Yuta Lee, CEO

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

Philadelphia, PA

(Private)

Accelerated Biosciences is a regenerative medicine company commercializing the human Trophoblast Stem Cell (hTSC). hTSCs are the earliest stem cells that can be sourced without ethical issues. The holy grail combination of scale and natural immune privilege makes it an ideal starting cell source for cell and gene therapies. Our indications are Parkinson’s Disease, Type 1 Diabetes, and Cell Senescence. We provide GMP cells with close to 100 patents to accelerate therapeutic developers’ product time to market.

www.acceleratedbio.com

Andrew Knudten, CEO

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

Cary, NC

(Private)

Advanced Medicine Partners delivers what patients deserve and what regulators expect.

The company provides partners with process development, manufacturing, analytical development and testing for advanced therapy products, specializing in viral vectors for gene and cell therapies. Advanced Medicine Partners leverages one of the most experienced genetic medicines teams in the industry, and its exclusive manufacturing platform consistently produces yields that are among the highest in the industry and purity profiles that push the boundaries of what’s possible. Advanced Medicine Partners’ proprietary adeno-associated virus (AAV) manufacturing process minimizes impurities and consistently generates industry-leading functional full capsid ratios. This manufacturing process prioritizes patient safety while reducing cost of goods. The company brings extensive operational experience and has manufactured upwards of 350 total non-GMP batches and supplied over 20 preclinical studies, including IND-enabling efficacy and GLP toxicology studies. The team has led CMC efforts for three globally approved gene therapies.

www.ampgtx.com

Tobias Winkler, M.D., Ph.D., Executive Board Member

Oct. 9 | 11:15am | FLW Ballroom G 

Berlin, Germany

(Private)

Advanced Therapies in Orthopaedics Foundation is an international specialist society founded as a non-profit association. Our aim is to provide an exclusive network of world-renowned researchers, clinicians and business experts to enable and accelerate the development of new innovative advanced therapies in the field of orthopaedics that will ultimately change the lives of patients worldwide. We wanted to create a solution to the disconnect between the scientific community and the world of finance and provide a multi-perspective approach to problems in the field. By breaking down the barriers and connecting qualified experts from around the world as an Alliance, the progression from initial ideas, over clinical trials to the final market adaptation reaches a new high in efficiency and advancement.

www.atioalliance.org

Rick Modi, CEO

Oct. 8 | 10:00am | FLW Ballroom G 

Waltham, MA

(Private)

Affinia Therapeutics is pioneering a new class of rationally designed gene therapies to treat rare and prevalent diseases. The Affinia Rationally designed Therapeutics (ART) platform synergistically improves the efficacy, safety, and manufacturability of adeno-associated virus (AAV)-based gene therapies through the development of next-generation capsids, promoters, and manufacturing approaches. Affinia’s novel capsids have shown significantly improved tropism to cardiac muscle, skeletal muscle, or the CNS with more uniform tissue distribution than AAV9 in nonhuman primates. Affinia has multiple best-in-class or first-in-class preclinical programs in genetic cardiomyopathies and diseases of the CNS.

www.affiniatx.com

James Brown, Ph.D., CEO & Co-founder

Oct. 9 | 9:15am | FLW Ballroom G 

Fargo, North Dakota

(Private)

Agathos Biologics is a biotechnology company pursuing transformational science in biomanufacturing, biologic payload delivery, and cell and gene therapy. Discoveries in bioprocessing and genetic characterization and control have created an abundance of scientific possibilities that can help us all lead better lives. Agathos has developed a proprietary cell line, AE1-BHK, for the production of recombinant adeno-associated viral vectors (rAAV). AE1-BHK is a potential alternative to HEK293 and avoids the ethical concerns regarding the use of cells derived from aborted fetal tissue. Our mission as the good science company is to create breakthrough products and services within a strong ethical and moral framework that benefit everyone. We believe in science that serves and have a relentless focus on serving our clients, employees, and society.

www.agathos.bio

Neil Cohn, Chief Operating Officer

Oct. 9 | 11:30am | FLW Ballroom F 

Dallas, TX

(Private)

Aileron uses public blockchain technology to create a secure data network, enabling stakeholders throughout the Cell and Gene Therapy ecosystem to share and access data seamlessly. The use of blockchain ensures robust security by providing immutable trust, data integrity, and traceability, safeguarding sensitive information at every step. Our decentralized data infrastructure facilitates peer-to-peer permissioned data transfer with fine-grained access control, allowing data creators to maintain sovereign ownership and control while ensuring robust security to protect sensitive patient data. Data harmonization of non-standardized sources enables interoperability across institutions and stakeholders’ data platforms. Key advantages:

– Data sovereignty across the platform

– Ensure robust security through immutable records, data integrity

– Trusted and transparent platform for secure data transactions.

– Ability to harmonize data without the data requiring standardization

www.ailerondata.com

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

Oct. 8 | 9:15am | FLW Ballroom G 

Ann Arbor, MI

(Private)

Akadeum Life Sciences is revolutionizing cell therapy separation workflows by enhancing quality of cells (better knockout efficiency), maximizing throughput, and by incorporating straightforward workflows. Akadeum’s new Alerion Microbubble Cell Separation System utilizes microbubble-based separation technology designed to provide the most gentle, fast, and high throughput instrument on the market. Akadeum’s flagship products focuses on the isolation of T cells for cell and gene therapy applications and are available as clinical ready GMP grade kits. Akadeum will be fully releasing the Alerion, providing automated capability for positive selection, negative selection, and depletion at scales previously not available in existing workflows.

www.akadeum.com 

Harald Stover, Ph.D., CEO

Oct. 7 | 2:30pm | FLW Ballroom G 

Hamilton, Canada

(Private)

Allarta Life Science is a Canadian regenerative medicine company, founded in 2019 by McMaster’s Chemistry Professor Dr. Harald Stover and Maria Antonakos. Allarta’s unique platform of synthetic hydrogel technology is enabled by 30+ years of foundational expertise and its modular design advances next generation cell therapies. Allarta’s hydrogels allow transplanted cells to connect metabolically while protecting them from the host’s immune system, both key requirements for allogeneic cell therapies. Allarta aims to deliver functional cell-based cures for inflammatory and metabolic disorders, including diabetes, without the need for immune suppression. In 2023, Allarta entered an IDDP partnership with Breakthrough T1D (formerly JDRF) to conduct key pre-clinical studies towards its cell-based therapy. In addition, Allarta develops proprietary cryoprotective gels that prevent cell aggregation and minimize ice crystal formation, and thus help ensure delivery of high value cell payloads at scale. This novel technology offers safe transportation and cryostorage of therapeutic cell products.

www.Allarta.com

Mark Farmery, Chief Development Officer

Oct. 8 | 11:30am | FLW Ballroom G 

Södertälje, Sweden

(Private)

Anocca is a rapidly growing Swedish biotechnology company that has built end-to-end capabilities to enable systematic T-cell receptor-engineered T-cell therapy (TCR-T) development at scale to redefine the treatment of solid tumor cancers. The proprietary Anocca technology platform precisely maps HLA-peptide T-cell targets on cancer cells and identifies highly specific and potent TCRs that recognise those targets, leading to the creation of validated TCR asset libraries that can be matched with a patient’s tumor profile and deployed in personalized TCR-T cell therapies. The Anocca pipeline comprises over 40 preclinical TCR assets that target driver mutations, dysregulated tumor-specific genes and onco-viral antigens. Using a novel gene-edited engineering approach, Anocca’s integrated in-house process development and GMP manufacturing capabilities enable the clinical development of its advanced autologous TCR-T cell therapy products. Anocca’s first multi-asset clinical program targets cancers with KRAS oncogenic driver-mutations and is entering clinical development in a hard-to-treat solid tumor indication.

www.anocca.com 

Rachel Salzman, CEO

Oct. 9 | 11:45am | FLW Ballroom G

Columbus OH

(Private)

Armatus Bio, a late preclinical stage company, is developing best-in-class vectorized RNAi therapeutics with technology licensed from Nationwide Children’s Hospital with the goal of addressing urgent unmet needs in genetically-driven neurological diseases. The company is advancing a lead candidate targeting Charcot-Marie-Tooth Type 1A (CMT1A) that is targeting an IND submission in 2025, and a second program for Facioscapulohumeral Muscular Dystrophy (FSHD).

www.armatusbio.com

Tom Bollenbach, Ph.D., Chief Technology Officer

Oct. 9 | 9:00am | FLW Ballroom G 

Manchester, NH

(Private)

ARMI|BioFabUSA’s mission is to develop a highly diverse, competitive, capable, and innovative domestic cell, tissue, and organ manufacturing ecosystem that will fundamentally change healthcare for chronic disease and traumatic injury; and the trained and ready workforce essential for that ecosystem, centered in Manchester NH, with national impact.

www.armiusa.org

Neha Krishnamohan, Chief Financial Officer and EVP, Corporate Development

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

San Diego, CA

(Private)

