallelized and automated cell therapy processing in an end-to-end fashion, from cellular starting material to the final drug product, and provides integrated real-time quality control capabilities. Through this holistic approach, Astraveus minimizes process development effort and maximizes manufacturing output, thus enabling access to care for patients in need at a cost per dose as low as $15k.

www.astraveus.com

Michael Maguire, Ph.D., CEO  

Oct. 7 | 9:15am | FLW Ballroom F

Kildare, Ireland  

(Private) 

Avectas is an enabling technology company advancing the scalable, cost-effective manufacturing of gene-modified cell therapies, including CAR-T therapy. The company has developed Solupore®: a closed, automated, integrated, GMP-ready manufacturing platform that enables the efficient production of cell therapies such as CAR-T for immuno-oncology and autoimmune disease, from development to commercial scale. In the past 12 months, Solupore has progressed significantly beyond its core non-viral transfection capability to incorporate multiple manufacturing steps including activation, transduction, transfection, proliferation, and harvest into a single-use consumable system. Standard manufacturing workflows typically rely on multiple devices and reagents, limiting scalability. Solupore addresses these constraints by integrating key processes and enabling safe parallel processing of closed consumables on a single controller device, increasing both equipment and suite throughput while maintaining viability, potency, and functionality of engineered cells. Avectas has filed Device and Drug Master Files (DMFs) to support regulatory adoption of the Solupore platform. 

www.avectas.com 

Lisa Deschamps, CEO

Oct. 7 | 11:30am | FLW Ballroom G

London, UK

(Private)

At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into potentially life-changing medicines for people living with neurological disease, beginning with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and suite of proprietary gene therapy platforms and innovative delivery technologies, we are working to overcome the challenges of delivering the right drug to the right place. Our innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially even reverse neurodegenerative diseases.  Our rich heritage in academia is complemented by a strong leadership team with extensive gene therapy development and commercialization experience. 

www.aviadobio.com

Tiffany Brown, Ph.D., CEO

Oct. 6 | 3:15pm | FLW Ballroom G

Newark, Delaware

(Private)

Avobis Bio is clinical-stage biotech company developing implantable cell therapies to heal debilitating conditions.  We were launched out of the Mayo Clinic in 2019 and our lead asset is in a Phase 2 clinical trial for treatment of Crohn’s perianal fistulas.  Our technology overcomes impaired healing using the synergistic combination of living cells and bioabsorbable materials.  Following the Phase 2 clinical trial results available in summer 2025, we are planning an investment round to enable the Phase 3 pivotal trial. 

www.avobisbio.com

Hunter Johnson, Ph.D., CEO

Oct. 7 | 5:15pm | FLW Ballroom F

Berkeley, CA

(Private)

Axent Biosciences is a pre-clinical stage company that aims to revolutionize the field of cell therapy by leveraging a simple, safe and scalable cell cultivation technology to produce best-in-class cells with industry-leading yields. Axent’s platform relies on a novel synthetic 3D matrix that supports robust cell growth and differentiation while enabling commercial-scale production. This system overcomes key challenges in stem cell manufacturing, including genomic instability, low yields, and poor reproducibility, by providing a xeno-free, shear-protected environment compatible with multiple bioreactor configurations. Cells produced in Axent’s platform exhibit high viability, genomic stability over 10 passages, and superior potency. This technology is compatible with standard differentiation media components and delivers significantly higher cell yields (5-10M/mL) and fold changes (>100 fold) than traditional methods. This breakthrough enables the first-ever commercial-scale production of cell therapies for high-demand indications including Type 1 Diabetes, Liver Disease and Heart Failure.

www.linkedin.com/company/axent-biosciences

Adrienne Mendoza, Chief Operating Officer

Oct. 8 | 11:00am | FLW Ballroom F

San Antonio, TX

(Private)

BBG Advanced Therapies delivers a fully integrated and custom portfolio of innovative solutions, including collection and processing of starting materials, testing and manufacturing services that bring transformative therapies to patients across the globe.The BioBridge Global family of nonprofits includes South Texas Blood & Tissue, QualTex Laboratories, BBG Advanced Therapies and The Blood & Tissue Center Foundation. For 50 years, we have worked with the healthcare community to provide lifesaving therapies to patients and to support advances in advanced therapies.

www.BBGAT.org

Seth Ettenberg, Ph.D., CEO 

Oct. 6 | 2:45pm | FLW Ballroom F

Cambridge, MA

(Private)

BlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. Two of our novel investigational cell therapies, bemdaneprocel for the treatment of Parkinson’s disease and OpCT-001 for the treatment of primary photoreceptor diseases are clinical stage programs. Bemdaneprocel has RMAT and Fast Track designation from the US FDA (Food and Drug Administration) and is being tested in a Phase 3 clinical trial, exPDite-2. OpCT-001 has Fast Track designation from the FDA and is being tested in a Phase 1 clinical trial, Clarico. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG.

www.bluerocktx.com 

Peter Keller, CEO

Oct. 8 | 10:15am | FLW Ballroom G

Philadelphia, PA

(Private)

At BlueWhale Bio, we’re focused on transforming T cell manufacturing with our Synecta™ T1 Cell-Derived Nanoparticles. Our platform is designed to improve T cell activation, proliferation, and overall quality—critical elements for successful CAR-T and other cell therapies. Unlike traditional methods that rely on beads or feeder cells, our nanoparticles offer a tunable, cell-free solution that mimics natural stimulation while simplifying and streamlining the manufacturing process. This means better consistency, higher yields, and reduced costs for our partners. We work closely with both academic labs and commercial biotech teams to support everything from early discovery through scale-up. Headquartered in the U.S., we collaborate with leaders across the immunotherapy space to help accelerate the next generation of cell therapies. Our goal is to remove the bottlenecks in T cell expansion and enable more reliable, scalable, and effective manufacturing—making life-changing therapies more accessible to patients.

www.bluewhale.bio

Felix Dobbs, Ph.D., CEO

Oct. 7 | 5:45pm | FLW Ballroom F

Cambridge, England

(Private)

Broken String Biosciences is advancing the safety and precision of cell and gene therapies through its proprietary INDUCE-seq® platform. INDUCE-seq® has become the leading choice for unbiased, PCR-free detection of genome-wide DNA double-strand breaks, enabling drug developers to simultaneously identify both on- and off-target edits with high resolution in days, not months. Offered as both a full-service solution and an in-lab kit, INDUCE-seq® supports therapeutic development from discovery through commercialization. It is validated for use in optimizing editing efficiency in iPSC- and T cell–based therapies, and for evaluating emerging technologies like base editors, and improving guide RNA design. INDUCE-seq®is quickly becoming an established standard in safety assessments through participation in the NIST Genome Editing Consortium with HESI, which is establishing industry standards for safe gene editing.

www.brokenstringbio.com

AJ Bergmann, M.D., Chief Financial Officer

Oct. 6 | 5:15pm | FLW Ballroom F

San Diego, CA

(NASDAQ: CAPR)

Capricor Therapeutics is a publicly traded biotechnology company with a mission to develop groundbreaking therapies that make a meaningful impact on patients’ lives. Capricor is focused on the development of biologics, primarily cell and exosome-based technologies to treat or prevent a broad range of diseases. Our lead product, deramiocel (also referred to as CAP-1002) is a cell therapy designed to treat Duchenne muscular dystrophy (DMD), the most severe form of muscular dystrophy, which results in muscle degeneration and premature death. Capricor’s innovative exosome technology is focusing on engineering exosomes to treat or prevent diseases with high unmet medical needs. Our translational approach to product development is based on the strong research foundation provided by our academic partnerships with leading scientists at top-tier research institutions. Capricor’s lead product candidate, deramiocel (CAP-1002), is an allogeneic cell therapy that is currently in Phase 3 clinical development for the treatment of Duchenne muscular dystrophy. Deramiocel is comprised of allogeneic cardiosphere-derived cells, or CDCs. The ability of deramiocel to slow disease progression in Duchenne muscular dystrophy lies in the immunomodulatory, anti-inflammatory, pro-angiogenic, and anti-fibrotic actions of CDCs, which are mediated by secreted exosomes containing bioactive cargo. Among the cargo elements known to be bioactive in CDC-exosomes are microRNAs. Collectively, these non-coding RNA species alter gene expression in macrophages and other target cells, dialing down generalized inflammation and stimulating tissue regeneration in DMD (and in a variety of other inflammatory diseases). Capricor has also established itself as one of the leading companies advancing exosome-based technology. Emerging as an exciting class of potential therapeutic agents and drug delivery vehicles, exosomes are nanosized particles with biologically active contents that are secreted by cells and serve to direct or re-direct the activities of other cells. Capricor is focused on developing a precision-engineered exosome platform technology with the ability to deliver defined sets of effector molecules which exert their effects through defined mechanisms of action. Our StealthX™ platform builds on advances in fundamental RNA and protein science, targeting technology and manufacturing, providing us the opportunity to potentially build a broad pipeline of new therapeutic candidates.

www.capricor.com 

Rupa Pike, Ph.D., Sr. Director, Global Head of Strategic Alliances and Field Scientists, Cell Therapy

