Innovation Spotlights

Jeff Wren, Vice President, Biotherapies

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

Bethesda, MD

(Private)

AABB is a global nonprofit association dedicated to advancing the quality, safety, and availability of blood and biotherapies. For decades, AABB has been a recognized authority for standards development, accreditation, education, and credentialing—building the quality expertise and implementation infrastructure that organizations across the therapy lifecycle depend on, from cellular starting material collection and processing to the handling and dispensing of advanced therapies in the healthcare setting. Working with researchers, developers, healthcare providers, regulators, and industry stakeholders, AABB’s programs establish the frameworks, qualified workforces, and operational excellence needed to support the development, delivery, and adoption of innovative therapies. With more than 6,000 members and over 1,300 accredited facilities in more than 50 countries, AABB serves as a trusted partner across the blood and biotherapies continuum—expanding patient access to life-changing therapies while driving continuous improvement across the field.

www.aabb.org

Stanley Bonn, Business Development Manager

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

San Jose, CA

(Private)

Founded in 2007, Accellix has been redefining cell phenotyping through rapid, automated flow cytometry solutions that simplify complex workflows and deliver critical quality insights directly to the point of need. Its automated ACCELLIX™ Platform delivers rapid flow cytometry–based analysis for cell therapy development, manufacturing, and quality control, enabling measurement of critical quality attributes throughout the manufacturing process and product release. The platform also supports characterization of blood-derived apheresis products used in research, donor screening, and the development of advanced therapies. In January 2026, bioMérieux, a world leader in the field of in vitro diagnostics since 1963, acquired Accellix, strengthening its Pharmaceutical Quality Control portfolio with rapid, automated flow cytometry solutions for cell and gene therapy. This strategic investment expands bioMérieux’s support for the growing advanced therapies market and reinforces its commitment to improving patient outcomes worldwide.

www.accellix.com

Luca Alberici, Executive Vice President, Global Cell & Gene Technologies and General Manager, Milan Facility; Vice President, CGT Global

Oct. 6 | 3:30pm | FLW Ballroom G

Bothell, WA

(Private)

AGC Biologics is a global CDMO headquartered in Seattle, Washington, USA. The company provides pharmaceutical development and manufacturing services for protein-based biologics, cell and gene therapies, and messenger RNA. With multiple sites and teams of scientists across three continents, we have the resources and the available capacity you need to accelerate your race to GMP. From development to clinical trials, to full-scale commercialization, we can help you reach your goals at any stage in the drug development and manufacturing process. We specialize in the following modalities and substances, mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), messenger RNA (mRNA), viral vectors, and genetically engineered cell therapies. Our services range from process development, analytical development, cGMP manufacturing, quality control and quality assurance, and process validation. We forge exceptionally strong partnerships with our customers, and we never lose sight of our commitment to delivering reliable and compliant drug substances.

www.agcbio.com

Devyn Smith, CEO

Oct. 5 | 10:45am | 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.

www.arbor.bio

Richard McFarland, Chief Regulatory Officer

Oct. 5 | 9:15am | FLW Ballroom F

Manchester, NH

(Private)

ARMI | BioFabUSA is a national public-private partnership advancing the manufacturing capabilities required to realize the promise of regenerative medicine and advanced therapies. Through a network of nearly 200 organizations spanning industry, academia, government, healthcare, and investment communities, ARMI works to address the manufacturing, workforce, regulatory, and commercialization challenges that limit scale and adoption. As one of the nation’s Manufacturing Innovation Institutes, ARMI develops manufacturing technologies, workforce solutions, and collaborative infrastructure that help translate scientific breakthroughs into scalable, commercially viable products. By bringing together stakeholders across the regenerative medicine value chain, ARMI is helping strengthen domestic biomanufacturing capabilities, accelerate commercialization, and establish the foundation needed to support the next generation of cell, tissue, and regenerative medicine products.

www.armiusa.org

Nicole van der Weerden, CEO

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

Preston, AUS

(ASX: ALA)

Arovella is a clinical‑stage biotechnology company developing a next‑generation CAR‑iNKT cell therapy platform for the treatment of haematological malignancies and solid tumours. Arovella’s lead programme, ALA‑101, is an allogeneic CD19-targeting CAR‑iNKT therapy with an accepted US FDA IND. Arovella is building a highly differentiated platform through the integration of a novel cell type (invariant Natural Killer T cells), unique manufacturing process, novel CAR targets (including claudin 18.2), and IL‑12‑TM cytokine armouring, designed to enhance anti‑tumour activity while limiting systemic toxicity. This combination of allogeneic iNKT biology, natural tumour targeting of iNKT cells via NKG2D ligands and CD1d, and cytokine armouring, is intended to overcome key limitations of first‑generation CAR therapies, particularly in solid tumours. Arovella is among a small number of companies globally advancing CAR‑iNKT therapies toward the clinic, and differentiated by its modular, multi‑asset platform approach.

www.arovella.com

John Kerwin, Senior Vice President, Operations

Oct. 5 | 4:30pm | FLW Ballroom G

Boston, MA

(Private)

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, renal, 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

Tamer Mohamed, CEO and Founder

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

Vancouver, B.C., Canada

(Private)

Aspect Biosystems is a biotechnology company pioneering the development of a novel category of cell-based regenerative medicines designed to restore or supplement biological function and deliver curative therapies for some of the most elusive diseases. Aspect is developing these bioengineered cellular medicines by applying its full-stack tissue therapeutic platform, which integrates proprietary AI-powered bioprinting technology, therapeutic cells, hypoimmune cell engineering, and advanced biomaterials. Aspect is advancing its therapeutic pipeline across multiple endocrine and metabolic diseases, including diabetes and rare endocrine disorders.

www.aspectbiosystems.com

Kim Raineri, Chief Technology Officer

Oct. 5 | 11:00am | FLW Ballroom F

San Diego, CA

(Private)

Aspen Neuroscience is a leading, clinical-stage regenerative medicine biotechnology company developing autologous induced pluripotent stem cell-derived (iPSC) therapies beginning with neurodegenerative diseases of high unmet need including Parkinson’s disease (PD). Its lead product candidate is sasineprocel (ANPD-001), the most advanced autologous investigational cell therapy in the United States for treating PD.

www.aspenneuroscience.com

Richard Wilson,Head, Primary Focus Lead, Genetic Regulation

Oct. 6 | 3:30pm | FLW Ballroom F

Tokyo, Japan

(NASDAQ: ALPMY)

Astellas is a global life sciences company committed to turning innovative science into VALUE for patients. We provide transformative therapies in disease areas that include oncology, ophthalmology, urology, immunology and women’s health. Through our research and development programs, we are pioneering new healthcare solutions for diseases with high unmet medical need.

www.astellas.com

Lisa Deschamps, CEO

Oct. 5 | 3:00pm | FLW Ballroom F

London, UK

(Private)