Artiva is a clinical-stage company whose mission is to deliver broadly accessible, off-the-shelf natural killer (NK) cell-based therapies to patients suffering from devastating autoimmune diseases and cancers. Artiva’s lead program, AlloNK®, is a non-genetically modified, allogeneic, cryopreserved NK cell therapy candidate being developed to enhance the activity of B-cell targeting monoclonal antibodies to drive B-cell depletion. AlloNK® is currently in clinical trials in combination with rituximab for treatment of patients with lupus nephritis and for treatment of NHL, as well as in combination with Affimed’s innate cell engager AFM13 for the treatment of patients with relapsed/refractory CD30+ lymphomas. Artiva’s pipeline also includes CAR-NK candidates targeting both solid and hematologic cancers. Artiva’s cell therapy manufacturing platform incorporates cell expansion, activation, and engineering technology developed by Artiva’s strategic partner, GC Cell Corporation, a member of the GC family of companies, a leading healthcare company in Korea.

www.artivabio.com

Daniel Rosan, Chief Financial Officer, Chief Business Officer 

Oct. 9 | 10:15am | FLW Ballroom F 

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 

Tamer Mohamed, Founder and CEO

Oct. 7 | 2:15pm | FLW Ballroom G 

Vancouver, BC, Canada

(Private)

Aspect Biosystems is a biotechnology company pioneering the development of bioprinted tissue therapeutics to transform the treatment of currently incurable diseases. Aspect is creating these next-generation cell therapies by applying its full-stack tissue therapeutic platform, which combines the company’s proprietary bioprinting technology, therapeutic cells, biomaterials, and computational design. Aspect’s bioprinted tissue therapeutics are designed to replace, repair, or supplement biological function inside the body. The company’s current therapeutic pipeline is focused on metabolic and endocrine diseases such as diabetes, obesity, and congenital liver disorders.

www.aspectbiosystems.com

Ha Tran, M.D., Medical Head, Cell and Gene Therapy

Oct. 7 | 3:00pm | FLW Ballroom F 

Tokyo, Japan

(NASDAQ: ALPMY)

At Astellas, we aim to discover, develop and deliver breakthrough gene therapies with life-changing VALUE for patients across many disease areas. We are developing one of the world’s most robust pipelines of gene therapies for genetic disorders and an end-to-end gene therapy powerhouse with industry-leading discovery, development, manufacturing and commercial capabilities. We have two candidates in clinical development for rare neuromuscular diseases: AT845, in Phase 1/2 for Pompe disease and AT132 for X-linked myotubular myopathy, currently on clinical hold. We are also working to build our portfolio across other indications including A2016 for cardiac complications associated with Friedreich ataxia, which has received FDA IND clearance. Alongside our 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

Stephane Degove, CEO

Oct. 8 | 5:45pm | FLW Ballroom F 

Paris, France

(Private)

Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases and cardiomyopathies. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD), with two clinical-stage programs targeting respectively LGMD2I/R9 and LGMD2C/R5.

www.atamyo.com

Greg Kunst, CEO

Oct. 7 | 5:15pm | FLW Ballroom F 

Seattle, WA

(Private)

Aurion Biotech is a clinical-stage biotech company, whose mission is to restore vision to millions of patients with life-changing regenerative therapies. It received the prestigious Prix Galien award for best start-up in biotech. Its first candidate is for the treatment of corneal edema secondary to corneal endothelial disease, and the first clinically validated cell therapy for corneal care, having received regulatory approval in Japan. The Company has completed enrollment and dosing of its Phase 1 /2 clinical trial in the U.S. and Canada. Privately held, Aurion Biotech is backed by Deerfield, Alcon, Petrichor, Flying L Partners, Falcon Vision / KKR, and Visionary Ventures.

www.aurionbiotech.com 

Alexander Seyf, CEO

Oct. 9 | 11:45am | FLW Ballroom F

London, UK

Autolomous specializes in developing, implementing, and integrating software-based solutions tailored to CGT challenges. By harnessing new, emerging, and disruptive technologies, we bring unparalleled efficiency, world-class security, and assured compliance to CGT manufacturing. We enable manufacturers to scale through a fully integrated, digitized supply chain ecosystem. We proudly serve a diverse range of clients, including Academia, CDMOs, and Biotech companies across UK, Europe, and US. Our flagship platform, autoloMATE, revolutionizes how our partners book, manufacture, and deliver treatments. This intuitive, zero-code solution increases efficiency by 40% across all CGT modalities, while ensuring products are compliant, safe, and cost-effective. autoloMATE seamlessly integrates with key adjacent solutions. We have successfully integrated with 21 external systems from key suppliers within the industry, creating a seamless, connected ecosystem. Founded in 2019 by seasoned professionals, Autolomous combines fifty years of experience in advanced therapies and medical practice with thirty-five years in technology, software, and business.

www.autolomous.com

Jordan Lancaster, Ph.D., CEO

Oct. 8 | 1:30pm | FLW Ballroom G 

Tucson, AZ

(Private)

Avery Therapeutics is a late-stage pre-clinical company developing a new class of restorative medicine for patients with intractable diseases of inflammation and fibrosis by levering the therapeutic potential within the immune system. Avery’s lead clinical candidate AT-102 uses our proprietary MPhase biologic platform to address heart failure, one of the leading causes of death in the world. In pre-clinical models, AT-102 structures the heart to improve and establishes best-in-class efficacy.

www.averythera.com

Lisa Deschamps, CEO and Executive Board Member

Oct. 7 | 5:30pm | FLW Ballroom F 

London, UK

(Private)

At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, we are working hard 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 reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the company for success in bringing transformative medicines to patients.

www.aviadobio.com

Elie Hanania, Ph.D., Vice President, Process Development, Viral Vector Technologies

Oct. 7 | 4:00pm | FLW Ballroom G 

Tustin, CA

(NASDAQ: CDMO)

Avid Bioservices is a dedicated contract development and manufacturing organization (CDMO) focused on development and CGMP manufacturing of biologics. The company provides a comprehensive range of process development, CGMP clinical and commercial manufacturing services for the biotechnology, biopharmaceutical, and cell & gene therapy industries. With over 30 years of experience producing biologics, Avid’s services include CGMP clinical and commercial drug substance manufacturing, bulk packaging, release and stability testing and regulatory submissions support. For early-stage programs the company provides a variety of process development activities, including cell line development, upstream and downstream development and optimization, analytical methods development, testing and characterization. The scope of our services ranges from standalone process development projects to full development and manufacturing programs through commercialization.

www.avidbio.com

Tiffany Brown, Ph.D., CEO

Oct. 9 | 10:45am | FLW Ballroom F 

Newark, Delaware

(Private)

Avobis Bio is a clinical-stage biotechnology company dedicated to the development of implantable cell therapies to treat debilitating conditions. The investigational portfolio leverages the combination of mesenchymal stem cells and bioabsorbable materials to create novel curative treatments. In an ongoing multicenter randomized Phase II clinical trial, the investigational cell therapy AVB-114 is being assessed for persistent Crohn’s anal fistulas. Formed as a joint venture between Mayo Clinic and W. L. Gore & Associates, Avobis leverages expertise in clinical care, cell biology, materials science and commercialization of medical products to transform patient lives.

www.avobisbio.com

Tina Self, Senior Vice President, Global Head of Biologics Manufacturing; Berkeley Site Head

Oct. 8 | 1:15pm | FLW Ballroom F 

Leverkusen, Germany

(NASDAQ:BAYN)

Bayer has invested €3.5 billion in the build-up of its cell and gene therapy pipeline. Its innovation engines, BlueRock Therapeutics and AskBio, have exciting programs across multiple indications in preclinical through Phase 2. The company has collaborations with Mammoth Biosciences and Acuitas in the CGT space as well. Bayer has partnered with BlueRock to industrialize processes and will manufacturer for late stage/commercial supply of DA01, BlueRock’s Parkinson’s candidate, and supports pre-clinical development programs at both BlueRock and AskBio. Bayer is also working with Cytiva to set industry standards with the first end-to-end allogeneic CT manufacturing platform. In our mission’s spirit, “Health for All”, Bayer has created a CGT-focused incubator in Cambridge, MA to support startups; additional global locations are coming online. We are also helping external innovators make their therapeutic candidates a reality through BioPartnering Solutions you can leverage industry-leading biotech process development and biomanufacturing capabilities.

www.bayer.com

Joanne Smith-Farrell, Ph.D., CEO

Oct. 8 | 3:30pm | FLW Ballroom G 

Cambridge, MA

(Private)

Be Biopharma (“Be Bio”) is pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of patients who are living with Hemophilia B and other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies. Be Bio was founded in October 2020 by B cell engineering pioneers David Rawlings, M.D., and Richard James, Ph.D., from Seattle Children’s Research Institute. Be Bio is backed by ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children’s Research Institute and others. Since our founding, Be Bio’s investors have committed over $180 million to enable the Company to re-imagine medicine based on the power of B cell therapy.

www.be.bio

Kathryn Corzo, President and Chief Operating Officer

Oct. 8 | 2:00pm | FLW Ballroom G 

Cambridge, MA

(Private)

bit.bio is a synthetic biology company focused on human cells, advancing medicine and enabling curative treatments. The company does this by industrializing the manufacture of human cells and making them more accessible. bit.bio’s opti-ox™ deterministic cell programming technology enables conversion of induced pluripotent stem cells into any desired human cell type in a single step, at industrial scale, while maintaining exceptional purity and consistency. The company has a cell therapy pipeline, based on txCells, focused on serious diseases that lack effective treatments. The ioCells research cell product portfolio is opening up new possibilities in research and drug discovery.