Oct. 6 | 12:00pm | FLW Ballroom F

Somerset, New Jersey

(NASDAQ: CTLT)

Catalent Cell & Gene Therapy is an industry-leading Contract Development and Manufacturing Organization (CDMO) for advanced therapeutics.
With extensive experience in viral vector development, scale-up, and manufacturing of gene therapies, Catalent serves as a full-service CDMO for plasmid DNA, adeno-associated viral (AAV), lentiviral, and other viral vectors, oncolytic viruses, and live virus vaccines. Additionally, with our advanced technologies and expertise in both autologous and allogeneic development and manufacturing, as well as experience across a wide variety of cell types including CAR-T, TILs, NKs, iPSCs, and MSCs, Catalent is your full-service CDMO partner for cell therapy. As an experienced and innovative partner, Catalent Cell & Gene Therapy boasts a global network of dedicated clinical and commercial manufacturing facilities, including an FDA-licensed viral vector facility and fill/finish capabilities located in both the U.S. and Europe.

biologics.catalent.com

Fabian Gerlinghaus, CEO and Co-Founder

Oct. 6 | 5:15pm | FLW Ballroom G

San Francisco, CA

(Private)

Cellares is the world’s first Integrated Development and Manufacturing Organization (IDMO) and takes an Industry 4.0 approach to mass manufacturing the living drugs of the 21st century. The company has developed integrated technologies, the Cell Shuttle™ and Cell Q™, to automate the end-to-end manufacturing and quality control of cell therapy products. These technologies will be deployed in Cellares’ IDMO Smart Factories around the world to meet total patient demand for cell therapies at global scale. Partnering with Cellares enables academics, biotechs, and pharma companies to accelerate drug development and scale out manufacturing, lower process failure rates, lower manufacturing costs, and make life-saving treatment more accessible to patients. The company is headquartered in South San Francisco, California with its commercial-scale IDMO Smart Factory in Bridgewater, New Jersey. The company is backed by world-class investors and has raised over $355 million in financing.

www.cellares.com

Matthew Hewitt, Ph.D., Vice President, Technical Officer Manufacturing

Oct. 7 | 10:00am | FLW Ballroom G

Wilmington, MA 

(NASDAQ: CRL) 

In-person Presentation 

Charles River provides essential products and services to support pharmaceutical and biotechnology companies, government agencies, and leading academic institutions worldwide in accelerating research and drug development. As a leader in scientific innovation, we are deeply committed to pioneering alternative methods that drive discovery while reducing reliance on traditional models. By embracing cutting-edge technologies and forward-thinking approaches, we enhance preclinical and clinical research. Our dedicated employees focus on delivering precisely what our clients need to improve, innovate, and expedite the discovery, early-stage development, and safe manufacture of new therapies for patients, because every moment matters.

www.criver.com 

Lawrence Forman, Founder and CEO

Oct. 7 | 5:00pm | FLW Ballroom G

South San Francisco, CA

CHO Plus, Inc. is a privately held company founded in 2014 with the mission of increasing the productivity of mammalian cells used for manufacturing life-saving therapeutic agents for treating human disease. CHO Plus has patents granted and pending for several different technologies in over ten jurisdictions around the world. Currently CHO Plus studies demonstrate that our engineered CHO cells produce up to 10-fold more therapeutic antibodies than un-engineered cells and our engineered HEK-293 cells produce up to 20-fold AAV vectors, with up to 55% full capsids before purification. With a focus on innovation and collaboration, the company is committed to advancing the production of therapeutic proteins and gene therapy vectors to address a wide range of clinical indications.

www.cho-plus.com

Lijun Wang, Ph.D., Founder and CEO

Oct. 7 | 2:45pm | FLW Ballroom G

Lower Gwynedd, PA

(Private)

Key Considerations of Plasmid Manufacturing for Viral Vector Production
In the ever-evolving world of cell and gene therapy, the demand for high-quality, scalable, and regulatory-compliant plasmid DNA is at an all-time high. Traditional plasmid manufacturing workflows rely heavily on column chromatography, a time-consuming and costly purification method that involves multiple steps, significant infrastructure, and complex scale-up challenges.
CoJourney’s Zero Chrom® Plasmid Platform eliminates the need for column chromatography entirely—ushering in a new era of simplified, high-throughput, and scalable plasmid production. This innovation not only streamlines manufacturing but also lowers regulatory risk and dramatically reduces production timelines. Key benefits of the platform include:

Faster Purification: No column chromatography means shorter production cycles and lower costs.Low Regulatory Risk: Use of single-use systems reduces the potential for cross-contamination—critical in clinical-grade manufacturing.Superior Quality: Produces plasmid DNA with >95% supercoiled structure, ideal for therapeutic applications.FDA & NMPA Approved: Zero Chrom® plasmids have been used in IND-approved programs across major markets.Scalable Output: Supports both research-grade and GMP-grade plasmid DNA production ranging from 1L to 30L, with a proven capacity of over 300 batches per year.

Meet the Visionary:
Dr. Lijun Wang, a molecular biologist and industry veteran, will be on-site to answer questions and discuss how CoJourney’s manufacturing platforms are accelerating gene and cell therapy programs worldwide. Her presentation will highlight the technical innovations behind Zero Chrom®, including its upstream and downstream integration, data on yield consistency, and adaptability across gene therapy modalities.

www.cojourney.com

Hendrik Dietz, Ph.D., CEO

Oct. 8 | 10:30am | FLW Ballroom F

Munich, Germany

(Private)

CPTx is redefining the future of genetic medicine through a single-stranded DNA (ssDNA) platform that enables safer, redosable, and scalable gene therapies. The lead proprietary development program is a new kind of in vivo CAR T-cell therapy using immune-silent single-stranded DNA (ssDNA) delivered with LNPs, enabling repeat dosing, and controllable, long-lasting effects, something not possible with viral vectors or mRNA.

www.cptx.bio

Timothy Schroeder, Chairman and CEO

Oct. 7 | 9:45am | FLW Ballroom F

Covington, KY

(Private)

CTI Clinical Trial and Consulting Services is a global, privately held, research service organization, delivering a complete spectrum of clinical trial and consulting services throughout the lifecycle of development, from concept to commercialization. CTI’s focused therapeutic approach provides pharmaceutical, biotechnology, and medical device firms with clinical and disease area expertise in rare diseases, regenerative medicine / gene therapy, immunology, transplantation, nephrology, hematology / oncology, neurology, infectious diseases, hepatology, cardiopulmonary, and pediatric populations. CTI also offers a fully integrated multi-specialty clinical research site and complete global laboratory services. Now in its third decade, CTI is one of the 20 largest contract research organizations in the world, with associates in more than 60 countries across six continents. CTI is headquartered in the Greater Cincinnati area, with operations across North America, Europe, Latin America, MEA, and Asia-Pacific.

www.ctifacts.com 

Mathias Kroll, Ph.D., Chief Business Officer 

Oct. 6 | 9:45am | FLW Ballroom G

Melbourne, Australia

(ASX:CYP)

Cynata Therapeutics Limited (ASX:CYP) is a listed, Australian stem cell and regenerative medicine company pioneering a revolutionary approach to cell therapy with our proprietary Cymerus™ platform technology. Developed from research at the University of Wisconsin-Madison, a world leader in stem cell research, our technology harnesses the power of induced pluripotent stem cells (iPSCs) to create a type of mesenchymal stem cells (MSCs) for therapeutic use via a unique precursor cell called the mesenchymoangioblast. Cynata has a rich pipeline with multiple active clinical trials and near-term catalysts including: Phase 2 clinical trial in acute graft-versus-host disease (aGvHD) – currently recruiting globally in final stages Phase 3 clinical trial in osteoarthritis – recruitment complete as of 2023 and last patient visit in November 2025Phase 1/2 clinical trial in kidney transplantation – recruiting patients in the Netherlands Phase 1 clinical trial in diabetic foot ulcer (DFU) – trial complete and results announced in December 2024.

www.cynata.com 

Jenny Pchelinski, Business Development Manager, Cell Lines

Oct. 6 | 9:45am | FLW Ballroom F

Marlborough, MA

(NYSE: DHR)

Genomic medicine, particularly cell and gene therapies, is revolutionizing healthcare. However, challenges in manufacturing and development timelines could hinder widespread adoption. Innovative manufacturing solutions are crucial to address these challenges. Optimizing process will not be enough, advanced technologies are needed to get to the next level. Cytiva is dedicated to accelerating the development and production of these life-saving therapies through innovative technology. Our expertise in advanced therapies, including viral vectors, cell therapies, and RNA-based therapies, enables us to support the entire development journey, from early-stage research to commercial manufacturing.

www.cytivalifesciences.com

Caralynn Nowinski Collens, M.D., CEO

Oct. 6 | 9:30am | FLW Ballroom G

Chicago, IL

(Private)

Dimension Bio is a biotechnology company pioneering regenerative therapeutics. We design life-saving solutions that restore organ function lost to disease, trauma, or aging. Our bioinspired cell delivery system, BioNidum, fuses materials science, 3D-printing, and digital manufacturing to transform cells into implantable, tissue-like therapies. By providing a three-dimensional architecture that promotes rapid vascularization and seamless tissue integration, our tissue therapeutics create the conditions cells need to survive, function, and persist. The result is a more durable and effective therapeutic response—unlocking the curative potential of cell therapies. Our lead program targets liver failure, providing immediate supplemental liver function to stabilize patients in crisis. By enabling the native liver to recover, it offers an alternative to transplant and fills a critical gap for those with limited treatment options.