AviadoBio is relentlessly chasing cures by developing and translating groundbreaking science and precision delivery into potentially life-changing medicines across neurodegenerative diseases. Powered by a precision approach built on three integrated levels — payload, target, and delivery — the company is advancing a pipeline of targeted, one-time AAV gene therapies across frontotemporal dementia with GRN mutations (FTD-GRN), retinitis pigmentosa (RP) and other retinal degenerative diseases,

www.aviadobio.com

Rob Lin, CEO

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

Pittsburgh, PA

(Private)

Avista Therapeutics is a preclinical gene therapy company whose mission is to break barriers in delivery of genetic medicines through AI-driven innovation. Through our ARTEMIS platform, Avista has engineered best-in-class vectors to treat retinal diseases through intravitreal injection.

www.avistatx.com

Pratima Cherukuri, Ph.D., General Manager and Vice President of pDNA

Oct. 5 | 2:15pm | FLW Ballroom F

Fremont, CA

(Private)

Bionova Scientific®, a commercial-scale biologics CDMO and proud member of the Asahi Kasei Group, partners with innovators to bring life-changing therapies to market with a strong commitment to quality and service. Founded in 2014 and acquired by Asahi Kasei in 2022, Bionova provides advanced technology platforms and scientific expertise across the full drug development lifecycle.
As an integrated CDMO, Bionova offers end-to-end services from discovery through cGMP commercial supply of complex therapeutic proteins and antibodies. Backed by a team of seasoned scientists with decades of experience, we help clients overcome early development challenges and accelerate programs from discovery to first-in-human studies with scalable, efficient solutions.
In 2025, Bionova expanded with a dedicated plasmid DNA (pDNA) development and manufacturing facility in Houston, Texas. With state-of-the-art facilities in Houston and the San Francisco Bay Area, Bionova remains a flexible, collaborative, and quality-focused partner for advanced therapy production.

www.bionovascientific.com

Hans Schambye, CEO

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

Copenhagen N, Denmark

(Private)

BOOST Pharma AB is a clinical-stage biotechnology company developing novel, first-in-class off-the-shelf stem cell therapies for rare, debilitating pediatric skeletal diseases. The Company’s lead program, BT-101, is an allogeneic mesenchymal stem cell therapy for osteogenesis imperfecta, a severe rare bone disorder, and has received Orphan Drug Designation in both the U.S. and Europe. BT-101 is currently advancing toward a planned Phase III clinical trial. BOOST is backed by a strong syndicate of life-science investors, including Industrifonden, KDventures, and Sound Bioventures, who support the Company’s strategy focused on high-impact science, capital-efficient development, and clear pathways to market. The Company is led by a team with deep experience in biotech innovation, clinical execution, and value creation.

www.boostpharma.com

Angela Nelson, Chief Strategy Officer and Partner

Oct. 5 | 11:15am | FLW Ballroom F

Boston, MA

(Private)

Founded in 2009, Boston Biodevelopment is a premium regulatory strategy consulting firm specializing in the development and approval of innovative therapies. Our team of highly experienced regulatory professionals, including former Heads of Regulatory Affairs, provides hands-on strategic leadership and execution across all stages of the product lifecycle. Our signature services include Interim Head of Regulatory Affairs Leadership and Embedded Regulatory Teams, providing clients with experienced regulatory leaders and functional experts who integrate seamlessly within their organizations. Whether augmenting existing capabilities or serving as a fully embedded regulatory function, our consultants deliver strategic continuity, operational excellence, and immediate impact. We bring deep expertise across biologics, small molecules, cell and gene therapies, gene editing, and other advanced modalities, with extensive experience in rare diseases and a broad range of therapeutic areas. Clients rely on Boston Biodevelopment to accelerate development programs, navigate complex regulatory challenges, and bring innovative therapies to patients with confidence.

www.bostonbiodevelopment.com

Philip Wills, Chief Commercial Officer

Oct. 5 | 10:45am | FLW Ballroom G

Tampa, FL

(Private)

At Catalent, we champion the missions that matter to help people live better and healthier lives. Every medicine, therapy and product that we help develop, manufacture and deliver is part of our commitment to put patients first in everything we do. We are a trusted partner because we are committed to an exceptional, high-quality experience. Our customers turn to us for tailored solutions to meet their needs as we work together to transform lives.

biologics.catalent.com

Matthew Durdy, Chief Executive

Oct. 6 | 10:45am | FLW Ballroom G

Central London, UK

(Private)

Cell and Gene Therapy Catapult (CGT Catapult) is an independent innovation and technology organization committed to the advancement of the cell and gene therapy industry. CGT Catapult’s vision for the UK Industry is: globally-leading companies delivering transformative medicine to the world. Its mission is to create a pathway to accelerate breakthrough research in advanced therapies to commercialization and scale. CGT Catapult works with Innovate UK.

www.ct.catapult.org.uk

Bernd Mühlenweg, Chief Business Officer and Chief Financial Officer

Oct. 5 | 10:00am | FLW Ballroom G

Rockville, MA

(Private)

Cellbox Solutions is an infrastructure biotechnology company enabling the safe transportation of living biological materials across the advanced therapy value chain. The company provides proprietary warm-chain technology and managed logistics solutions that preserve the viability, functionality and quality of living cells, organoids, tissues and other advanced biological products during transportation. Cellbox supports the transition of cell and gene therapies from GMP manufacturing to clinical trial sites and, ultimately, routine clinical application by enabling reliable, controlled transport without relying exclusively on cryopreservation. Its platform combines proprietary transport technology, digital monitoring and logistics services to address one of the emerging bottlenecks in the commercialization of advanced therapies: the movement of living biological products. Cellbox works with biotechnology companies, pharmaceutical manufacturers, CDMOs and healthcare providers to build the infrastructure required for scalable, decentralized and patient-centric delivery of next-generation cell therapies.

www.cellbox-solutions.com

Patrick Hanley, Chief and Director, Cellular Therapy Program

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

Washington, D.C.

(Private)

For 155 years, Children’s National has delivered expert pediatric care at every milestone. As the Washington, D.C., region’s only standalone children’s hospital, we provide comprehensive care for more than 250,000 kids each year. We are ranked one of the top 10 pediatric hospitals in the nation by U.S. News & World Report. We’re also tied for #1 children’s hospital in the Mid-Atlantic region. This recognition of our commitment to bringing health and well-being to all children continues to inspire our teams.

www.childrensnational.org

Victor Levitsky, Chief Scientific Officer

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

Huddinge (Stokholm), Sweden

(OSE: CRNA)

Circio is a public biotechnology company which has established a unique and proprietary intracellular circular RNA (circRNA) expression system, circVec, that substantially enhances the potency and durability of gene expression which is critical for gene therapies. circVec is designed for efficient biogenesis of circRNA from DNA and viral vectors, with primary application in AAV gene therapy and in vivo T-cell therapy. Following AAV delivery, circVec has demonstrated up to 50-fold enhanced gene expression compared to conventional mRNA-based AAV gene therapy in several organs and tissues such as heart and eye and six months expression durability for LNP-delivered payload in spleen. Circio is the global leader in vector-based circRNA expression systems with no known competitors with comparable versatility and performance. The circVec R&D activities are being conducted in the wholly owned subsidiary Circio AB located in the Novum Labs building on the Karolinska Institute Campus in Huddinge (Stockholm), Sweden.