www.bit.bio

Jamie Sharp, Ph.D., Associate Director of Programs

Oct. 8 | 5:45pm | FLW Ballroom G 

Montreal, Canada

(Private)

C3i Center Inc is a leading CDMO in Canada offering in-house turnkey services for CGTs from process development to commercialization. C3i is the only CDMO in Canada with a Drug Establishment Licesnse (DEL) for the manufacture of a commercial cell therapy. C3i expects to receive EMA approval for the commercial manufacture cell therapy for its facility in Canada for EU market which will make it one of a few CDMOs in NA with such approval. C3i has developed phase appropriate QC testing and CAP-accredited labs offering immune monitoring, diagnostics and biomarker development, clinical CRO servicesas well as investing in innovative CGT therapy companies. C3i personnel have been manufacturing cell therapies for over 15 years strong process development experience in diverse cell types including: CAR-Ts, Treg, TCRs, MSCs, DC, IPSCs, allogeneic, viral and non-viral transduction and CRISPR-editing. C3i is partner-focused, patient-driven whereby failure is not an option. C3i has a high manufacturing success rate of 99% as we understand that patient’s time is measured in days with no room for error. C3i works hard to integrate cost-efficiency, we offer very attractive COGS.

www.centrec3i.com

AJ Bergmann, Chief Financial Officer

Oct. 8 | 9:45pm | FLW Ballroom F 

San Diego, CA

(NASDAQ: CAPR)

Capricor Therapeutics is a publicly traded (NASDAQ: CAPR) 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 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.

www.capricor.com 

Peter Anastasiou, CEO and Board Member

Oct. 8 | 3:45pm | FLW Ballroom G 

Thousand Oaks, California

(Private)

Capsida Biotherapeutics is a fully integrated gene therapy company with a central nervous system (CNS) pipeline consisting of disease modifying and potentially curative treatments for rare and more common diseases across all ages. Capsida’s systemically delivered gene therapies utilize proprietary engineered capsids that enable high transduction levels to desired tissues and cells, while limiting tropism to non-target organs, such as the liver. Capsida has three wholly owned programs, including potential best-in-class treatments for genetic epilepsy due to STXBP1 mutations and Parkinson’s disease associated with GBA mutations (PD-GBA), both of which are in IND-enabling studies. In addition to its wholly owned programs, the Company has CNS partnerships with AbbVie, Lilly, CRISPR Therapeutics, and the AbbVie partnership was expanded to include ophthalmology disorders. Capsida was founded in 2019 by Versant Ventures and Westlake Village BioPartners and originated from the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech.

www.capsida.com 

Adrian Bot, M.D., Ph.D., Chief Scientific Officer and Executive Vice President of R&D

Oct. 7 | 4:15pm | FLW Ballroom G 

San Diego, CA

(Private)

Capstan’s mission is to multiply the therapeutic possibilities for patients by developing targeted in vivo RNA-based therapies. Our core platform technology comprises proprietary targeted lipid nanoparticles (tLNPs) that are composed of LNPs conjugated with a recombinant protein binder, such as a monoclonal antibody, and are designed to deliver payloads, including mRNA or gene editing tools, capable of reprograming specific cell types in vivo. Our technology enables us to pursue two distinct approaches to treating disease: engineering immune cells and targeting pathogenic cells. In vivo engineering of immune cells: we can deliver mRNA-encoded CARs to specific subsets of immune cells, programming them to eliminate pathogenic cells (autoimmune or cancerous) that express antigens recognized by the CAR. Targeting of pathogenic cells: by delivering mRNA-encoded gene editing machinery to pathogenic cells, we can modify the cells’ DNA to treat genetic disease. Capstan is a post series B stage company, based in San Diego.

www.capstantx.com

Tim Kelly, Chief Technology Officer 

Oct. 8 | 4:30pm | FLW Ballroom G 

Berkeley, CA 

(NASDAQ: CRBU) 

Caribou Biosciences is a clinical-stage CRISPR genome-editing biopharmaceutical company dedicated to developing transformative therapies for patients with devastating diseases. The company’s genome-editing platform, including its Cas12a chRDNA technology, enables superior precision to develop cell therapies that are armored to potentially improve activity against disease. Caribou is advancing a pipeline of off-the-shelf cell therapies from its CAR-T platform as readily available treatments for patients with hematologic malignancies and autoimmune diseases. 

www.cariboubio.com 

Frederic Cedrone, Ph.D., Vice President, Corporate Innovation

Oct. 7 | 3:30pm | FLW Ballroom G 

Somerset, New Jersey

(NASDAQ: CTLT)

Catalent Cell & Gene Therapy is an industry-leading technology, development, and manufacturing partner for advanced therapeutics. Its comprehensive cell therapy portfolio includes a wide range of expertise across a variety of cell types including CAR-T, TCR, TILs, NKs, iPSCs, and MSCs. With deep expertise in viral vector development, scale-up and manufacturing for gene therapies and viral vaccines, Catalent is a full-service partner for plasmid DNA, adeno-associated viral (AAV), lentiviral and other viral vectors, and oncolytic viruses. As an experienced and innovative partner, it has a global network of dedicated, development, clinical, and commercial manufacturing facilities, including an EMA- and FDA-licensed viral vector facility, and fill/finish capabilities located in the U.S. and Europe. With integrated solutions for plasmid DNA, viral vectors, and autologous and allogeneic cell therapies through clinical trial packaging and logistics, Catalent can provide full supply chain control, helping innovators get their advanced therapies to patients, faster.

biologics.catalent.com

Nabiha Saklayen, CEO

Oct. 8 | 11:45am | FLW Ballroom G 

Cambridge, MA

(Private)

Cellino is building an autonomous biomanufacturing platform for personalized regenerative medicines derived from autologous induced pluripotent stem cells (iPSCs), which can be differentiated into a wide range of cell and tissue types to treat conditions including Parkinson’s disease, spinal cord injury, and age-related macular degeneration. Current approaches to manufacturing iPSC-derived therapies are manual and artisanal and are not scalable to meet the needs of growing patient populations. Cellino’s AI-guided platform makes in-process decisions using image-guided machine learning (ML) algorithms and removes unwanted cells using laser-induced bubbles. This avoids contamination by enabling passage-free cell culture, offers single-cell precision, is closed-compatible using proprietary, patient-specific cell cassettes, and requires minimal operator oversight. This process brings a significant cost reduction in iPSC manufacturing compared to manual methods. Cellino’s platform automates iPSC manufacturing to make “Your cells, your cure” a reality for patients.

www.cellinobio.com

Gustavo Mahler, Ph.D., CEO

Oct. 7 | 9:30am | FLW Ballroom G 

Gosselies, Belgium

(Private)

Cellistic specializes in process development and manufacture of immune cell therapies based on human induced pluripotent stem cell (iPSC) technology using their Pulse and Echo Platform. Its focus and expertise in iPSC reprogramming, gene editing and differentiation development positions the company to be the partner of choice for innovative cell therapy developers to advance into clinic. Leveraging more than a decade of scientific and technical knowledge and experience, Cellistic possesses unique capabilities for the design and optimization of proprietary manufacturing platforms for iPSC- based cell therapies.

www.cellistic.com

Conrad Russell Cruz, M.D., Ph.D., Principal Investigator, Program for Cell Enhancement and Technologies for Immunotherapies

Oct. 8 | 5:30pm | FLW Ballroom G 

Washington, DC

(Private)

Children’s National Hospital, based in Washington, D.C., was established in 1870 to help every child grow up stronger. Today, it is the No. 5 children’s hospital in the nation and ranked in all specialties evaluated by U.S. News & World Report. Children’s National is transforming pediatric medicine for all children. The Children’s National Research & Innovation Campus opened in 2021, a first-of-its-kind pediatric hub dedicated to developing new and better ways to care for kids. Children’s National has been designated three times in a row as a Magnet® hospital, demonstrating the highest standards of nursing and patient care delivery. This pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty care locations in the D.C. metropolitan area, including Maryland and Virginia. Children’s National is home to the Children’s National Research Institute and Sheikh Zayed Institute for Pediatric Surgical Innovation. It is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. As a non-profit, Children’s National relies on generous donors to help ensure that every child receives the care they need.

www.childrensnational.org

Joseph Tarnowski, Ph.D., Chief Technology and Business Officer

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

South San Francisco, CA,

Founded in 2014 to address the high cost and capacity issues facing therapeutic protein, and cell and gene therapy, manufacturers with cutting-edge technologies for creating high-productivity cGMP compliant mammalian cell lines.

www.cho-plus.com

Taby Ahsan, Ph.D., Vice President, Cell and Gene Therapy Operations

Oct. 9 | 9:30am | FLW Ballroom G  

Duarte, California 

(Private) 