www.dimension.bio

Flagg Flanagan, CEO and Chairman

Oct. 6 | 11:45am | FLW Ballroom F

Salt Lake City, UT

(Private)

DiscGenics is a privately held, late-stage clinical biopharmaceutical company focused on developing regenerative therapies for musculoskeletal degeneration. Its lead product candidate, IDCT (injectable disc cell therapy, or rebonuputemcel), is a single-injection biologic treatment designed to halt the progression of lumbar disc degeneration and regenerate the disc from within. The active component of IDCT is a live, manufactured progenitor cell population derived from donated adult human intervertebral disc tissue, delivered via a viscous carrier. A successful combined Phase I/II clinical study of IDCT has been completed, and the company is preparing for a U.S.-based Phase III trial targeting pain and disability associated with lumbar disc disease. IDCT has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration. To maintain control over quality, compliance, cost, and timelines, DiscGenics has built and validated in-house manufacturing capabilities and is advancing a next-generation allogeneic cell platform for future indications.

www.discgenics.com 

Gilmore O’Neill, President and CEO

Oct. 6 | 11:30am | FLW Ballroom F

Cambridge, MA

(NASDAQ: EDIT)

As a pioneering gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision in vivo gene editing medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines.

www.editasmedicine.com

Philippe Chambon, M.D., Ph.D., CEO

Oct. 7 | 11:15am | FLW Ballroom G

Paris, France

(Private)

EG 427 is the global leader in non-replicating HSV-1 (nrHSV-1) vector technology in neurology. EG 427 has started a phase 1b/2a study in the US with its lead candidate, EG110A, in patients with neurogenic detrusor overactivity-related incontinence. EG110A is being developed to addresses multiple severe bladder diseases, including overactive bladder (OAB), and has the potential to be a major improvement over existing therapies, resulting in better care for patients and lower costs for healthcare systems. The company’s unique HERMES platform delivers pinpoint neurotherapeutics to treat prevalent diseases of the peripheral and central nervous system. Its vectors can achieve focal transduction in specific regions and then selective expression of transgenes in targeted subsets of neurons thanks to the control of sophisticated regulatory elements. With demonstrated clinical safety and possible repeated dosing, the large payload capacity of nrHSV-1 vectors allows for versatile DNA delivery for smarter genetic medicine.

www.eg427.com

Michael Paglia, Chief Technology Officer, BaseCamp

Oct. 6 | 10:00am | FLW Ballroom G

Waltham, MA 

(Private) 

ElevateBio is a technology-driven company built to power transformative genetic medicines today and for many decades to come. The Company commercializes its enabling technologies, manufacturing capabilities, and industry-leading expertise through partnerships to accelerate development across a breadth of therapeutic approaches and modalities. The ElevateBio ecosystem combines Life Edit, its gene editing and R&D technology business, with BaseCamp®, its end-to-end genetic medicine cGMP manufacturing and process development business, to accelerate the discovery and development of advanced therapeutics. ElevateBio aims to be the dominant engine inside the world’s greatest scientific advancements harnessing human cells and genes to alter disease.

www.elevate.bio 

Daniel Leblanc, Chief Technology Officer

Oct. 6 | 4:45pm | FLW Ballroom G

Boston, MA 

(Private) 

Ensoma is developing potentially curative medicines for genetic diseases, immune disorders and cancer through precision in vivo cellular engineering. Our platform combines class-leading proprietary base editing and high-efficiency gene integration systems with high-capacity virus-like particles (VLPs) with an aim of provide one-time, durable genetic medicines in an outpatient procedure. The delivery system is based on VLPs that preferentially bind to hematopoietic stem cells (HSCs), efficiently deliver DNA to the nucleus while minimizing delivery other cell types, including the liver. With a 35-kilobase cargo capacity, these VLPs can carry a diverse range of sophisticated genomic engineering tools capable of changes from single base edits to large multi-gene insertions, along with control elements for HSC-lineage cell specific expression. Ensoma is supported by top-tier investors and a passionate team committed to a bold, global vision for genomic medicines. Ensoma is based in Boston.

www.ensoma.com 

Sanjeev Luther, President & CEO

Oct. 7 | 2:45pm | FLW Ballroom F

Cambridge, MA

(NASDAQ: ERNA)

Precisely Engineered Cells. Powerful Therapeutics. At Eterna we believe the body holds powerful tools for healing. Our job is to help reactivate them. Through innovative stem cell therapies, we’re breaking through the biological defenses of cancer and autoimmune disease what illness tries to erase: strength, resilience, and hope. Our core technology focuses on engineering induced pluripotent stem cell (iPSCs) and transforming them into induced mesenchymal stem cells (iMSCs). Ernexa’s allogeneic synthetic iMSCs provide a scalable, off-the-shelf treatment solutions without needing patient-specific cell harvesting. ERNA-101 is the company’s lead cell therapy product, designed to activate and regulate the immune system’s response to recognize and attack cancer cells. ERNA-102 is acell therapy product designed to target inflammation and treat autoimmune disease. The company’s initial focus is to develop ERNA-101 for the treatment of ovarian cancer and ERNA 102 for RA.

Ernexatx.com

Allison Hubel, Ph.D., Chief Scientific Officer

Oct. 7 | 5:45pm | FLW Ballroom G

Minneapolis, MN

(Private)

Evia Bio is a life sciences tools and services company dedicated to delivering better and safer therapeutics for the Cell and Gene Therapy community. Evia Bio offers Cryo Engineering Services to help assess and optimize cryopreservation protocols and solutions for CDMOs and cell therapy developers. Evia is also developing and commercializing DMSO-free and DMSO-minimized solutions to mitigate toxicity and improve post-thaw performance while reducing manufacturing complexities and patient risk. Evia Bio was founded by leading cryobiologist Allison Hubel, PhD. We are actively partnering with cell therapy leaders to reduce DMSO, improve therapeutic efficacy, and establish robust cryopreservation processes.

www.eviabio.com

Andreas Scheel, Ph.D., Executive Vice President, Head of Cell Therapy

Oct. 6 | 11:00am | FLW Ballroom G

Hamburg, Germany

(NASDAQ:EVT)

Evotec is a biotechnology company driving innovation in drug discovery and development. Combining cutting-edge science, AI-driven innovation, and proprietary technologies such as PanOmics and iPSC, we accelerate the journey from concept to cure. With tailored partnering models, we work with Top 20 Pharma, biotechs, academia, and healthcare stakeholders. Within cell therapy, Evotec offers a fully integrated, end-to-end platform to advance iPSC-based therapies from inception to clinical application. This includes in-house GMP manufacturing capabilities for clinical supply. Evotec’s platform is among the most comprehensive in the industry, encompassing cell types for anti-tumor therapies (NK cells, macrophages, αβ T cells, γδ T cells), regenerative therapies (beta cells, cardiomyocytes, photoreceptors) and immune-modulation therapies. Evotec maintains strategic partnerships with companies such as Sernova and Novo Nordisk. Additionally, Evotec provides integrated drug development solutions, including safety assessment, CMC, process and analytical development, regulatory and clinical services for both autologous and allogeneic cell therapy candidates.

www.evotec.com

Thomas VanCott, Ph.D., CEO

Oct. 6 | 11:30am | FLW Ballroom G

San Diego, CA

(Private)

As a strategically focused CDMO, Excellos combines the best of both worlds: the tailored, consultative support of a boutique firm with the scientific and operational strength needed to support complex, high-stakes programs. We offer bespoke solutions across the full development lifecycle, from donor-sourced starting material to final product characterization. Our model is intentionally focused: we’re structured to move quickly, collaborate deeply, and adapt to your unique needs without compromising quality or scalability. With a foundation rooted in clinical experience, a close partnership with the San Diego Blood Bank, and a culture of scientific rigor, Excellos brings both precision and flexibility to every engagement — helping our partners accelerate progress with confidence.

www.excellos.com

David Millette, CEO

Oct. 7 | 3:15pm | FLW Ballroom F

Montreal, Canada

(Private)

ExCellThera is a late-stage biotechnology company focused on advancing next-generation cell therapies. Its lead product, Zemcelpro®, is a patented, one-time curative treatment designed for patients with hematologic malignancies who face limited or ineffective therapeutic options. This includes individuals without suitable donors or those with high-risk malignancies that respond poorly to stem cell transplants. Zemcelpro recently received a positive opinion from the European Medicines Agency (EMA), positioning it for marketing authorization and initial commercial launch in the second half of 2025.

www.excellthera.com 

Hamid Khoja, Ph.D., Chief Scientific Officer

Oct. 7 | 2:15pm | FLW Ballroom F

Houston, TX

(NASDAQ:FBLG)