www.circio.com

Rafiq Hasan, CEO

Oct. 5 | 4:15pm | FLW Ballroom G

Munich, Germany

(Private)

Complement Therapeutics (CTx) is a clinical-stage biotechnology company dedicated to developing novel therapeutics for complement-mediated diseases. The company’s lead investigational program is CTx001, a novel AAV2-based gene therapy targeting Geographic Atrophy (GA) secondary to dry age-related macular degeneration—a progressive disease causing irreversible blindness in over 5 million people globally. Designed as a single subretinal injection, CTx001 delivers mini-CR1, a truncated and secreted form of Complement Receptor 1, to enable sustained, targeted modulation of complement pathways. CTx is actively advancing CTx001 through the clinical pipeline, supported by the non-interventional Pre-GAIN natural history study and the Phase I/II Opti-GAIN clinical trial. Opti-GAIN is a multi-centre clinical study evaluating the safety, tolerability and pre-liminary efficacy of CTx001 administered via a single subretinal injection in 75 participants with Geographic Atrophy secondary to Age-related Macular Degeneration. Beyond GA, CTx is expanding its pipeline to target other complement-mediated conditions.

www.complementtx.com

Grant Boldt, Chief Operating Officer

Oct. 5 | 3:15pm | FLW Ballroom G

Munich, Germany

(Private)

CPTx is developing a non-viral, duration-tunable platform for in vivo CAR-T therapies. The approach combines immune-quiet DNA vectors encoding CAR genes with targeted lipid nanoparticles for delivery to T cells. CPTx’s DNA vectors can generate both time-limited, transient CAR-T responses and long-lasting, durable expression, while also supporting multi-gene payloads. This flexibility enables the development of therapies across oncology, autoimmune diseases, and other indications. Durable expression is particularly important for cancer, while a transient burst of CAR-T activity can support immune-reset applications in autoimmune diseases. CPTx has purpose-built every layer of the platform, including proprietary immune-quiet and duration-tunable DNA vectors, custom LNP formulations, and targeting strategies that direct the payload to T cells. The company has demonstrated B-cell depletion and tumor killing both in vitro and in vivo.

www.cptx.bio

Daniel Getts, CEO

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

Cambridge, MA

(Private)

CREATE Medicines is a clinical-stage biotechnology company pioneering in vivo immune programming. The company’s proprietary mRNA-LNP platform directly engineers T cells, NK cells, and myeloid cells inside the body to enable scalable, repeat-dose, off-the-shelf immunotherapies. CREATE is advancing a pipeline of in vivo CAR therapies across autoimmune diseases and cancer.

createmedicines.com

Tim Schroeder, Executive Chairman and Founder

Oct. 5 | 3:00pm | FLW Ballroom G

Covington, KY

(Private)

CTI Clinical Trial & Consulting Services is a global, privately held clinical research organization delivering end-to-end clinical trial and consulting services from concept through commercialization. With deep expertise in cell and gene therapy (CGT) and rare diseases, CTI partners with biopharmaceutical companies to navigate complex development and regulatory challenges. Our integrated model spans regulatory strategy, clinical execution, sites, and global laboratory services, enabling streamlined operations, accelerated timelines, and reduced risk. Operating in more than 60 countries, CTI brings specialized scientific and regulatory expertise to support advanced therapies worldwide.

www.ctifacts.com

Michelle Hussong, Director, Cell Line Development R&D

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

Marlborough, MA

(Private)

At Cytiva, we envision a world in which access to life-changing therapies transforms human health. With a rich heritage dating back hundreds of years, our wealth of technical expertise and talent, a broad and deep portfolio, and exceptional service help researchers and biopharma advance therapeutics at every stage from discovery to delivery. Cytiva is proud to be part of Danaher. Danaher is a global life sciences and diagnostics innovator committed to accelerating the power of science and technology to improve human health. Together, we partner with customers to solve their toughest challenges—with speed and certainty.

www.cytivalifesciences.com

Chris Murphy, CEO

Oct. 5 | 11:45am | FLW Ballroom G

Waltham, MA

(Private)

ElevateBio is a technology-driven advanced therapy contract development and manufacturing organization (CDMO) powering the creation of life-transforming therapies. The company helps biopharmaceutical partners design, develop, and manufacture therapies from early discovery through commercialization, combining proprietary gene editing technologies and discovery services, cGMP manufacturing capabilities, and industry-leading expertise to accelerate development across a breadth of therapeutic approaches and modalities. Through continuous investment in automation, AI, and next-generation technologies, ElevateBio delivers the quality, speed, and scale partners need to bring advanced therapies to more patients. For more information, visit www.elevate.bio.

www.elevate.bio

Ramin Valian, CEO

Oct. 5 | 9:45am | FLW Ballroom G

Menlo Park, CA

(Private)

Emmecell is a clinical-stage biotechnology company developing magnetic cell delivery technologies to treat corneal endothelial disease, geographic atrophy, and other ophthalmic conditions. Its platform enables controlled, targeted positioning of therapeutic cells to support minimally invasive, regenerative alternatives to traditional surgical and pharmacologic therapies. Emmecell is headquartered in Menlo Park, California.

www.emmecell.com

Sanjeev Luther, President and CEO

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

Cambridge, MA

(NASDAQ: ERNA)

Precisely Engineered Cells. Powerful Therapeutics. At Ernexa Therapeutics, we believe the body holds powerful tools for healing. Our job is to help reactivate them. Through innovative cell therapies, we are addressing the immune dysregulation that underlies cancer and autoimmune disease to help restore what illness seeks to erode: strength, resilience, and hope. Our core technology focuses on engineering induced pluripotent stem cells (iPSCs) and transforming them into induced mesenchymal stem cells (iMSCs). Ernexa’s allogeneic synthetic iMSCs provide a scalable, off-the-shelf treatment solution 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-201 is a cell 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.

www.ernexatx.com

Victor Aguilar Lopez, Head of Business Development Cell Therapy

Oct. 5 | 12:00pm | FLW Ballroom G

Northern Germany

(XETRA: EVT)

Evotec is a life sciences company focused on delivering highly effective new therapeutics to patients. The company leverages its multimodality platforms for proprietary projects and pipeline co-creation with partners including pharma, biotech, academics, and other healthcare stakeholders. Our business model is flexible, ranging from service-based support to strategic partnerships. Within cell therapy, Evotec offers a fully integrated end-to-end platform to move iPSC-based projects from inception to the clinic, including in-house GMP manufacturing and GMP gene editing capabilities. Our platform is one of the broadest in the industry. The company focusses on therapeutic indications requiring long term engraftment of cells like regenerative therapies (beta cells, cardiomyocytes, photoreceptors) and immune-modulation therapies (macrophages, T cells). In addition, Evotec provides integrated drug development solutions, covering safety assessment, CMC, process and analytical development, regulatory and clinical services for autologous and allogeneic cell therap

www.evotec.com

Rebecca Godfrey, Chief Operating Officer

Oct. 5 | 10:00am | FLW Ballroom F

Cambridge, UK

(Private)