City of Hope has over 20 years of experience and operates one of the nation’s largest academic GMP facilities. Our team of 100+ dedicated staff equally engages with internal programs and supports external academic institutes and biotechnology companies across the nation. We excel in swiftly advancing therapeutic concepts from the research stage to Phase 1/2 clinical trials. External groups can leverage our diverse array of established platform processes or work with our expert teams to develop bespoke manufacturing. We streamline and accelerate translation for intermediate and final drug products of: 

• Viral Vectors and Oncolytic Vaccines (e.g., LV, RV, POX, MVA, HSV)
• Stem Cell Therapies (e.g., iPSC-derived phenotypes, MSCs, NPCs)
• Cellular Immunotherapies (e.g., CAR-T, CAR-NK, TIL)

www.cityofhope.org/research/biological-and-cellular-gmp-manufacturing-facility 

Brian McVeigh, Chairman, CEO, and Co-Founder

Oct. 7 | 2:45pm | FLW Ballroom G 

Lower Gwynedd, PA

(Private)

Code Bio is a genetic medicine company pioneering the development of a new class of precision therapies to treat life-threatening genetic diseases. Code Bio’s proprietary, novel, multivalent, non-viral, synthetic, DNA delivery platform ‒ known as 3DNA® ‒ is unlike viral and other nonviral delivery approaches. The 3DNA platform offers more precise cell targeting, the ability to redose, a scalable, modular manufacturing process, and the ability to deliver large genetic payloads. Code Bio is advancing an internal pipeline of candidates while establishing partnerships to develop therapies to treat currently untreatable diseases.

www.codebiotx.com

Mo Heidaran, Ph.D., Chief Regulatory Scientist

Oct. 9 | 10:15am | FLW Ballroom G 

Rockville, Maryland

(Private)

The foundation, CHCF was formed in 2020 to promote research and product development to develop FDA cleared diagnostic tests that enables early detection of the diseases of brain with behavioral and psychological manifestations using unbiased state of the art genetic and epigenetic technologies and tools. In parallel our secondary goal is to promote translational and applied research by conducting well powered, prospective, longitudinal observational trials to identify root causes of serious mental health conditions by detecting blood based biomarkers that are either associative or causative at both molecular and biological levels using advanced measurement technologies. Identification of associative or causative biomarkers should pave the way toward the development of targeted gene therapies for the diseases of brain that involve life threatening mental health manifestations.

www.chcfbrain.org

Kilian Kelly, Ph.D., CEO

Oct. 8 | 10:45am | FLW Ballroom F 

Melbourne, Australia

(ASX:CYP)

Cynata Therapeutics Limited is focused on the commercialization of a proprietary induced pluripotent stem cell (iPSC)-based platform technology, Cymerus™. The Cymerus platform enables scalable manufacture of mesenchymal stem cell (MSC)-based therapeutic products. The primary advantage of the Cymerus platform is its ability to produce an effectively limitless quantity of consistent, high quality MSCs from a single blood donation. This avoids challenges associated with conventional MSC manufacturing methods, including the need for new donations from different donors on an ongoing basis, which is associated with substantial variability and potential impacts on MSC functionality. Cynata’s initial clinical trial in GvHD, which has led to two publications in Nature Medicine, was the first ever completed clinical trial with iPSC-derived cells. There are now four active Cymerus MSC clinical programs (GvHD, osteoarthritis, diabetic foot ulcers and kidney transplantation), with results of three randomized controlled trials expected between late 2024 and early 2026.

www.cynata.com 

Colleen Delaney, M.D., Founder and Chief Scientific Officer

Oct. 7 | 9:45am | FLW Ballroom F 

Seattle, WA

(Private)

Deverra is a clinical-stage biotechnology company in Seattle developing allogeneic, off-the-shelf universal cellular immunotherapies. Deverra’s mission is to ensure that no patient is left untreated due to requirements for donor matching, complex and high cost of manufacturing, or cell therapy product availability when it is needed most. Starting from FDA-regulated pooled umbilical cord blood, Deverra leverages a proprietary stem cell expansion technology for scale followed by directed differentiation to generate many distinct immune effector cell types. This results in a diverse pipeline of therapies, including the potential for combination cellular therapy approaches. Dilanubicel, a universal allogeneic ex vivo expanded progenitor cell therapy and Deverra’s lead clinical asset, demonstrated a 95% increase in initial remission rates when used in upfront AML therapy in a global randomized controlled phase 2 trial. Based on these safety and preliminary efficacy outcomes, the FDA has granted Dilanubicel RMAT, Orphan Drug, and Fast Track special designations.

www.deverratx.com

Caralynn Nowinski Collens, M.D., CEO

Oct. 8 | 11:15am | FLW Ballroom G 

Chicago, IL

(Private)

Dimension Inx is a tissue therapeutics company. The company designs, develops, and manufactures 3D-printed therapeutic products that restore tissue and organ function. Using its advanced biomaterials platform, Dimension engineers’ three-dimensional microenvironments that direct cell behavior. This pioneering approach overcomes many challenges in cellular therapeutics, including therapeutic potency, cell viability and engraftment, and costs of manufacturing. Dimension focuses on creating clinical solutions not otherwise possible. The company’s current pipeline includes lead programs in Type 1 Diabetes and liver disease.

www.dimensioninx.com

Flagg Flanagan, CEO and Chairman

Oct. 7 | 11:15am | FLW Ballroom F 

Salt Lake City, UT

(Private)

DiscGenics is a privately held, late-stage clinical biopharmaceutical company developing allogeneic, cell-based regenerative therapies for musculoskeletal degeneration. It’s lead product candidate, IDCT (injectable disc cell therapy, or rebonuputemcel), is a standalone, single-injection biologic treatment designed to halt progression of lumbar disc degeneration and regenerate the disc from the inside out. IDCT is entering pivotal Phase III trials in the U.S. to target pain and disability in patients with lumbar disc degeneration. The active ingredient in IDCT is a live, manufactured progenitor cell population derived from donated adult human intervertebral disc tissue, mixed with a viscous carrier. IDCT has been granted regenerative medicine advanced therapy (RMAT) and Fast Track designations by the U.S. Food and Drug Administration. DiscGenics is also developing a follow-on allogeneic cell platform to enable new musculoskeletal indications. To further development of these unique therapies, DiscGenics has built and validated an in-house scalable, allogeneic cell manufacturing process.

www.discgenics.com 

Don Haut, Ph.D., Chief Business Officer

Oct. 9 | 11:00am | FLW Ballroom F 

Paris, France

(Private)

EG 427 develops a unique vector platform to deliver pinpoint neurotherapeutics to treat prevalent diseases of the peripheral and central nervous system. Our 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 either for long-term gene therapy, or all-in-one gene editing approaches. EG 427 is the second company to bring a non-replicating HSV-1 vector into clinical development with an IND filed with the FDA in May 2024. Our first product, EG110A, addresses multiple severe bladder diseases (including Neurogenic Bladder and Overactive Bladder) by targeting selectively sensory neurons and has the potential to be a major improvement over existing therapies, resulting in better care for the patients and lower costs for the healthcare systems.

www.eg427.com

Will Olsen, CEO

Oct. 8 | 9:15am | FLW Ballroom F 

San Carlos, CA

(Private)

Engage Bio is developing Tethosomes, a new class of non-viral genetic medicines engineered for potency, safety, and programmability. Tethosomes achieve over 100x higher expression than traditional DNA vectors. Further, Tethosomes evade detection by the innate immune system, finally enabling safe and redosable non-viral DNA therapies. The programmability and specificity of Tethosomes enable broad therapeutic applications ranging from gene replacement to cancer immunotherapy.

www.engagebio.com

Andreas Scheel, Ph.D., EVP Head of Cell Therapy

Oct. 8 | 9:00am | FLW Ballroom G 

Hamburg, Germany

(NASDAQ:EVT)

Evotec is a biotechnology 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 from inception to the clinic, including in-house GMP manufacturing capabilities with clinical supply. Our proprietary iPSC-based cell therapy pipeline is one of the broadest in the industry and includes anti-tumor therapies (NK cells, macrophages, αβ T cells, γδ T cells), regenerative therapies (beta cells, cardiomyocytes, photoreceptors) and immune-modulation therapies. 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 therapy candidates.

www.evotec.com

Robert Tressler, Ph.D., Chief Scientific Officer

Oct. 7 | 10:15am | FLW Ballroom G 

San Diego, CA

(Private)

Excellos is a CDMO specializing in servicing the adaptive immunotherapies space on behalf of our Biopharma clients. Our services span source material supply, characterization, cell manufacturing and gene editing for CAR, T, NK, TIL and HSPC-based immunotherapies for the treatment of oncology, autoimmune and regenerative medicine indications. We have developed a novel E360 donor and cell assessment platform that is a unique approach to identify preferred allogeneic donors to serve sources of high quality starting materials for allogeneic adaptive immunotherapy-based approaches. The E360 assessment tool also can be used to monitor the quality of manufacturing intermediates, support QbD process development activities and characterize final product to assure an optimal final product for the patient. We also offer custom testing and characterization for potency, identity and purity as well as cell expansion services with a focus on supplying more consistent optimal products.

www.excellos.com

David Millette, CEO

Oct. 7 | 2:45pm | FLW Ballroom F 

Montreal, Canada

(Private)