FibroBiologics is a clinical-stage biotechnology company focused on developing and commercializing fibroblast cell-based therapies for patients suffering from chronic diseases. Our proprietary fibroblast platform serves as the foundation for developing biologic cures and treatments for chronic diseases, including wound healing multiple sclerosis, degenerative disc disease, psoriasis, cancer, and anti-aging extension of life applications such as thymic involution reversal. Currently, FibroBiologics holds 240+ U.S. and International issued/pending patents across a variety of clinical pathways, including wound healing, multiple sclerosis, cartilage repair, psoriasis, degenerative disc, cancer, and extension of life programs.

www.FibroBiologics

Frederic Padilla, Ph.D., Director, Gene and Cell Therapy Program

Oct. 8 | 10:30am | FLW Ballroom G

Charlottesville, VA

(Private)

Focused Ultrasound Foundation: Enabling Targeted Delivery for Gene and Cell Therapies
The Focused Ultrasound Foundation is a non-profit organization pioneering noninvasive therapeutic delivery using focused ultrasound—an enabling platform that transiently opens biological barriers such as the blood-brain barrier (BBB), enhancing the precision and efficacy of gene and cell therapies.
We support translational research across multiple organ systems, with a strategic focus on neurological and oncological indications, as well as large-organ delivery. Our programs bridge academic discovery and industry innovation with the goal of catalyzing first-in-human trials. The Foundation’s portfolio spans viral and non-viral vectors, cell therapies, and immuno-oncology.
By addressing key delivery challenges, we aim to expand the reach and impact of regenerative medicine. Our presentation will highlight recent milestones, collaborative models, and opportunities to leverage focused ultrasound as a scalable, noninvasive delivery modality for gene and cell therapy developers.

www.fusfoundation.org

John Maslowski, President and CEO

Oct. 6 | 2:45pm | FLW Ballroom G

Columbus, OH

(TYO: 2802)

Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a gene therapy contract development and manufacturing organization (CDMO) enabling access to life-changing gene therapies by bringing them from concept to reality. Forge’s 200,000 square foot facility, the Hearth, is headquartered in Columbus, Ohio, and houses 20 custom-designed cGMP suites with 20,000L of bioreactor capacity. Forge’s end-to-end, scalable plasmid and AAV manufacturing services include research-grade manufacturing, process and analytical development, cGMP manufacturing, fill and finish, and integrated regulatory support to help accelerate the timelines of transformative medicines for patients with genetic diseases.

www.forgebiologics.com 

Amicia Elliott, Ph.D., Vice President of Product

Oct. 7 | 4:30pm | FLW Ballroom G

Austin, Texas 

(Private) 

Form Bio is the leading provider of AI/ML-powered in silico solutions that accelerate and de-risk AAV gene therapy development. We deliver data-driven insights into AAV genome integrity to identify risk profiles across the development pipeline—enabling better characterization of candidate vectors. Our proprietary models further optimize safety, efficacy, and manufacturability by enabling smarter decisions earlier in development. Together, these capabilities help teams reduce time, cost, and risk on the path to lead candidate selection.

www.formbio.com 

Zoya Gluzman-Poltorak, Ph.D., CEO and Co-Founder

Oct. 8 | 10:00am | FLW Ballroom G

San Francisco, CA

Forta Bio is a novel California-based biotech company developing precision DNA therapies that selectively eliminate disease-driving immune, cancer and other pathogenic cells— without compromising healthy immunity. Our proprietary cell targeting 6-thio-deoxyguanosine oligonucleotides (6tdGO) platform uses a dual mechanism of action to directly disrupt telomeres in specific telomerase positive (TERT+) pathogenic cells while activating the body’s immune response. This approach is uniquely suited for treating aggressive cancers, autoimmune and age-related diseases. We focus on high-burden, underserved conditions — particularly those affecting women and children — and pursue accelerated regulatory pathways. Backed by validated science from City of Hope and led by an experienced team with proven therapeutic development and deal-making track record, Forta Bio is on a mission to bring smarter, safer, and more targeted therapies to patients who need them most.

www.forta.bio

Harith Rajagopalan, M.D., Ph.D., Co-Founder and Chief Executive Officer

Oct. 7 | 10:15am | FLW Ballroom F

Burlington, Massachusetts

(NASDAQ: GUTS)

Fractyl Health is a metabolic therapeutics company pioneering pattern-breaking treatments for obesity and type 2 diabetes (T2D), with the goal of transforming metabolic disease treatment from chronic symptomatic management to durable disease-modifying therapies that target the organ-level root causes of disease. RJVA-001, a candidate from Fractyl’s Rejuva gene therapy platform, is delivered via an endoscopic ultrasound-guided infusion into the pancreas. The construct is designed to express glucagon-like peptide-1 (GLP-1) locally in beta cells using nutrient-responsive control, potentially enabling physiologic hormone secretion without the high circulating levels that contribute to side effects seen with systemic GLP-1 therapy. This distinction opens the door to efficacy that matches or exceeds drugs with potentially superior tolerability profiles due to low circulating levels of drug. Fractyl Health submitted the first Clinical Trial Application (CTA) module to regulators and, if authorized, expects to dose T2D patients and report preliminary data in 2026.

www.fractyl.com

Rebecca Selzer, Ph.D., Executive Vice President, R&D

Oct. 6 | 9:15am | FLW Ballroom F

Madison, WI

(Private)

FUJIFILM Cellular Dynamics, Inc. (FCDI) is a leading developer and manufacturer of human induced pluripotent stem cells (iPSCs) utilized in drug discovery, contract development and manufacturing services, and cell therapies. FCDI is using its expertise in iPSC technologies to develop robust cell therapeutics products to address unmet medical needs. For its partners, FCDI utilizes its iPSC platform to advance the progress of therapeutic candidates in the clinic and provides contract development and manufacturing (CDMO) services. In addition to cell therapy, FCDI also offers life science research tools and custom cell services including the company’s inventoried iCell® products, which are available in almost any cell type and are sourced from multiple cell lines that can be applied for target identification as well as toxicity testing. FCDI’s goal is to leverage the vast utility of iPSCs to advance human health and improve the quality of life for patients around the world.

www.fujifilmcdi.com

Azadeh Golipour, Ph.D., Head of Technical Operations, Senior Vice President

Oct. 8 | 9:30am | FLW Ballroom F

Cambridge, MA

(Private)

GC Therapeutics (GCTx) is a biopharmaceutical company created to scale and unlock the next generation of cell therapy-based medicines. GCTx’s induced pluripotent stem cell (iPSC) programming platform, TFome™, integrates the latest advancements in synthetic biology, gene editing, cell engineering and machine learning to overcome the therapeutic development and scaling complexities associated with cell therapy and improve patient access across a broad range of disease areas. GCTx’s initial focus is on developing first-in-class and best-in-class cell therapies for gastrointestinal, neurological and immunological diseases. Founded in 2019, GCTx is based in Cambridge, MA and has raised $75 million to date from leading life sciences investors.

www.gc-tx.com

Imen Mestiri, Ph.D., Business Development Manager

Oct. 8 | 9:15am | FLW Ballroom G

Évry, France

(Private)

A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a unique non-profit organization created by a patient association, the AFM- Telethon. A first gene therapy drug, to which Genethon contributed, has obtained marketing for spinal muscular atrophy. With 200+ scientists and professionals, Genethon is pursuing its mission to bring life-changing therapies to patients suffering from rare genetic diseases. Thirteen products resulting from Genethon’s research are in clinical trials for liver, blood, immune system, muscle and eye, diseases. A further seven products are in the preparation phase for clinical trials over the next five year.

www.genethon.com 

Sarah Busch, Ph.D., Chief Scientific Officer

Oct. 6 | 2:30pm | FLW Ballroom F

Tokyo, Japan 

(TSE: 4593) 

Healios is a clinical stage biotechnology company harnessing the potential of stem cells for regenerative medicine. Healios is a pioneer in the development of regenerative medicines in Japan and owns proprietary, global platforms utilizing both somatic stem cells and iPS cells. In the somatic stem cell field, Healios is developing invimestrocel (HLCM051), a proprietary cell product comprised of multipotent adult progenitor cells (“MAPCs”) derived from the bone marrow of healthy adult donors. Healios is advancing invimestrocel globally for ischemic stroke, ARDS, and trauma. The company has confirmed its path to conditional approval in Japan for the use of invimestrocel for ARDS and is preparing to file for approval and for commercial launch.

www.healios.co.jp 

Sean Ainsworth, CEO and Chairman of the Board

Oct. 7 | 1:45pm | FLW Ballroom F

Seattle, WA 

(Private)

Immusoft is developing a cutting edge approach to sustained delivery of protein therapeutics using a patient’s own cells. The approach is called Immune System Programming (ISP™). ISP entails collecting a type of the patient’s immune cells, called B cells. In response to immune stimulation, B cells can turn into a biofactory state known as a plasma cell. Plasma cells manufacture and secrete thousands of antibodies per second. We harness this biofactory capability of the plasma cell by programming B cells to produce a given protein therapeutics. Once we have programmed the B cells we expand the number of them many times and differentiate them into plasma cells (or, stated simply, coax them into the biofactory state) that produce massive amounts of our therapeutic protein. Thereafter we infuse the ISP™ programmed cells back into the same patient, where they take up residence and produce therapeutic proteins for extended periods of time.