ExpressionEdits is a University of Cambridge spin-out advancing biotechnology by enabling it to “speak” nature’s genetic language. Founded by Dr Kärt Tomberg and Professor Allan Bradley, the company has developed the world’s first AI-driven Genetic Syntax Engine, trained on billions of data points from years of proprietary experiments. Today’s synthetic transgenes lack the intron–exon architecture characteristic of natural genes, a fundamental mismatch that limits RNA processing, export, and stability. ExpressionEdits’ intronized designs recreate this natural structure, yielding superior RNA quality and higher protein expression across biologics and gene therapies. The company has built the largest and highest-quality dataset in the field, validated its platform through licensing deals, including a multi-asset partnership with Boehringer Ingelheim in gene therapy, and is advancing an internal pipeline, with a lead program in hematology.

www.expressionedits.com

John Maslowski, President and CEO

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

Grove City, 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

Michelle Chen, CEO

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

Austin, TX

(Private)

Form Bio is a leader in developing AI-powered solutions for genetic medicine and genome engineering. Since spinning out from Colossal Biosciences in 2022, the company has become a trusted innovation partner to cell and gene therapy developers and beyond. Form Bio’s advanced in silico technology and computational platforms deliver deep insights into genome integrity, optimized payload design, and other critical factors-enabling safer, more efficacious, and more manufacturable therapeutics. By reducing risk, shortening timelines, and optimizing outcomes, Form Bio helps drug developers accelerate the path from discovery to the clinic.

www.formbio.com

Allyson Berent, Chief Scientific Officer, Foundation for Angelman Syndrome Therapeutics; Chief Development Officer. Co-founder, AS2Bio

Oct. 5 | 9:45am | FLW Ballroom F

Austin, TX

(Private)

FAST, a non-profit patient advocacy organization launched an accelerator called AS2Bio, a venture philanthropy model to advance platform agnostic and disease specific therapies forward for the rare monogenic disorder called Angelman syndrome, serving as a platform for other rare monogenic neurodevelopmental disorders.

www.as2bio.com

Angela Columbano, Head Business Development and Partnership

Oct. 5 | 9:30am | FLW Ballroom F

Évry-Courcouronnes, 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. Genethon has contributed to the development of the first gene therapy to receive market authorization for spinal muscular atrophy. With more than 200 scientists and professionals, Genethon continues to pursue its mission of delivering life-changing therapies to patients suffering from rare genetic diseases. Currently, Fifteen products resulting from Genethon’s research are in clinical trials for liver, blood, immune system, muscle and eye diseases. In addition, seven other products are in the preparation phase for clinical trials over the next five year.

www.genethon.com

Brian Riley, President and Chief Technology Officer

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

Somerville, MA

(Private)

Genetix Biotherapeutics Inc. is a privately held, commercial-stage biotechnology company dedicated to delivering one-time transformative treatments for patients with severe rare diseases. Backed by more than 30 years of pioneering genetic medicine innovation, the company has FDA-approved treatments for sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy that directly treat the underlying cause of disease. Genetix is committed to commercial execution, scaling patient access, and improving the treatment experience for patients and providers.

www.genetixbiotx.com

Ashwin Nandakumar, CEO and Co-Founder

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

San Francisco, CA

(Private)

Granza Bio is developing a new category of programmable immune medicines to overcome the major limitations of current cell therapies in solid tumours. Its proprietary SMAP-T platform is built on the discovery of immune “superkiller” attack particles (Science, 2020) — potent cytotoxic structures produced by CD8 T cells that act as the immune system’s natural ammunition for persistent tumour-cell killing. By reverse-engineering this biology, Granza is developing therapies that reprogramme T cells in vivo to generate enhanced cytotoxic function against difficult-to-treat solid tumours. This approach enables a new class of immune medicines with the potential to overcome key limitations of current cell therapies and transform the treatment of solid tumours.

www.granzabio.com

Laura Herbs Herbst, Scientific Expert Advanced Therapies

Oct. 5 | 4:45pm | FLW Ballroom G

Allmersbach im Tal, Germany.

(Private)

Harro Höfliger is a leading global technology provider for production and packaging systems in the pharmaceutical, medical technology, consumer goods, and healthcare industries. Within the field of Advanced Therapies, the company specializes in innovative manufacturing solutions for Cell & Gene Therapies (CGT), regenerative medicine, and other Advanced Therapy Medicinal Products (ATMPs). Combining decades of expertise in aseptic processing, automation, and machine engineering, Harro Höfliger develops scalable and GMP-compliant solutions that support the transition from clinical development to commercial manufacturing. The company’s modular platforms enable closed and automated processes, helping therapy developers improve product quality, reduce contamination risks, and increase manufacturing efficiency. A key innovation is the Happy Cell Factory, a flexible automation platform designed to streamline cell therapy production through standardized and interconnected process modules. By integrating advanced process control, digitalization, and single-use technologies, Harro supports customers in overcoming the manufacturing challenges associated with personalized and allogeneic therapies.

www.hoefliger.com/en/products/pharmaceutical/cell-and-gene-therapies

Greg Merril, CEO

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

Rockville, MA

(Private)

Perpetuu Bio (previously HBVtech) is developing transformative gene therapies for serious viral diseases using a proprietary in vivo AAV platform designed to provide durable therapeutic benefit following a single administration. The lead program is a potentially curative therapy for chronic hepatitis B virus (HBV) infection that directly eliminates viral cccDNA and rcDNA, addressing the fundamental source of viral persistence. A second lead program is a long-acting prophylactic therapy for cytomegalovirus (CMV) in hematopoietic stem cell transplant recipients through sustained expression of a potent CMV-neutralizing antibody, with the potential to replace months of antiviral therapy. These programs demonstrate the versatility of Perpetuu’s platform to both eradicate persistent viral infections and provide durable protection against life-threatening viral disease. Led by an experienced team with expertise in gene therapy, antiviral development, FDA regulatory strategy, and biotechnology commercialization, Perpetuu is advancing a focused pipeline addressing large markets with significant unmet medical need.

www.perpetuu.bio

Gregory Block, CEO

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

Seattle, WA

(Private)

Histone Therapeutics is a Seattle-based biotechnology company developing its proprietary epigenetic ON (eON) technology. While traditional genetic medicines focus on tuning or silencing overactive genes, eON activators can potently and precisely reach new targets for diseases with high unmet need.

www.histonetx.com

Jennifer Chow, CEO

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

Stony Brook, NY

(Private)

iCell Gene Therapeutics is a private, clinical-stage biopharmaceutical company with a robust pipeline of autologous and in vivo CAR T cell therapies for autoimmune diseases and cancer. In addition to our lead asset, ICG318, iCell Gene has a robust pipeline of in vivo and autologous CAR T cell therapies for autoimmune diseases, solid tumors and hematological malignancies.

www.icellgene.com

Sean Ainsworth, CEO

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

Seattle, WA

(Private)