ExCellThera is world leader in enhanced blood stem cell expansion and therapies. Its most advanced product UM171 Cell Therapy (INN-dorocubicel), is in late stages of development. A Market Authorization Application for UM171 Cell Therapy has been accepted for an accelerated assessment by EMA for the treatment of patients with hematological malignancies requiring an HSCT who lack a suitable donor. Following a favorable EOP2 meeting with FDA, a Phase-3 trial is planned in high and very-high risk acute leukemias and myelodysplasias for which limited treatment options exist with low survival and high incidence of relapse under standard of care. UM171 Cell Therapy in other indications, including pediatric patients, mutliple myeloma, and non-malignant hematological diseases, is also explored. ExCellThera partners with biopharmas to them help develop best-in-class cell and gene therapies by leveraging proprietary molecule UM171 which has first-in-class mechanism of action for ex-vivo expansion and metabolic fitness of HSCs.

www.excellthera.com 

Romain de Rauville, Chief Business Officer

Oct. 8 | 10:15pm | FLW Ballroom G 

Liege, Belgium

(Private)

Exo Biologics is a clinical stage company based on Belgium and a pioneer of the EV (extra-cellular) field. EXOB-001 is our lead asset and is currently in clinical phase I in EU through a multicentric trial. EXOB-001 is composed of MSC derived EVs and is used to treat Broncho Pulmonary Dysplasia which is a rare disease related to inflammation of the lung of pre-term babies. Exo Biologics close its first cohort in Q2 2024. EXOB-001 is the first ever MSC-EV product to enter clinical trial in EU making Exo Biologics the most advanced company in this regard. More specifically, Exo Biologics is building the regulatory pathway for EV in EU. In addition Exo Biologics has developed the capacity to load EV with RNA in a GMP compliant way. This technology is key to build non-viral drug delivery systems as alternative to AAV and LNP. The ability to use the safety profile of the EV makes it a viable candidate for safe product and redosing.

www.exobio.be

Hamid Khoja, Ph.D., Chief Scientific Officer

Oct.7 | 5:45pm | 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 technology serves as the foundation for developing biologic cures and treatments for chronic diseases, including chronic wounds (diabetic foot ulcers), multiple sclerosis, degenerative disc disease, psoriasis, anti-aging extension of life applications such as thymic involution reversal, and cancer. FibroBiologics holds 150+ US and internationally issued patents/patents pending across various clinical indications, including disc degeneration, orthopedics, multiple sclerosis, autoimmune disorders, wound healing, reversing tymus involution, and cancer. FibroBiologics represents the next generation of medical advancement in cell therapy.

www.FibroBiologics

Timothy Kieffer, Ph.D., Chief Scientific Officer

Oct. 9 | 9:15am | FLW Ballroom F 

Burlington, Massachusetts

(NASDAQ: GUTS)

We are a metabolic therapeutics company focused on pioneering new approaches for the treatment of metabolic diseases, including type 2 diabetes and obesity. Our goal is to transform metabolic disease treatment from chronic symptomatic management to durable disease-modifying therapies that target the organ-level root causes of T2D and obesity. We believe there is significant clinical and economic opportunity for new approaches to achieve a major leap forward with new disease-modifying strategies that are designed to target and potentially reverse root cause pathology of these diseases.

We have assembled a mission-driven team of innovators and experts in the root causes of metabolic disease, all united in their passion to develop breakthrough therapies—the type of therapies we would want for ourselves, our family members, and our loved ones with these diseases.

www.fractyl.com

Laura M. Borgelt, Chief Operating Officer, Gates Institute

Oct. 7 | 10:00am | FLW Ballroom G 

Aurora, CO

(Private)

Gates Institute is a premier cell therapy translational research institute with biomanufacturing capabilities delivering first-in-human therapies. Based at the University of Colorado Anschutz Medical Campus, we are part of a rich biomedical ecosystem, enabling the development of life-saving treatments. We bring together and support researchers and clinicians who specialize in regenerative, cell and gene therapies, accelerating their discoveries from concepts to cures. The Gates Institute and the Gates Biomanufacturing Facility also partner with private industry for the manufacture and administration of cell therapies for Phase I trials.

www.gates.cuanschutz.edu

Angela Columbano, Ph.D., Head Business Development and Partnership

Oct. 7 | 3:30pm | FLW Ballroom F 

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 six products are in the preparation phase for clinical trials over the next five year.

www.genethon.com 

Pratima Cherukuri, Ph.D., Chief Scientific Officer 

Oct. 9 | 11:00am | FLW Ballroom F 

Indianapolis, IN 

(Private) 

We are a contract development and manufacturing organization (CDMO) with unique technical expertise in lentiviral, retroviral, and adeno-associated viral vectors. Offering process development, GMP viral vector manufacturing, and analytics, we are dedicated to helping you deliver safe, pure, and potent advanced therapies to the patients who need them. 

www.genezen.com 

Carol Houts, Chief Strategy Officer 

Oct. 9 | 10:45am | FLW Ballroom G 

Ormond Beach, Florida   

(Private) 

Germfree Laboratories has been a leader in the life sciences industry for over six decades, providing innovative solutions that revolutionize pharmaceutical manufacturing and compounding. Our integrated approach combines state-of-the-art facilities, advanced equipment, and comprehensive services to meet the essential resources required for manufacturing processes. Our facilities are designed to the highest standards, enabling the production of life-saving advanced therapies and drugs. We specialize in providing turnkey solutions that include modular cleanrooms, mobile labs, and custom-built facilities, ensuring flexibility and scalability for our clients. Germfree’s philosophy centers on delivering integrated solutions that enhance operational efficiency, product quality, and patient outcomes. By leveraging our expertise and strategic partnerships, we develop cutting-edge manufacturing models that address critical industry challenges such as drug shortages and decentralized manufacturing. With a commitment to innovation, sustainability, and excellence, Germfree Laboratories continues to set the standard for healthcare manufacturing solutions worldwide. 

www.germfree.com 

Francisco LePort, Ph.D., Co-founder and CEO

Oct. 9 | 11:45am | FLW Ballroom G 

San Francisco, CA

(Private)

Gordian has scaled in vivo discovery of gene therapies, bringing gene therapy to complex disease. We inject a pooled library of hundreds of gene therapies, each targeting a different gene or combination, into an individual animal model of disease, turning the target tissue into a mosaic. Using single cell sequencing as phenotypic readout, we separately measure the in vivo efficacy of each gene therapy simultaneously for the indication. We have screened over 1000 gene therapies across three indications to date: Heart Failure, Metabolic Associated Steatohepatitis (MASH), and Osteoarthritis, building the largest in vivo gene perturbation dataset on the planet. Top hits have been validated in preclinical gene therapy POC experiments and will advance toward IND enabling studies and clinical development both internally and through pharma partnerships. For these collaborations, we solve target risk where a partner wants to leverage established gene therapy delivery technology for a complex indication.

www.gordian.bio

Matteo Piazza, Ph.D., Business Development and Commercial Director

Oct. 7 | 10:15am | FLW Ballroom F 

Modena, Italy

(Private)

Holostem s.r.l., founded in 2008, is Italy’s first biotechnology company dedicated to the development, manufacture, registration, and commercial distribution of Advanced Therapy Medicinal Products (ATMPs) based on epithelial stem cell cultures for both cell and gene therapy. These therapies target personalized medicine for patients with severe epithelial orphan diseases that have significant unmet needs.

www.holostem.com

Sumant Ramachandra, M.D., Ph.D., CEO and Board Director

Oct. 8 | 10:15am | FLW Ballroom F 

West Hills, CA

(Private)

ImmPACT Bio USA, Inc., is a clinical-stage company dedicated to the discovery of transformative chimeric antigen receptor (CAR) T-cell therapies that address key biological challenges in treating cancer and autoimmune diseases. The company’s logic-gate-based CAR T-cell platforms, licensed from University of California, Los Angeles (UCLA) Technology Development Group, are specifically designed to prevent antigen escape, overcome the immunosuppressive tumor microenvironment, and deplete pathogenic B cells. The company’s technology is based on the work of pioneering scientists Yvonne Chen, Ph.D., and Antoni Ribas, M.D., Ph.D., both from UCLA.

www.immpact-bio.com

Michelle Duquette, Ph.D., Chief Scientist

Oct. 8 | 4:00pm | FLW Ballroom G 

San Diego, CA

(NASDAQ: NTLA)

Invetech is your trusted partner in solving commercial manufacturing challenges for the global advanced therapies market. With over 20 years of experience and successful collaborations with leading cell therapy companies, we specialize in helping you transition from early process development to commercial-scale manufacturing. A collaborative approach is in our DNA and we work closely with clients to co-create solutions that address process demands. Leveraging our expertise in automation platforms and manufacturing systems, we offer Assured Pathways™ to integrated commercial manufacturing. Our comprehensive services include: Turnkey Modules: Ready-to-use solutions for specific manufacturing unit operations. Custom Technology Platforms: Tailored technology platforms to meet your unique requirements. Consulting Lab Services: Expert guidance and design support from our UX team. Configurable Systems: Adaptable solutions for seamless integration

www.invetechgroup.com

Jeff Zurwaski, Ph.D., Senior Manager Bioprocess Development, DiscGenics

Oct. 8 | 2:30pm | FLW Ballroom G 

Boston, MA

(Private)