www.immusoft.com

Nick Manusos, CEO

Oct. 7 | 2:00pm | FLW Ballroom G

San Diego, CA

(Private)

Kenai Therapeutics is a very soon to be clinical stage, neurodegenertive disease focused, biotechnology company utilizing an iPSC platform to replace the lost neurons in the central brain caused by Parkinson’s Disease (RNDP-001). RNDP-001 is an allogeniec, cryopreserved alternative. Leveraging best in class pre clinical data and partnerships with leaders in the field such as FujiFilm Cellular Dynamics (iPSC’s) and ClearPoint Neuro (surgical device), Kenai successfully completed an $82mm series A financing in February of 2024. Use of proceeds for the series A are focused on Kenai’s phase I trial which is set to initiate prior to the Mesa Meeting. Kenai’s pipeliine further expands on the lead program (RNDP-001) and addresses additonal unmet needs across the Parkinson’s disease patient spectrum such as cognition and inflammation. Also, Kenai is part of the Lilly Gateway Lab’s ecosystem and is based in San Diego.

www.kenaitx.com

Miguel Forte, M.D., Ph.D., CEO

Oct. 6 | 2:30pm | FLW Ballroom G

Paris, France

(Private)

Kiji Therapeutics is a private company in between Paris and Madrid, created in 2023.Kiji Tx is developing multi-gene engineered stem cells for the treatment of autoimmune diseases. The vision is to leverage iPSC sourcing and gene engineering to optimize the therapeutic effect of MSCs, addressing their shortcomings and delivering a platform that can address multiple indications for unmet medical needs.The first asset is planned to be in the clinic for refractory aGvHD in 12 months to be followed by the second asset targeting IBD and Psoriasis.Kiji Tx is currently pursuing an extension of the seed to deliver clinical data and progress with the iPSC derived gene engineered MSC platform for additional indications.

www.kiji-tx.com

Warner Biddle, CEO

Oct. 7 | 1:30pm | FLW Ballroom G

Emeryville, CA

(NASDAQ: KYTX)

Kyverna Therapeutics is a clinical-stage biopharmaceutical company focused on liberating patients through the curative potential of cell therapy. Kyverna’s lead CAR T-cell therapy candidate, KYV-101, is advancing through late-stage clinical development with registrational trials for stiff person syndrome and myasthenia gravis, and two ongoing multi-center Phase 1/2 trials for patients with lupus nephritis. The company is also harnessing other KYSA trials and investigator-initiated trials, including in multiple sclerosis, to inform the next priority indications for Kyverna to advance into late-stage development. Additionally, its pipeline includes next-generation CAR T-cell therapies in both autologous and allogeneic formats, including efficiently expanding into broader autoimmune indications and the potential to increase patient reach with KYV-102 using its proprietary whole blood rapid manufacturing process.    

www.kyvernatx.com 

P. Peter Ghoroghchian, M.D., Ph.D., CEO

Oct. 8 | 10:15am | FLW Ballroom F

Philadelphia, PA

Latus is a biotechnology company dedicated to addressing devastating CNS and peripheral diseases via gene therapy. The Company is advancing an innovative therapeutics pipeline based on novel AAV capsid variants with potency and specificity. Latus is powered by a diverse team of visionary scientists, experienced clinicians, and leading industry executives. The Company has offices in Philadelphia, PA and in the Seaport in Boston, MA.

www.latusbio.com

Tom Payne, Ph.D., Chief Operating Officer

Oct. 7 | 12:15pm | FLW Ballroom F

London, UK

(Private)

Laverock Therapeutics is a gene-silencing company with a uniquely powerful technology which harnesses the cell’s natural gene regulatory mechanisms to develop a new generation of programmable advanced therapies to tackle major diseases. Laverock is applying its technology through its own pipeline – targeting regenerative medicine and oncology – and through partnerships. Laverock’s product focus encompasses iPSC derived macrophages engineered to polarise to a pro-inflammatory state in the tumour microenvironment, and donor-derived CAR-T cells for hard-to-treat cancers where multiple check-point pathways can be conditionally silenced (for example, only when the CAR-T cell is engaged with its target) providing improved efficacy without negative affects on cell fitness. In addition, we are developing the platform for in vivo editing to achieve tunable gene silencing in genetic diseases where gene dosage needs to be tightly controlled (over or under expression is detrimental).

www.laverocktx.com 

Amy Pooler, Ph.D., Senior Vice President, Research and Development

Oct. 6 | 5:00pm | FLW Ballroom G

Durham, NC 

(Private) 

Life Edit is ElevateBio’s next-generation gene editing and R&D technology business, offering one of the world’s largest and most diverse libraries of RNA-guided nucleases (RGNs), base editors, and reverse transcriptase (RT) editors that provide flexible editing and unprecedented access to the genome. The Life Edit gene editing platform is available to make the entire spectrum of edits – from small insertions or deletions to rewriting sections of DNA – to lead the genetic medicine revolution in ex vivo and in vivo therapeutic development to treat and potentially cure disease. These technologies can be commercialized through various partnership structures, ranging from exclusive licensing and R&D collaborations to technology democratization deals. Each technology is also integrated within ElevateBio’s ecosystem, enabling the Company to discover, develop, manufacture, and commercialize an unprecedented, broad spectrum of cell and gene therapies.

www.lifeeditinc.com 

Stella Vnook, Ph.D., CEO

Oct. 7 | 10:15am | FLW Ballroom G

Kansas City, MO

(Private)

Likarda is a biotech company pioneering next-generation drug and cell therapy delivery using its proprietary Core-Shell Spherification™ (CSS™) platform. This injectable hydrogel technology enables precise, localized, and extended-release delivery of cells, biologics, peptides, and small molecules—improving therapeutic durability, reducing immune responses, and enhancing patient outcomes. Likarda’s platform is uniquely designed to support subcutaneous administration, cryopreservation and dry ice shipping, and immune protection for allogeneic or off-the-shelf therapies. With applications across regenerative medicine, oncology, metabolic disease, and rare disorders, Likarda is partnering with biopharma and advanced therapy developers to solve their toughest delivery challenges and accelerate therapies to market.

www.likarda.com

Yasunori Aizawa, Ph.D., Co-Founder and Chief Scientific Officer

Oct. 7 | 9:15am | FLW Ballroom G

Tokyo, Japan

(Private)

Logomix is transforming iPSC engineering with Geno-Writing™, a groundbreaking genome engineering platform, and currently focusing on iPSC development for Type 1 Diabetes (T1D) treatment. Geno-Writing™ platform enables precise, large-scale genomic rewriting. This allows bi-allelic modification of loci up to 100kb and stable integration of up to 12 transgenes within 2.5 months, setting a new benchmark in iPSC engineering.
Complementing this, our semi-automated CloneMax system simultaneously creates over 5,000 distinct iPSC lines with varying genome designs, accelerating the identification of optimal therapeutic candidates. This powerful synergy addresses key T1D challenges: achieving robust immune tolerance and significantly enhancing β-cell differentiation and insulin secretion. Logomix’s integrated platforms offer a robust solution for next-generation, allogeneic iPSC-derived cell therapies, promising more effective and widely accessible T1D treatments

www.logomix.bio

Leah DiMascio, Ph.D., Chief Operating Officer  

Oct. 7 | 10:00am | FLW Ballroom F

Houston, Texas

(Private)

March Biosciences is dedicated to addressing challenging cancers unresponsive to current immunotherapies. Its lead asset, MB-105, is a CD5-targeted CAR-T cell therapy currently in Phase 2 clinical development for patients with relapsed / refractory T-cell lymphoma. The company has raised over $52 million to date, inclusive of venture financing, support from the Cancer Prevention & Research Institute of Texas (CPRIT), and the NIH SBIR program.

www.march.bio

Aric Bopp, Executive Director, Discovery Oasis

Oct. 8 | 9:30am | FLW Ballroom G

Rochester, MN

Mayo Clinic is the largest integrated, not-for-profit medical group practice in the world and is focused on building the future of healthcare and conducting research that turns into earlier diagnoses and new cures. At Mayo Clinic, experts work together to solve the most challenging unmet needs of patients and an unwavering drive to create better medical care has earned Mayo Clinic more top rankings for high-quality patient care than any other health care organization.

www.mayoclinic.org

Antonio Lee, Ph.D., CEO

Oct. 6 | 3:15pm | FLW Ballroom F

Cambridge, MA

(Private)

MEDIPOST (KOSDAQ 078160) is headquartered in South Korea, with its wholly-owned subsidiaries in the U.S. (MEDIPOST Inc.) and Japan (MEDIPOST K.K.), as well as a CGT CDMO (OmniaBio Inc.) joint-venture subsidiary based in Ontario, Canada. CARTISTEM® (allogeneic Umbilical Cord Blood-derived MSCs with hyaluronic acid composite administered surgically) received BLA approval in 2012 in Korea, for the treatment of knee articular cartilage defects (ICRS grade IV) in patients with osteoarthritis (K&L grade II or III) without age-limit. Over 33,500+ patients have been treated with CARTISTEM® in Korea with an excellent long-term safety and efficacy profile. Currently, on-market Korean RWE study is underway for the demonstration of long-term health-economic benefit. In Japan, CARTISTEM® Phase III pivotal trial will be completed in 2025, while the Phase III pivotal trial in the U.S. will begin in 2026.
MEDIPOST’s other clinical-stage MSC pipelines focus on early-stage osteoarthritis (OA), broncho-pulmonary dysplasia (BPD) and Alzheimer’s disease (AD).