Immusoft is to develop novel therapies, by programming B cells to produce therapeutic proteins that deliver functional improvement, to people living with rare and other diseases with significant unmet need.

www.immusoft.com

Joe DePinto, Head of Cell, Gene and Advanced Therapies

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

Irving, TX

(Private)

InspiroGene™ by McKesson is a dedicated business focused on advancing the commercialization of cell and gene therapies (CGT). It delivers an integrated, end-to-end solution that connects distribution, specialty pharmacy, patient support, and technology to simplify the complexity of bringing breakthrough therapies to market. Designed to bridge the gap between innovation and real-world access, InspiroGene helps biopharmaceutical manufacturers, providers, and patients navigate the CGT value chain with greater speed, confidence, and efficiency. Its scalable services—including logistics, cold chain storage, and patient hub support—optimize operations while improving the treatment experience. Backed by McKesson’s expertise, InspiroGene enables broader, faster, and more reliable patient access to life-changing therapies

inspirogene.com

Daniel Hargrove, CEO

Oct. 5 | 2:00pm | FLW Ballroom G

Dallas, TX

(Private)

InterAct Therapeutics, Inc. is a preclinical gene therapy company pioneering novel treatments for metastatic cancer, beginning with breast cancer liver metastasis (BCLM), a disease affecting an estimated 30,000 to 60,000 patients in the US with a five-year survival rate of less than 10% and no approved targeted therapies. Our lead program is an AAV8-mediated, liver-directed gene therapy that restores hepatocyte fitness to counteract the cell competition mechanism by which metastatic breast cancer cells colonize the liver. The science originates from foundational research at UT Southwestern Medical Center. Powering our broader pipeline is the InterAct Print™ platform, a modality-agnostic computational discovery engine that identifies genes enabling metastatic cells to outcompete healthy host tissue. The platform supports therapeutic development across AAV gene therapy, siRNA, and LNP-mRNA modalities, positioning InterAct to expand across multiple metastatic cancer indications.

www.interacttx.com

Blake Arnold, Chief Business Officer

Oct. 5 | 2:45pm | FLW Ballroom F

Cambridge, MA

(Private)

Kenai Therapeutics is a clinical-stage biotechnology company pioneering next generation approaches to cure neurological conditions. By leveraging a proprietary, Nobel Prize-winning iPSC platform, Kenai is developing off-the-shelf, allogeneic neuron replacement and gene-modified cell therapies designed to be disease-modifying. Kenai’s lead candidate, RNDP-001, is in Phase 1 clinical development for moderate to moderate-severe forms of idiopathic Parkinson’s disease. The company’s additional programs target inherited and genetically driven subtypes of Parkinson’s disease and other neurological conditions, with the goal of delivering long-lasting restoration of function. Kenai’s exclusive manufacturing partnership with FUJIFILM Cellular Dynamics, Inc. enables scalable, cryopreserved production of high-potency cell therapies. Founded in 2022, Kenai is backed by leading life science investors and is headquartered in San Diego, CA.

www.kenaitx.com

Warner Biddle, CEO

Oct. 5 | 2:30pm | FLW Ballroom F

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, CEO

Oct. 5 | 9:30am | FLW Ballroom G

Philadelphia, PA

(Private)

We develop gene therapies designed to target the right cell types in the right tissues, to fundamentally impact genetic diseases like CLN2 and Huntington’s Disease

www.latusbio.com

Michael Hufford, CEO

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

Pittsburgh, PA

(Private)

LyGenesis is a clinical-stage biotechnology company developing a novel cell therapy platform leveraging patients’ lymph nodes as bioreactors to grow functioning ectopic organs. LyGenesis’ lead allogeneic cell therapy program is currently in a Phase 2a clinical trial for patients with end-stage liver disease (ClinicalTrials.gov Identifier: NCT04496479). LyGenesis’ cell therapy platform including therapies in development designed to produce an ectopic thymus (for autoimmune diseases, oncology, aging and multiple other potential indications), pancreas (for Type 1 diabetes), and kidney (for renal disease). Privately held, LyGenesis is headquartered in Pittsburgh, Pennsylvania. To learn more, please visit our website at www.LyGenesis.com.

www.lygenesis.com

Sarah Hein, CEO, Cofounder

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

Houston, TX

(Private)

March Biosciences, launched from the Center for Cell and Gene Therapy (Baylor College of Medicine, Houston Methodist Hospital, Texas Children’s Hospital), 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. March has a pipeline of assets including novel approaches to solid tumor targeting using a novel synapse stabilizing receptor. The company has raised over $55 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

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

Rochester, MN

(Private)

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, Global President

Oct. 5 | 4:00pm | FLW Ballroom G

Rockville, MD

(078160 KOSDAQ)

MEDIPOST found in 2000, is a fully integrated regenerative medicine biopharmaceutical company with integrated capabilities across cord blood banking, stem cell research, GMP manufacturing, clinical development and commercialization, with a strong focus on allogeneic umbilical cord blood-derived mesenchymal stem cell (hUCB-MSC) therapeutics. MEDIPOST has successfully commercialized CARTISTEM®, the world’s first regulatory-approved allogeneic umbilical cord blood-derived MSC therapeutic for knee osteoarthritis. Since its approval in 2012, more than 37,000 patients have received CARTISTEM® treatment in South Korea, generating extensive real-world clinical data. Recently, positive topline data from the pivotal Phase III study in Japan was announced in May 2026, while the Phase III study in the U.S. & Canada has recently started enrolling patients in February 2026. MEDIPOST is also developing several late-stage hUCB-MSC pipelines including SMUP-IA (an intra-articular injectable early-stage OA treatment) and PNEUMOSTEM® (prophylactic treatment for Broncho-Pulmonary Dysplasia – rare indication for prematurely-born infants).

www.medi-post.com

Jian Irish, CEO and President

Oct. 5 | 11:45am | FLW Ballroom F

Emeryville, CA

(NASDAQ: MGX)

Metagenomi is a biotechnology company developing next-generation genome editing technologies to enable curative genetic medicines. Its proprietary metagenomics-based discovery platform identifies and engineers novel gene editing systems designed to expand the range of treatable diseases and improve precision, delivery, and performance. The company is advancing a pipeline of therapeutic programs while also collaborating with industry partners to develop innovative genome editing solutions for patients with serious diseases.

www.metagenomi.co

Sarah Johnson, Chief Business Officer

Oct. 5 | 11:15am | FLW Ballroom G

Kawasaki, Japan

(Private)

Metcela Inc. is a late clinical-stage biotechnology company advancing JRM-001, an autologous cardiac stem cell therapy currently in Phase 3 clinical trials in Japan for patients with single ventricle disorders and reduced ejection fraction. Clinical studies to date have demonstrated favorable safety, improvements in cardiac function, and reductions in adverse events. With commercial launch in Japan anticipated by 2030, Metcela is actively seeking strategic partners to support global expansion, including entry into the U.S. market.

www.metcela.com

Daniel Teasley, Senior Director, CDMO Global Business Lead

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

Burlington, MA

(Private)