InVitria, Inc. was founded in 2008 to revolutionize biomanufacturing through the development, manufacturing, and marketing of recombinant proteins. Our mission is to empower scientists and biomanufacturers by providing sustainable, scalable, and high-performing technologies for cell & gene therapies, vaccines, biological products, IVF, and medical devices. We offer plant-based proteins and growth factors that eliminate the need for blood and serum components that are traditionally used in the manufacturing and formulation of therapeutics. The current supply of human and animal components cannot scale to support the future demand of therapeutics. Regulatory bodies like the FDA and EMA discourage the use of blood-derived proteins and serum due to potential risks and batch-to-batch variation, making InVitria’s technologies indispensable for industries seeking safer and more efficient biomanufacturing processes.

www.invitria.com

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

Oct. 7 | 12:15pm | 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. 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

Curt Herberts, President and Chief Operating Officer

Oct. 9 | 11:30pm | FLW Ballroom G 

Palo Alto, CA

(Private)

Kriya is a biopharmaceutical company developing gene therapies to address common diseases affecting millions of people around the world. The company’s pipeline includes gene therapies for diseases such as diabetes, NASH, geographic atrophy, thyroid eye disease, epilepsy, and trigeminal neuralgia. Kriya’s platform integrates cutting edge infrastructure and technology across translational research, computational biology, and scalable manufacturing to accelerate the timeline and drive down the cost of designing, developing, and manufacturing transformational gene therapies.

www.kriyatherapeutics.com

David Venables, Ph.D., CEO

Oct. 7 | 12:00pm | FLW Ballroom G 

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’s truly innovative technology re-directs naturally-occurring micro RNAs (miRNAs) to conditionally silence genes in a way which is programmable, tunable, stable and specific. This opens a path to more effective, safer and accessible advanced therapies. Laverock is applying its technology through its own pipeline – targeting regenerative medicine and oncology – and through partnerships. Laverock has a highly experienced leadership team with proven track records in biotechnology, pharma and academia and an exceptionally strong Board. Laverock completed a £13.5m seed round in September 2023. Its high-calibre investors include Calculus Capital, Eli Lilly and Company, Mercia Ventures, Maven Capital Partners, Eos, UK Innovation & Science Seed Fund and Tekfen Ventures.

www.laverocktx.com 

Clare Murray, Ph.D., Senior Vice President, Corporate Development and Operations 

Oct. 9 | 11:00am | FLW Ballroom G 

Durham, NC 

(Private) 

Life Edit, ElevateBio’s next-generation gene editing business, offers 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 platform allows Life Edit to target any genomic sequence and potentially develop novel human therapeutics for the most challenging genetic diseases by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. In addition to developing its own pipeline of cell and gene therapies, Life Edit will continue to strengthen its platform of genome-editing enzymes, provide gene-editing expertise to strategic partners, and form other third-party partnerships to discover and develop new therapies. 

www.lifeeditinc.com 

Joe DePinto, Head of Cell Gene Advanced Therapies

Oct. 7 | 3:00pm | FLW Ballroom G 

Irving, TX

(NASDAQ: MCK)

McKesson Corporation is a diversified healthcare services leader dedicated to advancing health outcomes for patients. Our team partners with biopharma companies, care providers, pharmacies, manufacturers, governments, and others to deliver insights, products and services to help make quality care more accessible and affordable. Our Cell, Gene, and Advanced Therapies platform offers best-in-class capabilities to meet the unique needs associated with bringing these innovative products to market. Our solutions span the continuum of care innovation and delivery — from early clinical development, pre-launch planning, to commercialization — and provide an integrated approach toward addressing the pain points encountered by biopharma, providers, and payers. Informed by nearly a decade of experience within the space, this ecosystem of clinical, distribution, market access, technology, and data science capabilities offers an unmatched combination of expertise that has been designed to accelerate and enhance clinical and commercial outcomes for innovators operating within the market today.

www.mckesson.com 

Jian Irish, Ph.D., President, Chief Operating Officer, Executive Board Director

Oct. 8 | 9:30am | FLW Ballroom G 

Emeryville, California

(NASDAQ: MGX)

Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived toolbox. Metagenomi is harnessing the power of metagenomics, the study of genetic material recovered from the natural environment, to unlock four billion years of microbial evolution to discover and develop a suite of novel editing tools capable of correcting any type of genetic mutation found anywhere in the genome. Its comprehensive genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems (including prime editing systems and clustered regularly interspaced short palindromic repeat associated transposases). Metagenomi believes its diverse and modular toolbox positions the company to access the entire genome and select the optimal tool to unlock the full potential of genome editing for patients.

metagenomi.co

Lavakumar (Kumar) Karyampudi, Ph.D., Senior Director, Cell Therapies

Oct. 7 | 11:15am | FLW Ballroom G 

Tampa, Florida

(Private)

Moffitt Cell Therapy Facilities (CTFs) experienced team of 80+ professionals offer dynamic cell therapy manufacturing services including product development, optimization, and clinical product manufacturing to assist CTF partners in bringing their assets to clinical trials. Moffitt’s CTF staff and faculty work collaboratively with biopharmaceutical companies to translate cell therapy assets into clinic, and the CTF currently supports 19 INDs which includes biopharmaceutical partners developing cell therapies. Moffitt maintains a patient-centered focus and has extensive experience leading clinical trials in oncology. Recently, Moffitt played a pivotal role in helping bring the first tumor-infiltrating lymphocyte (TIL) product to market in melanoma with the FDA’s approval of Iovance’s Amtagvi. Apart from TIL, Moffitt’s CTF is involved in the development of a wide range of cell therapy modalities, including dendritic cell vaccines, CAR T and gamma delta T cell therapy, with programs in both solid tumors and hematological malignancies..

www.moffitt.org/cell-manufacturing

Robert Hofmeister, Ph.D., Chief Scientific Officer

Oct. 7 | 9:15am | FLW Ballroom G 

Cambridge, MA

(Private)

At Myeloid, we have applied our extensive knowledge of advanced gene and cell therapies to build novel platform technologies that harness the innate abilities of myeloid cells. We have translated the learnings from our backgrounds and novel platform to create a clinical-stage, mRNA-immunotherapy pipeline focused on breakthrough therapies for cancer.

www.myeloidtx.com

Sean Kevlahan, Ph.D., CEO and Co-founder

Oct. 7 | 11:45am | FLW Ballroom G 

Boston, MA

(Private)

Nanite is a non-viral gene delivery company developing programmable, tissue-specific polymer nanoparticles (PNPs) for a broad range of modalities and indications. Powered by our AI-driven platform, SAYERTM, Nanite is building the next generation of non-viral delivery vehicles – programmable, targeted, and safe – for a new wave of genetic medicines. SAYER applies state-of-the-art machine learning-methods to combine high-throughput experimental and computational polymer chemistry to design fit-for-purpose delivery vehicles.

www.nanitebio.com

Raphael Ognar, President and CEO, Co-founder

Oct. 7 | 4:30pm | FLW Ballroom G 

Houston, TX

(Private)

NKILT Therapeutics, Inc. is a new cell therapy biotech developing a novel approach of engineering Natural Killer (NK) cells, and other immune cells, to directly target leukemias and solid tumors. Our novel Chimeric ILT-Receptor (CIR™), featuring a unique proprietary binding technology, targeting the inducible immune checkpoint, HLA-G (expressed in over 50% of human cancers), will allow our CIR™NK cells to be able to exquisitely target and directly kill cancer cells but will also activate innate immunity and directly target the tumor’s defense mechanisms.

www.nkilt.com

Fredric Manfredsson, Ph.D., Chief Scientific Officer

Oct. 9 | 10:00am | FLW Ballroom F 

Phoenix, AZ

(Private)

Generation of novel AAVs for in-house use, or custom tailored for outside clients. Preclinical program investigating payloads for neurological disease.

www.nvector.com

Mansi Shinde, Head of Corporate Development and Strategy

Oct. 7 | 5:30pm | FLW Ballroom G 

Cambridge MA

(Private)

Designed to address the limitations of currently available delivery technologies, Nvelop’s modular platforms offer expanded cargo capacity, engineered tissue and cell specificity, enhanced delivery efficiency and safety advantages. Built on two validated in vivo delivery platforms, Nvelop’s customizable particles have the potential for selective and programmable targeting, high In vivo delivery efficiency of a wide range of cargo types and sizes, and full humanization. By enabling a modality-agnostic arsenal of genetic medicines, Nvelop’s platforms are uniquely positioned to expand the reach of precision medicines to many previously undruggable diseases.

www.nveloptx.com

Dan Kirby, Chief Commercial Officer

Oct. 7 | 5:00pm | FLW Ballroom F 

Menlo Park, CA

(Private)