www.medi-post.com 

Madhusudan Peshwa, Ph.D., CEO

Oct. 6 | 5:45pm | FLW Ballroom G

Jacksonville, FL

(Private)

MKC Biotherapeutics is a therapeutics company, operationalized in June 2024. The company is focused on regenerative medicine applications using “disease modifying” cell-based drugs for treatment of chronic & age-related diseases. Our business model is to identify & in-license therapeutic platforms / product assets with demonstrated human safety & efficacy data (with durable clinical benefit) and undertake any required bridging clinical / regulatory development for purposes of commercial marketing approval in US & Europe. Our current Product Portfolio consists of our first in-licensed IP, technology & know-how along with clinical data (pivotal trials & RWE from commercial treatment) for an allogeneic Mesenchymal Stromal Cell (MSC) platform that has resulted in regulatory approval for three disease indications in India. The licensed IP, technology and knowhow provides robust, scalable, cost-effective, industrial scale manufacture with consistent biological activity and potency. These attributes enable commercial delivery of disease-modifying cell-based drugs along conventional pharmaceutical drug product margins. We have elected to pursue knee osteoarthritis as our lead indication for US commercial development and anticipate IND filing in mid-2026 for an adaptive Phase 2/3 trial design as pivotal study for BLA filing / licensure.

mkcbiotherapeutics.com

Daniel Abate-Daga, Ph.D., Scientific Director, Moffitt Cell Therapy Facility

Oct. 6 | 10:00am | FLW Ballroom F

Tampa, Florida

(Private)

Moffitt Cancer Center is a renowned National Cancer Institute-designated Comprehensive Cancer Center based in Tampa, Florida. It is dedicated to the prevention and cure of cancer through research, education and patient care. Its unique ecosystem integrates preclinical development, translational research, and clinical care in collaboration with world-renowned scientific experts and leaders in the field of Cell and Gene Therapy. As part of this ecosystem, Moffitt’s onsite Cell Therapy and Gene Engineering facility provides GMP manufacturing of cell therapies and viral vectors for clinical trials, supporting both Moffitt faculty and industry partners. With over 15 years of expertise of translating cell therapy assets into the clinic, Moffitt has manufactured and supported over 50 INDs. Moffitt’s cell manufacturing expertise includes TILs, CAR-Ts (alpha beta & gamma delta), TCR-T, Tregs, and DC vaccines.

www.moffitt.org/cell-manufacturing

Michael Muhonen, M.D., Chief Medical Officer

Oct. 7 | 4:00pm | FLW Ballroom G

New York, NY

(Private)

Myrtelle is a patient-centered, innovation-driven gene therapy company transforming the treatment of neurological diseases. Our mission is to develop and deliver novel treatments for devastating myelin-based disorders of the central nervous system for which few, if any, treatment options exist. Myrtelle’s clinical program for Canavan disease is the first gene therapy to specifically target oligodendrocytes. rAAV-Olig001-ASPA has been selected for FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program and granted Orphan Drug, Fast-Track and Rare Pediatric Disease Designations for the treatment of Canavan disease in the US, with similar designations in Europe. Myrtelle recently announced published interim results of its Phase 1/2 Clinical Trial of rAAV-Olig001-ASPA for Canavan disease in Nature Medicine reporting the investigational oligodendrocyte targeted drug leads to decreased N-acetylaspartate (NAA) levels and increased brain myelin volume, resulting in promising functional improvements in children with Canavan disease.

www.myrtellegtx.com 

Samarendra Mohanty, Ph.D., President

Oct. 6 | 12:15pm | FLW Ballroom G

Dallas, TX

(Private)

Inherited retinal dystrophies (IRDs) are devastating neurodegenerative conditions resulting in progressive, permanent vision loss. Over 8000 different mutations in more than 300 genes cause IRDs. No effective treatment for IRD patients with severe vision loss has been approved to date. Nanoscope Therapeutics was founded in Dallas (TX) to change that by advancing a novel gene therapy as a life-changing treatment for these patients, regardless of mutation, leaving no patient behind. Nanoscope’s clinical pipeline aims to provide return-to-life vision restoration in patients with Retinitis Pigmentosa (RP), Stargardt Macular Degeneration (SD), which account for over 80% of IRDs. Nanoscope has received Orphan Drug and Fast Track designations from the FDA for MCO-010 (sonpiretigene isteparvovec) in RP and SD. Following positive end-of-study results from the RESTORE multicenter, randomized, double-masked, sham-controlled clinical trial for RP, the company announced its plan to initiate a BLA submission for MCO-010 to treat RP in 2025.

www.nanostherapeutics.com

Jeff Walsh, CEO

Oct. 7 | 12:15pm | FLW Ballroom G

Boston, MA

(Private)

nChroma Bio is a genetic medicines company committed to addressing the limitations of existing therapies through a disease-first approach. By combining programmable in vivo delivery and gene-regulating technologies, nChroma is designing optimal solutions to deliver precise, potent and durable treatments for patients with high unmet needs. nChroma’s lead candidate, CRMA-1001, is a near clinical-stage, liver-directed epigenetic therapy in development as a potential functional cure for chronic hepatitis B. Guided by a world-class team at the forefront of genetic medicine, founded by renowned pioneers in the field, and supported by top-tier investors, nChroma is redefining targeted in vivo genetic medicine with the initial goal of treating diseases affecting the liver, blood, and central nervous system.

www.nchromabio.com

Abhi Gupta, Executive Vice President, Commercial and Business Development

Oct. 6 | 4:00pm | FLW Ballroom G

Malvern, PA

(NASDAQ: OCGN)

Ocugen is a pioneering biotechnology company focused on discovering, deveoping, and commercializing novel gene therapies to address major blindness diseases and offer hope for patients across the globe. We are making an impact on patients lives through courageous innovation–forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach.

www.ocugen.com 

George Magrath, M.D., CEO

Oct. 6 | 9:15am | FLW Ballroom G

Durham, NC

(NASDAQ: IRD)

Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing important new therapies that target mutations in genes causing loss of vision and Inherited Retinal Diseases (IRDs) and other retinal and refractive disorders. Our portfolio spans approved drugs, Ph III programs, as well as a deep gene therapy pipeline in bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP) to aim to solve inherited blinding conditions. Our current clinical focus is on Leber Congenital Amaurosis (LCA) type 5, a type of LCA that deals with the LCA5 gene which encodes lebercilin, a protein involved in centrosomal or ciliary functions. This program recently received a RMAT designation from FDA. Our bestrophinopathy program is kicking off in 2025 in the US, with other assets in RDH12 building a path towards IND later this year. It’s estimated that over 300,000 patients are waiting for treatments for their individual genetic conditions, and more than 280 genes are known to cause inherited retinal diseases. Opus’ mission is to pave a proven, derisked, efficient path to the clinic for these urgently needed new therapies. Our IRD programs address mutations in genes that cause ophthalmic disease based on world-class science from gene therapy pioneers at the University of Pennsylvania (including the lab of Jean Bennett, MD, PhD), Harvard Medical School, and the University of Florida.

www.opusgtx.com

Eric Soller, Ph.D., Chief Business Officer

Oct. 7 | 1:30pm | FLW Ballroom F

Seattle, WA

(Private)

Outpace Bio is a clinical-stage biotechnology company reprogramming cell therapies to overcome the challenges in solid tumors. By integrating unrivaled AI-powered protein design with advanced T cell engineering, Outpace creates programmable medicines designed to function more safely, effectively and at scale. The company’s modular platform—developed by scientific leaders in computational protein design and synthetic biology—builds on the breakthroughs recognized by the 2024 Nobel Prize in Chemistry. Outpace’s technologies are designed to improve function inside patients – enhancing trafficking, functional persistence, and tumor killing while minimizing toxicity. Backed by a world-class syndicate of investors, Outpace is advancing a pipeline of next-generation CAR and TCR T cell therapies with the potential to deliver curative outcomes for patients who have limited treatment options. The company is headquartered in Seattle, Washington.

www.outpacebio.com

Carter Cliff, CEO

Oct. 7 | 12:00pm | FLW Ballroom G

San Diego, CA

(Private)

Papillon Therapeutics is a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited disease. We are advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited disease, targeting neurodegenerative disorders. Our clinical stage program, a gene modified hematopoietic stem and progenitor cell (HSPC) therapy for treatment of Cystinosis, reported positive data in a Phase 1/2 clinical trial and is now licensed to Novartis who are advancing “DFT383 in Pediatric Participants With Nephropathic Cystinosis”. Our lead preclinical stage program targeting Friedreich’s ataxia (PPL-001) aims to submit IND within a year and has received funding from CIRM. Our second preclinical stage program targeting Danon disease (PPL-002) will hold Pre-IND this year and received funding from CIRM.