MilliporeSigma is the U.S. and Canada Life Science business of Merck KGaA, Darmstadt, Germany. Our Millipore CTDMO® services provide a streamlined experience in a single partner to mitigate the burden of managing multiple vendors, supply chain needs, and complex priorities along the drug life cycle. Our integrated Millipore CTDMO® Services combines our expertise in development, manufacturing, contract testing and technologies from pre-clinical to commercial to accelerate your milestones and breakthroughs – with one experienced partner.

www.sigmaaldrich.com

Madhusudan Peshwa, CEO

Oct. 5 | 2:15pm | FLW Ballroom G

Jacksonville, FL

(Private)

Our objective is to develop/commercialize therapeutics ATMPs for chronic conditions, that halt or reverse disease progression. Our business model is to search for technologies / assets with substantial human clinical data from ex-US geographies; and in-license IP/technology; for clinical/commercial development in US & Europe. Our first in-licensed platform is allogeneic pooled donor Mesenchymal Stromal Cells, which has resulted in commercially approved products for three indications in India. In the US, we have identified & selected donors, manufactured (at US CDMO) and released GMP Master & Working Cell Banks. Drug Product GMP WCB was demonstrated to have CQAs comparable to that of the India commercial product. All of the data was submitted / reviewed with US FDA in a pre-IND meeting, and following alignment are on path to file an IND to initiate US Phase 2 clinical study designed as adequately sized, RCT with active control arm.

mkcbiotherapeutics.com

Massimiliano Paganelli, Founder and CEO

Oct. 5 | 9:15am | FLW Ballroom G

Laval, Quebec, Canada

(Private)

Morphocell Technologies is a regenerative medicine company developing stem cell-derived engineered tissues to replace failing organs. Initially focused on severe liver disease, the company has built a fully integrated platform based on allogeneic, immune-shielded iPSC-derived tissues designed to deliver scalable, off-the-shelf therapies. Headquartered in the Greater Montreal Area, with operations in Boston/Cambridge, Southern California and Rome, Morphocell is advancing its lead product, ReLiver®, an engineered liver tissue for acute and acute-on-chronic liver failure that aims to restore liver function without immunosuppression. Following a US$50 million Series A financing, the company is progressing ReLiver® through IND/CTA-enabling studies toward first-in-human clinical trials. Its proprietary manufacturing and cell delivery platform supports a broad pipeline of future organ replacement therapies designed to address major unmet medical needs.

www.morphocell.com

Samarendra Mohanty, President

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

Dallas, TX

(Private)

Nanoscope Therapeutics has achieved a historic technological breakthrough in the field of gene therapy by developing Multi-Characteristic Opsin (MCO), an innovative optogenetic gene therapy platform to treat Retinal Disease-related blindness. Retinal Dystrophies (RDs) are the leading cause of blindness in working-age people, and there is presently no effective treatment for these diseases. Nanoscope developed MCO therapy as the first-ever treatment to restore return-to-life vision in patients that are profoundly blind due to RDs. MCO has been utilized in human patients across three clinical trials. Hundreds of gene mutations cause RDs, meaning a classical gene therapy approach would require developing hundreds of therapies to address RDs. Because RD progression is permanent, classical gene therapies can only benefit patients early in disease, leaving little hope for patients that have already lost their vision. MCO therapy has the potential to treat all RD patients, regardless of their mutation, or state of disease.

www.nanostherapeutics.com

Magdalena Tyrpien, Co-Founder and CEO

Oct. 5 | 2:45pm | FLW Ballroom G

New York City, NY

(Private)

Nionyx Bio is engineering the future of gene therapy, starting with the kidney. Our next-generation AAV capsid platform was designed first and foremost with a human-selected approach, screened directly in whole human kidneys across the cell types that drive kidney disease, podocytes, proximal tubule cells, and endothelial cells. This is a first-of-its-kind approach to an organ AAV has historically struggled to reach. On top of this platform, we’re building a pipeline of next-generation therapies for rare kidney diseases that have no disease-modifying treatments today. Nionyx pairs precision-medicine capsid engineering with a focused rare-disease pipeline. This is gene therapy designed for humans, built for the kidney.

www.nionyxbio.com

Jennifer Johnston, CEO

Oct. 5 | 9:00am | FLW Ballroom F

Mill Valley, CA

(Private)

NysnoBio is advancing genetic medicines for Parkinson’s Disease and other serious disorders of the CNS

www.nysnobio.com

Mike Hirschmann, Chief Commercial Officer

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

Menlo Park, CA

(Private)

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of blood cancer and autoimmune diseases. The company’s manufacturing platform uses single-cell precision to create proprietary, uniquely defined products designed to replace a patient’s diseased blood and immune system with a healthy one. At Orca Bio, we are on a mission to redefine what’s possible for patients by transforming the field of curative allogeneic cell therapy.

www.orcabio.com

Kyung Jin Lee, Chief Technology Officer

Oct. 6 | 2:15pm | FLW Ballroom G

Austin, TX

(Private)

Organoids by Southwest (OXSW) is a U.S.-based regenerative medicine company advancing the commercialization of the Adult Tissue derived Organoid Regenerative Medicine (ATORM) platform, a first-in-class intestinal organoid therapy originally developed by Organoid Sciences. Unlike conventional treatments that suppress inflammation, ATORM is designed to regenerate damaged intestinal tissue and restore epithelial barrier function through transplantation of adult stem cell-derived intestinal organoids. The platform has achieved several industry-leading milestones, including first-in-human clinical experience, 80+ clinical-scale GMP manufacturing runs, and one of the world’s most advanced regulatory pathways for organoid-based therapeutics. The lead program targets refractory intestinal ulcers associated with severe gastrointestinal disorders where current treatment options remain limited. Through OXSW, the technology is positioned for U.S. clinical development and commercialization while expanding into additional regenerative medicine opportunities involving epithelial repair and tissue restoration. OXSW seeks strategic partners, investors, and collaborators to accelerate global adoption of organoid-based regenerative therapies.

www.organoidsw.com

Christopher Carlson, Chief Scientific Officer

Oct. 5 | 1:45pm | FLW Ballroom G

San Francisco, CA

(Private)

ParcelBio is engineering a new class of mRNA medicines designed to deliver unprecedented potency and durability. Its proprietary APEXm™ platform uses novel RNA domains to enhance protein expression and extend activity, enabling therapies that reach the thresholds required for meaningful, and potentially curative, clinical outcomes. The company is advancing programs in autoimmune disease, oncology, and encoded protein therapeutics.

www.parcelbio.com

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

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

Gdansk Poland

(NASDAQ: PTG)