Orca Bio is a late-stage cell therapy company engineering high-precision cell therapies for the treatment of blood cancer and autoimmune diseases. Our pipeline of T-cell immunotherapies are designed to replace patients’ diseased blood and immune system with healthy ones. Today, we’re focused on redefining the traditional stem cell transplant by developing cell therapies that provide significantly better outcomes with dramatically fewer toxicities, like GvHD, than the standard of care. Our lead investigational product, called Orca-T, is currently being evaluated in Precision-T, a pivotal Phase 3 clinical trial for the treatment of blood cancers and other hematological malignancies. We announced in June we had completed enrollment and anticipate sharing topline results in the first half of 2025. In parallel, we’re advancing our next-generation programs to expand our pipeline to bring cell therapy to all patients who could benefit, including potential future application in autoimmune disease and genetic disorders. Our mission is to make curative cell therapies both safer and more effective, and in doing so, push past the field’s current boundaries and redefine its future.

www.orcabio.com

Marc Lajoie, Ph.D., CEO

Oct. 8 | 9:00am | FLW Ballroom F

Seattle, WA

(Private)

OUTPACE is a Seattle based biotech using unrivaled AI-powered protein design to deliver on the promise of cell therapy by programming cells for improved function inside the patient. The company designs proteins that mechanistically solve the key barriers to efficacy in solid tumors (poor access to the tumor, weak survival, rapid inactivation, antigen escape, and high required doses drive toxicity) and encodes them into the cell therapy using standard manufacturing processes. Leveraging this powerful platform, in one year the company constructed its lead program, OPB-101, which robustly cures solid tumor xenograft models with two orders of magnitude better efficacy than other programs in the clinic, driven by an ability to expand and functionally persist in stringent solid tumor models. Based on its tumor-localized activity and low required cell dose, OPB-101 has the potential to offer an exceptional therapeutic index. Outpace is on track to submit its first IND in 2024.

www.outpacebio.com

Carter Cliff, CEO

Oct. 7 | 9:30am | FLW Ballroom F 

San Diego, CA

(Private)

Papillon Therapeutics is a clinical stage biotechnology company advancing multi-systemic genetic medicines for diseases with neurologic, cardiac, and other tissue pathologies. Degenerative diseases impact multiple tissues throughout the body, inspiring our unique approach to deliver therapeutic protein to diseased tissues via an innate system within the body. Gene modified hematopoietic stem and progenitor cell (HSPC) therapy is a commercially proven approach for disease modifying and potentially curative treatments in certain inherited disorders. Published studies by scientific founder, Dr. Cherqui, demonstrate multi-systemic engraftment and functional rescue utilizing our gene modified HSPCs for Friedreich’s ataxia, Danon disease, and Alzheimer’s disease. 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 being advanced by Novartis. Funding awarded by the California Institute of Regenerative Medicine (CIRM) and NIH to our scientific founders totals over $30 million towards our Cystinosis, Friedreich’s ataxia, and Danon disease programs.

www.papillon-tx.com

Linda Tempelman, Ph.D., CEO

Oct. 9 | 9:00am | FLW Ballroom F 

Ithaca, NY

(Private)

Persista Bio® enables the promise of cellular biotherapeutics with long-term implants that mimic the body’s processes. The O2Line™ technology is an oxygenated, macroencapsulation system for delivering and maintaining allogeneic therapeutic cell implants. Two features of the system include a unique anti-fibrotic cell capsule and an in-situ oxygen generator to support the membrane encapsulated cells. The product will be fully implantable with no need for immunosuppression. Initial preclinical data is in the treatment of Type 1 diabetes. The O2Line™ system will enable the major advancements in stem cell biology to become long-term, curative cell therapy treatments for chronic diseases including metabolic diseases. Persista Bio is a seed stage company located in Ithaca NY. Persista Bio’s co-founders are inventors on a series of key technologies licensed from Cornell University and Giner, Inc. Two co-founders, Dr. Linda Tempelman and Dr. Minglin Ma, are experienced entrepreneurs.

www.persistabio.com

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

Oct. 8 | 4:30pm | FLW Ballroom F 

Gdansk Poland

(NASDAQ: PTG)

PolTREG is an advanced clinical stage company based in Poland developing engineered and polyclonal Tregulatory cell therapies for Autoimmune disease. Our lead Treg asset, PTG-007, has been evaluated in over 101 patients, in 4 clinical studies, across three indications. PTG-007 is ready to enter pivotal studies in T1D. We are also developing next-generation antigen specific, CAR, and TCR-Tregs including Allogenic Treg cell therapies. PolTREG has also built a state of the art cGMP cell therapy manufacturing facility in Gdansk to supply clinical to commercial scale production of Tregs. While fully allogeneic therapies are 10 years away, PolTREG will commercialize polyclonal Tregs in the near term while working towards the same goal of off-the-shelf Tregulatory cell therapies.

www.poltreg.com

Kristin Yarema, Ph.D., President and CEO

Oct. 8 | 10:00am | FLW Ballroom F 

San Diego, CA

(NASDAQ: PSTX)

Poseida Therapeutics is a clinical-stage biopharmaceutical company advancing differentiated allogeneic cell therapies and genetic medicines with the capacity to cure certain cancers and rare diseases. The Company’s pipeline includes investigational allogeneic CAR-T cell therapies for both solid tumors and hematologic cancers as well as investigational in vivo genetic medicines that address patient populations with high unmet medical need. The Company’s approach is based on its proprietary genetic editing platforms, including its non-viral piggyBac® DNA Delivery System, Cas-CLOVER™ Site-Specific Gene Editing System, Booster Molecule and nanoparticle gene delivery technologies, as well as in-house GMP cell therapy manufacturing. The Company has formed strategic collaborations with Roche and Astellas to unlock the promise of cell therapies for cancer patients.

www.poseida.com

Helen Sabzevari, Ph.D., President and CEO

Oct. 8 | 11:00am | FLW Ballroom F 

Germantown, MD
(NASDAQ: PGEN)

Precigen is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated therapies toward clinical proof-of-concept and commercialization.

www.precigen.com

Mohammed Asmal, M.D., Ph.D., SVP, Head of Clinical

Oct. 7 | 11:00am | FLW Ballroom F 

Cambridge, MA

(NASDAQ: PRME)

Prime Medicine is a leading biotechnology company dedicated to creating and delivering the next generation of gene editing therapies to patients. The Company is deploying its proprietary Prime Editing platform, a versatile, precise and efficient gene editing technology, to develop a new class of differentiated one-time curative genetic therapies. Designed to make only the right edit at the right position within a gene while minimizing unwanted DNA modifications, Prime Editors have the potential to repair almost all types of genetic mutations and work in many different tissues, organs and cell types. Taken together, Prime Editing’s versatile gene editing capabilities could unlock opportunities across thousands of potential indications. Prime Medicine is currently progressing a diversified portfolio of investigational therapeutic programs organized around core areas of focus: hematology and immunology, liver, lung, ocular and neuromuscular.

www.primemedicine.com

Josefina, Nilsson. Ph.D., CEO

Oct. 8 | 5:30am | FLW Ballroom F

Stockholm, Sweden

(Private)

QuTEM offers GMP-certified TEM services for nanoparticle analysis. In this presentation, I will showcase our service portfolio, emphasizing key vectors for gene therapy and drug delivery systems. I will highlight QuTEM’s recently patented method for AAV.

www.qutem.com

Peter Francis, M.D., Ph.D., Chief Scientific Officer, Chief Medical Officer

Oct. 7 | 1:45pm | FLW Ballroom G 

San Francisco, CA

(Private)

Ray Therapeutics is a late-stage preclinical biotechnology company pioneering optogenetic gene therapies for blinding retinal diseases. Founded in 2021 and based in San Francisco, the company raised $100 million in a Series A financing led by Novo Holdings in May 2023. Ray’s lead candidate RTx-015, an AAV gene therapy delivering a light-sensitive protein, is expected to enter clinical trials soon for retinitis pigmentosa. The company is also developing RTx-015 for other inherited retinal degenerations and RTx-021 for Stargardt macular dystrophy and geographic atrophy. With a strong syndicate backing and promising preclinical data, Ray aims to provide potentially first-in-class, mutation-agnostic vision restoration therapies for patients with few treatment options.

www.raytherapeutics.com 

Barbara Ressler, Ph.D., Vice President, Manufacturing Process Sciences

Oct. 7 | 4:45pm | FLW Ballroom G 

Hopkinton MA, Edinburgh, Scotland

(Private)

RoslinCT is a leading global contract development and manufacturing services organization (CDMO) focused on Advanced Cell and Gene Therapies. Established in 2006 and built upon the ground-breaking technology cloning of Dolly the Sheep at the Roslin Institute in 1997, RoslinCT has harnessed cutting-edge science to advance the development of human medicines. With a remarkable heritage in the field, the company has achieved significant milestones. These include being among one of the first in the world to produce clinical-grade human pluripotent stem cells. In collaboration with the partners, RoslinCT also developed the first CRISPR-edited cell therapy product for a major inherited disorder, taking it from early development to commercialization. Equipped with 22 purpose-built cGMP cell therapy processing suites in Edinburgh, Scotland, and Hopkinton, Massachusetts, RoslinCT provides innovative process and analytical development, cGMP clinical and commercial manufacturing for a range of cell types for both autologous and allogeneic processes, and cGMP iPSC cell line development, gene editing, and differentiation. With tailored CDMO solutions, RoslinCT enables partners to efficiently progress from development to commercialization and deliver life-saving Cell and Gene Therapies worldwide. RoslinCT is a GHO Capital portfolio company.

www.roslinct.com

Saeil (Tony) Kang, President and CEO

Oct. 8 | 4:45pm | FLW Ballroom F 

Seoul, Korea

(KOSDAQ: 304360) 

S.Biomedics is one of South Korea’s leading cell medicine corporation focusing on regenerative medicine. Through two robust platform technologies, 7 cell therapy programs are under development with 4 programs under clinical stage. One notable success is the development of TED-A9, a promising treatment for Parkinson’s Disease by utilizing hESC derived ventral-midbrain specific dopamine precursors. Promising results in the in-vitro and in-vivo studies were published on “Cell Stem Cell” on December 2023. TED-A9 is currently in Phase 1/2a in Korea for 12 patients, and all 12 patients were administered. Mid-term data of the study is expected to be released this year. Furthermore, TED-N (hESC derived NPC for SCI Phase 1/2a) and FECS-Ad (MSC spheroid for CLI Phase 1/2a) are under development.

www.sbiomedics.com/eng/

Tom Lowery, Ph.D., President and Chief Technology Officer

Oct. 7 | 1:30pm | FLW Ballroom G 

Watertown, MA

(Private)

Satellite is harnessing the therapeutic potential of hepatocytes to tackle advanced liver disease.