www.papillon-tx.com

Linda Tempelman, Ph.D., CEO

Oct. 7 | 12:00pm | FLW Ballroom F

Ithaca, NY

(Private)

Do you know someone with Type 1 Diabetes? Do you know someone who has had a cell or gene therapy? Persista Bio is poised to bring cell therapies to type 1 diabetic patients as a curative therapy – no more injections, glucose sensors or long-term disabilities. Persista Bio brings a cell implant system to enable use of the breakthrough insulin secreting stem cells without immunosuppression. Persista will present its preclinical results showing diabetes cure in immunocompetent rats with the oxygenated cell encapsulation system. Persista Bio’s implantable cell implant system is a platform to deliver other allogeneic, long term cell therapies for chronic conditions such as enzyme disorders, inflammatory diseases and chronic pain. Persista Bio is a preclinical stage company founded by the inventors of technology licensed from Cornell University and Giner, Inc.

www.persistabio.com

Daniel Shelly, Ph.D., Chief Business Development Officer

Oct. 7 | 11:45am | FLW Ballroom G

Gdansk Poland

(NASDAQ: PTG)

Poltreg SA and its wholly owned Subsidiary, Immuthera, are pioneers in T-Regulatory cell therapy (Tregs). PolTREG is a fully integrated clinical stage company with extensive clinical experience with Tregs, a pipeline of advanced (modified, engineered, and Allogeneic) Treg and mRNA immunotherapies, and commercial stage cGMP cell manufacturing. Immuthera has recently launched in the US and will be bringing Treg cell therapies and other technologies licensed from PolTREG, into the US, starting with our Presymptomatic (Stage 1 & 2) T1D study (primarily in children as young as 6) and Progressive MS. We will follow with our CAR-Treg for ALS and MS and our mRNA immuno therapy for T1D.

www.poltreg.com

Cindy Atwell, Chief Development Officer and Chief Business Officer

Oct. 6 | 11:45am | FLW Ballroom G

Durham, NC

(NASDAQ: DTIL)

Precision BioSciences, Inc. (Nasdaq: DTIL), is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform designed with therapeutic safety, delivery, and control in mind. Using ARCUS, Precision is developing in vivo gene correction therapies for rare and infectious diseases.

www.precisionbiosciences.com 

Hilary Eaton, Ph.D., Chief Business Officer

Oct. 6 | 5:30pm | FLW Ballroom G

Emeryville, CA

(Private)

Profluent is an AI-first protein design company that aims to make biology programmable. Whereas biological discovery has largely depended on chance or traditional engineering approaches, Profluent is finally changing this with an AI-powered platform that learns from the underlying “grammar” of natural protein sequences at scale to design new proteins with specific functions for medicine, climate, and sustainable agriculture.
Profluent made history as the first company to edit the human genome with an AI-created, open-source gene editor, OpenCRISPR-1. It has since released one of the largest AI biological models to-date, which was used to design an ultra-compact gene editor and redesign 20 blockbuster antibody drugs.
As Profluent redefines traditional drug discovery, it is also transforming biopharma partnerships with a flexible model – enabling collaborators to license bespoke proteins (such as antibodies for BioSpecifics or gene editors for therapeutics) or tap into its foundational technology platform.

www.profluent.bio

Josefina, Nilsson. Ph.D., CEO

Oct. 6 | 10:15am | FLW Ballroom G

Stockholm, Sweden

(Private)

QuTEM, a global leader in GMP-certified Transmission Electron Microscopy (TEM) services from Stockholm, Sweden, specializes in nanoparticle quality control for the pharmaceutical industry. We transform TEM images of vaccines and gene therapy vectors into quantifiable data, directly linking to biological effects and safety profiles. Our proprietary, GMP-compliant Gridsee software uses artificial intelligence and convolutional neural networks to precisely recognize and classify nanoparticles. This allows us to accurately determine capsid content, genome length, and other critical quality attributes for vectors like AAV, adenoviral vector, LVV, EV, VSV, MVA, Liposome and LNP. By providing high-quality, traceable data on CQAs, QuTEM accelerates the development of next-generation treatments, enhancing the safety and efficiency of groundbreaking gene therapies worldwide.

www.qutem.com

Patrick Christmas, Chief Strategy and Legal Officer

Oct. 6 | 3:00pm | FLW Ballroom F

Rockville, Maryland

(NASDAQ: RGNX)

REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO’s AAV platform, including those receiving Novartis’ ZOLGENSMA®. REGENXBIO’s investigational gene therapies have the potential to change the way healthcare is delivered for millions of people.

www.regenxbio.com 

Richard Freed, CEO

Oct. 8 | 10:00am | FLW Ballroom F

Aurora, CO

(Private)

RheumaGen is a private biotechnology company engineering a new class of therapeutics to cure major autoimmune diseases. RheumaGen focuses on editing the human leukocyte antigen (HLA) gene, or “immune gene,” to develop one-time, curative cell and gene therapies. We are building a pipeline of treatments to address autoimmune diseases in which HLA alleles play a critical epidemiological role, including programs for multiple sclerosis, type 1 diabetes, and ankylosing spondylitis currently in development. In December 2024, we closed a $15M Series A financing that will support RheumaGen in completing a Phase I clinical trial of our lead program, RG0401, for treatment-resistant or refractory rheumatoid arthritis (RA). RheumaGen is headquartered in Aurora, Colorado.

www.rheumagen.com

Guarav Shah, M.D., CEO

Oct. 7 | 3:00pm | FLW Ballroom F

(NASDAQ: RCKT)

Rocket Pharmaceuticals is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket’s multi-platform approach and pipeline of LV hematology and AAV cardiovascular programs allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives.

www.rocketpharma.com 

Saeil Kang, CEO

Oct. | 2:15pm | FLW Ballroom G

Seoul, Korea

(KOSDAQ: 304360) 

S.Biomedics focuses on developing disease-specific cell therapies utilizing two proprietary, high-efficiency platforms. One of our core platforms, TED, enables robust and consistent differentiation of pluripotent stem cells into lineage-specific neural precursor cells. Using TED, we developed TED-A9, a cell therapy candidate for Parkinson’s disease comprising ventral midbrain dopaminergic precursors derived from human embryonic stem cells. A Phase 1/2a clinical trial (NCT05887466) was conducted in patients with moderate-to-severe Parkinson’s disease to assess safety and exploratory potential clinical outcomes. Twelve patients received bilateral putamen transplantation of a low (3.15 million cells) or high (6.30 million cells) dose of A9-DPC. No dose-limiting toxicities or graft-related adverse events were reported. At 12 months, improvements in motor function (off-medication MDS-UPDRS Part III and Hoehn & Yahr) were observed, especially in the high-dose group. These findings support the safety and therapeutic potential of TED-A9 in Parkinson’s disease.

www.sbiomedics.com/eng/

Tom Lowery, Ph.D., President and CEO

Oct. 7 | 9:30am | FLW Ballroom G

Newton, MA

(Private)

Satellite Biosciences is a liver cell therapy company with cutting-edge hepatocyte technologies aimed at addressing a broad range of severe liver diseases. We have two drug candidates with regulatory feedback and are entering the clinic in 2026. Our platform encompasses a proprietary primary hepatocyte expansion technology that allows dosing of thousands of patients from a single donor liver. Additionally, we specialize in stabilizing hepatocyte function to optimize for engraftment and durability as well as for long-term stability and shipment. Our therapies have the profile that more closely resembles a traditional drug than a typical cell or gene therapy: they are off-the-shelf, stable, worldwide shippable, ready-to-infuse, and have a very low cost-of-goods.

www.satellite.bio

David Parrot, Chief Financial Officer

Oct. 6 | 1:45pm | FLW Ballroom G

Alameda, CA

(Private)

Scribe Therapeutics is revolutionizing medicine by developing optimized in vivo CRISPR-based genetic medicines designed to become standard of care treatments for patients suffering from prevalent diseases, starting with cardiometabolic disease. The company is on a mission to build the first CRISPR-based therapeutics that are effective and safe enough to transform everyone’s lifetime risk for disease. Scribe’s CRISPR by Design™ approach engineers bacterial immune systems into a premier suite of genome and epigenome editing tools built for unique molecular advantages in activity, specificity, and deliverability, enabling the creation of therapies with a broader therapeutic window and safe for use as a preventative treatment. To broaden and accelerate the impact of its engineered CRISPR technologies for patients, Scribe strategically collaborates with world-leading pharmaceutical companies including Sanofi and Eli Lilly. Co-founded by Nobel Prize winner Jennifer Doudna and backed by leading life sciences investors, Scribe is engineering the future of genetic medicine.

www.scribetx.com

Jodi Cook, Ph.D., CEO and President

Oct. 7 | 11:00am | FLW Ballroom G

Cambridge, MA

(Private)

Skylark Bio is a gene therapy company working on targeted local delivery of gene therapies, initially focused on addressing pediatric genetic hearing loss, but with the potential to rapidly progress into other diseases. Genetic hearing loss is a devastating disorder that is diagnosed at birth and can impede speech and language development and subsequent learning, that can have life-long consequences. Through innovative gene therapy solutions, Skylark has a mission to create a world where anyone, anywhere can experience life in all its sensory dimensions.