Immuthera and Poltreg SA (Parent) are pioneers in T-Regulatory cell therapy (Tregs). In the US, Immuthera is advancing the novel, first-in-class CD6-CTLA4 CAR-Treg which has been clinically validated in 6 cGVHD patients and has a second IND in Stage 3 T1D approved. Immuthera intends to open INDs for dermatological and neuroinflammatory basket studies. PolTREG is a fully integrated clinical stage company with extensive Treg experience, a pipeline of advanced Treg & mRNA immunotherapies, and commercial stage cGMP manufacturing. Our lead asset, PTG-007+Rituximab is in a Phase II study in presymptomatic Stage 1 T1D down to 3 yrs old. PTG-007 + Rituximab was deemed eligible for a European Marketing Authorization (MAA expected Q4 2027). Our anti-MOG CAR-Treg for Neuroinflammatory diseases will enter the clinic in Q4 2026. Our multi-edited Allogeneic CAR-Tregs and our In-vivo antigen specific Treg platforms are in late preclinical development.

www.poltreg.com

Cindy Atwell, Chief Development Officer and Chief Business Officer

Oct. 5 | 11:30am | 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 infectious diseases as well as rare diseases.

www.precisionbiosciences.com

Nicholas Richardson, Vice President, Clinical Development

Oct. 5 | 12:00pm | FLW Ballroom F

Bethesda, MA

(Private)

Precision for Medicine is an integrated global CRO and lab partner built for complex science—accelerating oncology and rare disease therapies from clinical development to commercialization with smarter execution, fewer handoffs, and faster decisions.

www.precisionformedicine.com

Curran Simpson, President and CEO

Oct. 5 | 11:00am | FLW Ballroom G

Rockville, MD

(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. For more information, please visit www.REGENXBIO.com.

www.regenxbio.com

Andres Isaias, CEO and Chairman

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

Miami, FL

(Private)

Restem is a clinical-stage biotechnology company focused on the development of next-generation, off-the-shelf cell therapies designed to modulate the immune system and address autoimmune, inflammatory, and age-related diseases.

www.restem.com

Richard Freed, CEO

Oct. 6 | 9:00am | FLW Ballroom G

Aurora, CO

(Private)

RheumaGen is a Colorado biotech company founded in 2019 that is engineering a cure for rheumatoid arthritis (RA), multiple sclerosis (MS) and other major autoimmune diseases using HLA gene editing. Put simply, RheumaGen can make small changes to the HLA gene, which will “turn off” the body’s ability to continue attacking itself. Our lead indication targets the ~1 million RA patients in the US, EU, Australia and Japan who are refractory to multiple biologics and face the most severe symptoms.

www.rheumagen.com

Guarav Shah, M.D., CEO

Oct. 5 | 11:30am | FLW Ballroom F

Cranbury, NJ

(NASDAQ: RCKT)

Rocket Pharmaceuticals, Inc. is a fully integrated biotechnology company advancing gene therapies for rare and devastating cardiovascular diseases, with additional programs in hematology and immunology. Rocket’s cardiovascular pipeline includes three clinical stage programs that each target one of the major inherited cardiomyopathy subtypes: hypertrophic, arrhythmogenic, and dilated cardiomyopathies. Together these conditions represent more than 100,000 patients in the U.S. and EU. The Company’s platform is supported by proprietary AAV manufacturing capabilities, multi-year efficacy and safety data in cardiac gene therapy, and experience treating several cardiac patients across late-stage AAV programs.

www.rocketpharma.com

Keita Mori, SanBio Co., Ltd. President

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

Tokyo, Japan

(4592.T)

Founded in California in 2001, SanBio develops, manufactures, and commercializes regenerative therapies for CNS disorders, addressing significant unmet medical needs.

www.sanbio.com

Tom Lowery, President and CEO

Oct. 6 | 3:45pm | FLW Ballroom G

Water Town, MA

(Private)

Satellite Bio is a clinical-stage regenerative medicine company developing the first sustainable, scalable, off-the-shelf hepatocyte cell therapies for severe liver disease. Our GMP-compliant hepatocyte expansion technology enables 10M+ fold expansion of primary human hepatocytes while preserving adult function, solving a supply bottleneck the field has faced for decades. Our manufacturing is more like a traditional drug than a cell therapy: centrally produced, cryopreserved, and shipped off-the-shelf at a cost of goods under $10,000 per dose. Our lead program, SB-101, is a first-in-human Phase 1/2 trial delivering functional metabolic rescue for infants with severe, early-onset urea cycle disorders (UCD), a population with 25–50% mortality under current standard of care. The FDA has granted SB-101 Rare Pediatric Disease Designation, placing us on a clear path to a Priority Review Voucher upon approval. Our pipeline extends to broader populations suffering from hepatic insufficiency, including both acute and chronic liver disease.

www.satellite.bio

David Parrot, Chief Financial Officer

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

Alameda, CA

(Private)

Scribe Therapeutics is a clinical-stage biotechnology company engineering CRISPR-based technologies into purpose-built in vivo genetic medicines designed to become standard-of-care treatments for patients suffering from highly prevalent diseases, starting with cardiometabolic disease. Leveraging its CRISPR by Design™ approach and nature’s blueprint for improved cardiovascular health, Scribe’s initial programs focus on addressing the key lipid drivers of ASCVD. The company’s lead candidate, STX-1150, is a novel liver-targeted therapy designed to epigenetically silence PCSK9 and reduce LDL-C levels without inducing permanent DNA changes. To broaden and accelerate the impact of its engineered CRISPR technologies for patients, Scribe has formed strategic collaborations 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 advancing scalable, transformative, and preventative genetic medicines with the goal of improving outcomes and democratizing access to protective effects of beneficial human genetics.

www.scribetx.com

Natalie Reid, Head of Cell Therapy

Oct. 5 | 2:30pm | FLW Ballroom G

Baltimore, MD

(Private)

SereNeuro Therapeutics is a biotechnology company developing next-generation cell and gene therapies to provide durable, non-opioid treatments for chronic pain and neurological disorders. By addressing the underlying biological causes of disease, SereNeuro aims to deliver transformative therapies that improve patients’ quality of life while advancing the future of regenerative medicine.

www.sereneuro.com

Jodi Cook, CEO

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

Cambridge, MA

(Private)

Skylark Bio is a clinical-stage genetic medicine company developing precision, locally delivered gene therapies for monogenic diseases, with the potential to meaningfully improve patient outcomes and quality of life. Its lead program, SKY-GJB2, is being evaluated in SONIX, a Phase 1/2 clinical trial for GJB2-related hearing loss, the leading cause of inherited hearing loss worldwide. Additional programs include SKY-PEN for SLC26A4-related hearing loss (Pendred syndrome) and an undisclosed program in CNS. Skylark’s proprietary gene therapy platform is built on an engineered capsid designed for broad transduction, enabling targeted microdose delivery, cell-specific expression control, and the potential for durable benefit in non-dividing cells. Led by a team with deep expertise in gene therapy and supported by world-class advisors, Skylark is advancing innovative therapies for hearing loss and other genetic disorders that profoundly affect patients and families.

skylarkbio.com

Brent Stead, CEO

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

Toronto, Ontario, Canada

(Private)

Specific Biologics is a venture-backed biotechnology company developing Dualase® to treat the untreatable with an industry-leading genome editing platform for precise, efficient, and programmable in vivo genome editing, with an initial therapeutic focus on the predictable collapse of pathogenic DNA repeat expansion disorders. The unique Dualase® 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. Specific is advancing its first program to clinical proof-of-concept in C9ORF72 amyotrophic lateral sclerosis (ALS), the most frequent inherited genetic form of terminal ALS with no disease-modifying therapies available today.