skylarkbio.com

Brent Stead, Ph.D., CEO

Oct. 6 | 9:30am | FLW Ballroom F

Toronto, Ontario, Canada

(Private)

Specific Biologics Inc. is a venture-backed biotechnology company developing Dualase®, an industry-leading genome editing platform for precise, efficient, and programmable in vivo genome editing. Our initial therapeutic focus is on the precise collapse of pathogenic repeat expansions, leaving non-pathogenic numbers of repeats, using intrathecally delivered single AAV9 in a neurodegenerative disease. Its unique two-site mechanism enables the seamless removal, repair, or insertion of both small and large DNA sequences, offering broad therapeutic potential, coupled with single AAV or all-RNA delivery. Dualase® achieves best-in-class accurate editing efficiency with undetectable off-target effects which has been demonstrated in preclinical cell and animal models at diverse targets and indications with clear paths to clinic in CNS, liver and lung diseases.

www.specificbiologics.com

Mimi Healy, Ph.D., CEO

Oct. 7 | 4:45pm | FLW Ballroom G

Houston, Texas

(Private)

StimulusBio revolutionizes the development of cell and gene therapies through innovative product solutions that overcome the most critical barriers to widespread use: manufacturing time and cost. Our scalable technologies leverage biologically-directed surface engineering to drive consistent cellular expansion and streamline complex workflows to more efficiently translate discoveries into clinical solutions. The StimulusBio product pipeline centers around a programmable cellular interface that enables precise nanoscale control over cellular interactions, supporting reproducible outcomes and faster iteration in both research and clinical translation. In addition to development, our business-focused scientists guide companies seeking to commercialize their own technologies through the challenges of transitioning innovations to market. Our team brings deep expertise in business planning, product vision and operational development. By combining solution-driven laboratory innovation with expert market strategy, StimulusBio is committed to ensuring that great science successfully makes the leap from the bench to the bedside.

www.stimulusbio.com

Faraz Ali, CEO

Oct. 6 | 12:00pm | FLW Ballroom G

South San Francisco, CA

(NASDAQ: TNYA)

Tenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Tenaya’s is focused on advancing its two lead gene therapy candidates, TN-201 for MYBPC3-associated hypertrophic cardiomyopathy (HCM) and TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC). A third clinical-stage candidate, TN-301, a small molecule HDAC6 inhibitor intended for heart failure with preserved ejection fraction (HFpEF), and multiple early-stage programs in preclinical development were discovered internally. Tenaya has employed a suite of integrated internal capabilities, including modality agnostic target validation, capsid engineering and manufacturing, to generate its portfolio of genetic medicines aimed at the treatment of both rare genetic disorders and more prevalent heart conditions.

www.tenayatherapeutics.com

Hari Pujar, Ph.D., Chief Operations Officer

Oct. 7 | 5:30pm | FLW Ballroom G

Boston, Massachusetts

Tessera Therapeutics is pioneering Gene Writing™—a new genome engineering technology that writes therapeutic messages into the genome to treat diseases at their source. Our mission is to cure disease by writing in the code of life.

www.tesseratherapeutics.com

Jill Makin, Ph.D., Chief Scientific Officer

Oct. 6 | 5:00pm | FLW Ballroom F

Hampton, UK

(Private)

Touchlight is a privately-owned CDMO based in London, U.K., focused on the provision of DNA services and manufacture of enzymatically produced dbDNA™ (doggybone DNA) to enable the development of genetic medicines and the first synthetic DNA technology to receive a GMP certificate globally. Touchlight provides rapid, enzymatic DNA development and manufacturing for all advanced therapy production, including mRNA, viral and non-viral gene therapy and DNA API. dbDNA is a minimal, linear, covalently closed structure, which eliminates bacterial sequences. Touchlight’s revolutionary enzymatic production platform enables unprecedented speed, scale, and the ability to target genes with a size and complexity that is impossible with current technologies. Clients can be supported from pre-clinical through development and supply, to licensing and tech transfer for use in-house.

www.touchlight.com

Jason Slingsby, Ph.D., CEO

Oct. 8 | 10:45am | FLW Ballroom G

England, UK

(Private)

Tozaro is developing Smart Polymers, a novel class of affinity ligands, to improve methods for affinity purification of viral vectors. Our mission is to lower the manufacturing cost per dose of viral vectors by increasing % recovery and yields in downstream processing.
Using in silico design and ML from wet lab experiments, we can rapidly design and develop polymers with high affinity and specificity to optimally selected epitopes. By using >500 diverse chemical monomers as opposed to 20 amino acids, we can massively increase the design space for our affinity ligands. Our lead program is affinity purification of lentiviral vectors and our second is a novel AAV affinity purification across multiple capsid serotypes. We can also enable 2-minute titre measurements of viral vectors on leading BLI analytical platforms.
Tozaro has 30 employees in the UK and is backed by a syndicate of VCs.

www.tozaro.com

Daniel Castro, Chief Business Officer

Oct. 6 | 1:30pm | FLW Ballroom G

Pessac, France

(Private)

TreeFrog Therapeutics is a R&D biotech set to unlock access to cell therapies for millions of patients. The company’s unique approach to cell therapy development brings together biophysicists, cell biologists and bioproduction engineers to address the challenges of the industry – producing and differentiating cells of quality at unprecedented scale, cost-effectively. Their programs are based on their proprietary technology, C-Stem™ which encapsulates cells, allowing them to amplify and differentiate in a closed GMP-complaint system. Their lead program is developing a best-in-class iPS-derived cell therapy for Parkinson’s Disease. To realize their mission of Cell Therapy for all, TreeFrog operates a business model that includes its own programs and partnerships with leading biotech and industry players in other areas. In April 2024, TreeFrog signed a landmark licensing agreement and collaboration deal with Vertex Pharmaceuticals for the use of C -Stem™ to optimize production of Vertex’s cell therapies for Tupe 1 diabetes.

www.treefrog.fr

Richard Porter, Chief Business and Scientific Officer

Oct. 6 | 2:00pm | FLW Ballroom G

Amsterdam, Netherlands

(NASDAQ: QURE)

uniQure is delivering on the promise of gene therapy –– through an innovative adeno-associated virus (AAV)-based technology platform supported by proprietary commercial-grade manufacturing. The recent approvals of our gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and usher in a new treatment approach for patients living with hemophilia. We’re now advancing a clinical program in Huntington’s disease with a growing list of preclinical candidates.

www.uniqure.com 

Andy Holt, Chief Commercial Officer

Oct. 7 | 1:45pm | FLW Ballroom G

San Sebastian, Spain

(Private)

Viralgen is a leading CDMO specializing exclusively in AAV-based gene therapies, offering end-to-end support from early development to commercial production. Using our proprietary Pro10™ suspension platform, we achieve high-yield, scalable manufacturing across all AAV serotypes. With cGMP-certified facilities in San Sebastián, Spain, we provide integrated services including plasmid production, process optimization, fill-finish, and in-house QC testing. Our approach accelerates development timelines while controlling cost-of-goods. With over 1,500 AAV batches produced and a strong regulatory track record, Viralgen delivers reliable, efficient solutions that help bring gene therapies to patients with greater speed and consistency.

www.viralgenvc.com 

Becky Cap, Senior Vice President, Biotherapies

Oct. 8 | 9:45am | FLW Ballroom G

Scottsdale, AZ

(Private)

Vitalant is a community-based healthcare enabling organization in more than 1000 hospitals. Vitalant’s biotherapies program has expanded and is strategically positioned to address a critical gap in the rapidly evolving cell and gene therapy landscape, bridging the space between patient needs and the current capabilities of the healthcare environment. Our clinical services team is delivering first and last miles services connecting hospitals, therapeutic developers and patients to enable delivery of care and monitoring to the communities where patients live.
We provide clinically-focused services including apheresis collections – whether in the hospital with credentialed nurses or in our collection centers located around the country where patients live. Our intermediate processing supports manufacturing through cell product processing and characterization, and cryopreservation. Our pharmacy services allow us to prepare and deliver manufactured product to the bedside Our FDA-registered labs, facilities and operations are accredited by AABB, FACT, CAP, CLIA, and ASHI, as relevant.

www.vitalanthealth.org

Stijn Heessen, Ph.D., Chief Business Officer

Oct. 8 | 11:30am | FLW Ballroom G

Lund, Sweden

(Public: XINT)

Xintela is a clinical-stage biopharma company developing novel allogeneic mesenchymal stem cell (MSC) therapies based on its proprietary stem cell marker technology. Xintela’s best-in-class stem cell product XSTEM® is in Phase I/IIa clinical development in knee osteoarthritis (OA) (completed) and difficult-to-heal venous leg ulcers (VLU). The 18-month interim analysis demonstrated safety and significant sustained pain reduction and improvement of joint function as well as improvement of bone and cartilage structure in the OA study. The final study report includes up to 24-month follow-up data. The VLU study is also near completion. XSTEM® differentials:
– Consists of homogenous, integrin α10β1-selected MSCs with superior potency and consistency
– Clinical data demonstrate safety, efficacy and a disease-modifying mode-of-action
– Protected with granted product patents for all therapeutic uses
– Manufactured in Xintela’s in-house GMP facility

www.xintela.se