www.specificbiologics.com

Johanna Rossell, Senior Vice President, General Manager Regenerative Medicine

Oct. 6 | 3:45pm | FLW Ballroom F

Marlborough, MA

(Private)

Sumitomo Pharma Co., Ltd. is a global pharmaceutical company based in Japan with key operations in the U.S. (Sumitomo Pharma America, Inc.) and Canada (Sumitomo Pharma Canada, Inc.) focused on addressing patient needs in oncology, urology, women’s health, rare diseases, cell & gene therapies and CNS. With several marketed products in the U.S., Canada, and Europe, a diverse pipeline of early- to late-stage assets, we aim to accelerate discovery, research, and development to bring novel therapies to patients sooner.

www.us.sumitomo-pharma.com

David Nelles, CEO

Oct. 5 | 1:30pm | FLW Ballroom G

South San Francisco, CA

(Private)

Tacit Therapeutics is a development-stage biotechnology company developing Splicing-Directed Repair (SDR), an RNA trans-splicing platform that corrects disease-causing mutations at the pre-mRNA level. SDR rewrites mutant transcripts while preserving each gene’s native expression, splicing, and regulatory context, restoring healthy protein under the body’s own control. The platform generates 100K candidate designs per month, enabling rapid discovery across targets. Tacit’s lead programs address Huntington’s disease, repairing the HTT expansion to both reduce mutant HTT and reconstitute normal HTT levels, and Dravet syndrome, restoring SCN1A. A pipeline program addresses Rett syndrome. The company has raised approximately $20M, including a partnership with the Rett Syndrome Research Trust, and is advancing toward non-human primate proof-of-concept data supporting a Series B financing.

www.tacittx.com

Faraz Ali, CEO

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

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 pipeline includes clinical-stage candidates TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM); TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC); and TN-301, a highly specific small molecule HDAC6 inhibitor with broad potential clinical utility in cardiac, metabolic and muscular conditions, including heart failure with preserved ejection fraction (HFpEF) and Duchenne muscular dystrophy (DMD). Tenaya has employed a suite of integrated internal capabilities including modality agnostic target discovery and validation, to generate a portfolio of novel medicines based on genetic insights, aimed at the treatment of both rare genetic disorders and more prevalent heart conditions.

www.tenayatherapeutics.com

Hari Pujar, Chief Operations Officer

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

Cambridge, MA

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

Maddalena Pacelli, CEO and Founder

Oct. 5 | 9:00am | FLW Ballroom G

Rome, Italy

(Private)

Tiber Biotech is developing personalized monoclonal antibody therapies designed for individual patients, enabling unprecedented selectivity in the elimination of disease-causing lymphocytes whitout the off-target effects that limit today’s therapies. Its proprietary SAMBA platform combines bioinformatics and AI to identify patient-specific immune cell fingerprints and generate highly selective antibodies that spare healthy immune cells, creating a new therapeutic approach for blood cancers, autoimmune diseases, and transplant rejection. The company’s core IP lies in a scalable manufacturing platform for personalized N-of-1 therapies. By addressing process consistency rather than product consistency, Tiber Biotech is aligned with the evolving regulatory framework for individualized medicines and is pioneering the world’s first commercial platform for N-of-1 biologics.

www.tiberbiotech.it

Verna McErlane, Chief Commercial Officer

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

London, UK

(Private)

Touchlight is a leading, innovation-driven CDMO pioneering enzymatic GMP DNA production to enable the genetic medicine revolution. With an FDA Drug Master File accepted in 2022, followed in 2025 with the world’s first cell-free DNA GMP license, Touchlight’s enzymatic DNA technology is on the cutting edge of AAV, mRNA, DNA vaccine, and gene editing technology with its state-of-the-art facility recognised by ISPE’s Facility of the Year Awards (FOYA) for Innovation. With multiple client products already in the clinic, Touchlight’s evidence-based, synthetic DNA manufacturing solutions offer a scalable, sustainable alternative to plasmid DNA (pDNA) for all stages of pre-clinical, clinical and commercial development. The company and technology are built on the conviction that DNA is fundamental to the future of medicine. Founded in 2007, Touchlight’s team is inspired by breakthroughs in genetic medicine, recognising the urgent need for innovative DNA production techniques to support the future of genetic therapies.

www.touchlight.com

Mark Rothera, CEO

Oct. 5 | 4:45pm | FLW Ballroom F

(Private)

TreeFrog Therapeutics is an R&D-stage biotechnology company set to unlock access to cell-based therapies for patients with unmet needs. TreeFrog’s unique approach combines expertise in biophysics, cell biology and biomanufacturing to address key industry challenges – producing and differentiating high quality cell-based therapies at scale, cost-effectively. TreeFrog has developed a proprietary capsule technology platform C-stem™, which enables amplification of cells at an unprecedented scale and controlled differentiation into microtissues for regenerative medicine applications. To date, the platform has been demonstrated across multiple different cell types and tissues. To advance their purpose of ‘Cell Therapy for All’, TreeFrog operates a hybrid business model that includes programs in Parkinson’s and liver disorders aimed for co-development and out-licensing of the C-Stem platform. The company is open to partnerships with leading biotech and industry players in these and other therapeutics areas.

www.treefrog.fr

Richard Porter, Chief Business and Scientific Officer

Oct. 5 | 3:15pm | FLW Ballroom F

Amsterdam, Netherlands

(NASDAQ: QURE)

uniQure is delivering on the promise of gene therapy through an innovative adeno-associated virus (AAV)-based technology. 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

www.uniqure.com

Raimund Strehl, Chief Technology Officer

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

Gothenburg, Sweden

(Private)

VERIGRAFT was founded as a university spin-off and is headquartered in Gothenburg, Sweden. The company is developing personalized tissue-engineered grafts for regenerative medicine. Clinical development of P-TEV grafts, the first product, is ongoing, and safety of P-TEV has been confirmed.

www.verigraft.com

Andy Holt, Chief Commercial Officer

Oct. 6 | 2:15pm | FLW Ballroom F

San Sebastian, Spain

(Private)

At Viralgen, we’re all-in on AAV; in fact, that’s all we do. It’s made us a world leader in rAAV-based gene therapy manufacturing, built on a foundation of unmatched expertise and the world’s largest manufacturing capacity dedicated to AAV—and it’s led to a track record that includes more than 30 AAV serotypes, 1,500+ AAV batches and a 100% success rate for IND/IMPD dossiers. Our Aava™ manufacturing platform is a high-performance, real-world-ready solution that can get high-potential gene therapies to market quickly and efficiently at impressive scale, with better predictability and deeper data. We combine this cutting-edge infrastructure with end-to-end support, including plasmid manufacturing, construct optimization, all-inclusive pricing models, and total IP freedom to operate.

www.viralgen.com

Becky Cap, Senior Vice President, Biotherapies

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

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.

October 5-7 | Arizona Biltmore

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