A shortage of organs coupled with high complexity of surgery makes transplantation unrealistic for >85% of those in need. Cell therapies have promise, but widespread use of primary hepatocytes is severely limited by availability, and stem cell-derived hepatocytes do not have the appropriate level of functionality. As pioneers of regenerative medicine, we are the first company to develop protocols to effectively isolate, expand and mature hepatocytes, generating fully functional cells. Akin to a liver transplant via infusion, our powerful and precisely organized cells are delivered into the body where they engraft and vascularize, acting like a mini “satellite” organ that boosts or restores liver functions. By creating a sustainable cell source, we can shatter the 1:1 paradigm of liver transplants. With Tissue Therapeutics, we could benefit a thousand patients from a single organ – an enormous opportunity for the hundreds of thousands of patients in need.

www.satellite.bio

Benjamin Oakes, Ph.D., CEO

Oct. 9 | 10:30am | FLW Ballroom F 

Alameda, CA

(Private)

Scribe Therapeutics is a genetic medicines company that is revolutionizing the development of in vivo genetic medicines to transform human health. The company employs the proprietary CRISPR by DesignTM approach to engineer bacterial immune systems into a premier suite of genome and epigenome editing tools built for unique advantages in activity, specificity, and deliverability that translate into safer and more effective therapies. Founded by renowned experts in genetic medicine, including CEO Benjamin Oakes and Nobel Laureate and CRISPR co-inventor Jennifer Doudna, Scribe is developing a leading portfolio of optimized in vivo genetic medicines to treat a spectrum of cardiometabolic diseases. $140M generated upfront from recently entered strategic partnerships with Sanofi and Lilly. Raised $100M Series B financing in 2021 led by Avoro, OrbiMed and Andreessen Horowitz, among other premier investors.

www.scribetx.com

Jodi Cook, Ph.D., CEO and President

Oct. 9 | 11:15am | FLW Ballroom F 

Cambridge, MA

(Private)

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

skylarkbio.com

Nina Bauer, Ph.D., Chief Business Officer

Oct.8 | 5:00pm | FLW Ballroom F 

Tullinge, Sweden

(Private)

SmartCella, founded in 2014, is an innovative biotechnology company based in Stockholm, Sweden. SmartCella’s vision is to combine first-in-class delivery platforms with cutting-edge cell and mRNA therapies to unleash the full potential of targeted therapies. The company has three main business units, Smartwise, SmartCella Solutions and ProCella. ProCella is a therapeutic company developing proprietary stem cell therapies. The first therapy, a cardiac progenitor cell, has now been taken through GLP tox and is approaching a clinical phase 1 trial in heart failure. The company recently completed the construction of its clinical GMP manufacturing facility, which has been approved by the Swedish authorities. This infrastructure along with the team’s technical expertise and capabilities will underpin the company’s growing pipeline going forward. Smartwise has developed a first-in-class endovascular device, the Extroducer, that enables direct-to-tissue drug delivery. This technology addresses a significant unmet need in the field novel therapies, enabling targeted delivery of a wide range of modalities for solid tumor treatment and tissue repair. Using standard equipment and routine interventional radiology approaches, the Extroducer provides access to hard-to-reach tissues by safely penetrating the vessel wall and delivering payload directly to the target location. Smartwise received regulatory clearance from the US FDA for the Extroducer and is now actively licensing out the technology globally. SmartCella Solutions has developed novel mRNA therapies and cell-mediated delivery platforms enabling localized protein expression. The company is now actively engaging in partnering discussions for out-licensing, as well as developing its own proprietary therapies.

www.smartcella.com

Geoff Hamilton, Co-founder and CEO

Oct. 7 | 9:15am | FLW Ballroom F 

San Diego, CA

(Private)

Stemson Therapeutics is a clinical stage company whose mission is to redefine hair loss treatments by developing breakthrough cell therapies capable of safely restoring hair follicles in patients suffering from hair loss, including medically challenging hair loss conditions such as Alopecia Areata, Scarring Alopecia, and Chemo-Induced Alopecia. Today there are no available treatments capable of stopping hair loss or regenerating hair after it has been loss. Stemson’s talented team of cell therapy developers, tissue engineers, and seasoned biotech executives aims to change this and provide the effective solutions needed for these patients who suffer the stigmas and mental health effects of hair loss.

www.stemsontx.com

Michael Luther, Ph.D., Chief Executive Officer and Board of Directors

Oct. 7 | 3:15pm | FLW Ballroom G 

Cambridge, MA

(Private)

STRM.BIO is developing megakaryocyte-derived extracellular vesicles (MVs) to deliver gene therapies in vivo. Our platform is differentiated by: (1) a striking tropism to bone marrow and HSPCs while bypassing the liver; (2) tunable loading that supports nucleic acid and protein cargo delivery in vivo; and (3) ability to repeat dose. The company’s lead programs are targeting rare blood diseases. We recently completed mouse in vivo protein expression studies in several reporter systems. In mice, reporter protein is expressed preferentially in bone marrow cells following in vivo delivery of nucleic acid by MVs; no cargo-derived expression was observed in the liver. In prior NHP repeat dosing/tolerability and biodistribution studies, pDNA-loaded MVs preferentially delivered cargo to bone marrow. Cargo loading did not alter biodistribution profile in either animal model. Mouse safety studies and the NHP tolerability & repeat dosing study confirms unloaded EVs are non-toxic and amenable to repeat dosing.

www.strm.bio

Vikram Ranade, Ph.D., Senior Vice President, Corporate Development

Oct. 9 | 9:45am | FLW Ballroom G 

Boston, Massachusetts

Tessera Therapeutics, Inc. is pioneering Gene Writing™—a novel genome editing technology that writes therapeutic messages into the genome to treat rare diseases and cancer. Our RNA Gene Writers can make permanent small and large alterations to the human genome using non-viral delivery of RNA molecules. We recently presented data at ASGCT that demonstrated our RNA Gene Writers are 1) highly efficient at in vivo correction of the mutation responsible for alpha-1 antitrypsin deficiency (AATD) to wild-type, 2) able to achieve therapeutically relevant levels of hemoglobin beta (HBB) rewriting in vivo in humanized mice when combined with our proprietary lipid nanoparticle (LNP) delivery – we believe these results can enable true correction to wild-type of the HBB locus responsible for sickle cell disease (SCD) and 3) can integrate CAR transgenes into T cells in vitro creating functional CAR-T cells that show tumor killing in vitro and in vivo.

www.tesseratherapeutics.com

Frédéric Desdouits, Chief Executive Officer, TreeFrog Therapeutics

Oct. 7 | 11:30am | FLW Ballroom G 

Pessac, France

(Private)

TreeFrog Therapeutics is a French-based biotech company set to unlock access to cell therapies for millions of patients. TreeFrog is unique in its approach to cell therapy development bringing 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. To realize their mission of Cell Therapy for all, TreeFrog operates a hybrid business model that includes its own therapeutic programs and partnerships with leading biotech and industry players in other areas. In the last 3 years, the company has raised $82 million to advance a pipeline of stem cell-based therapies in regenerative medicine with a lead program in Parkinson’s disease.

www.treefrog.fr

Philip Mathuis, Co-Founder and CEO 

Oct. 9 | 9:45am | FLW Ballroom F 

Gent, Belgium 

(Private) 

Trince, a Belgian company based in Ghent, devlops the LumiPore platform, a revolutionary intracellular delivery technology facilitating the transport of functional compounds into cells. With a focus on genetic engineering, Trince targets immune cells for cancer therapy, stem cells like iPSCs, and live cell imaging. Their technology enables precise genetic modifications while preserving cell function and therapeutic potential. Using pulsed laser light and photothermal nanoparticles, Trince achieves localized forces to create temporary pores in cell membranes, termed “photoporation.” This approach allows efficient, flexible, and safe delivery of molecules into various cell types. Particularly suitable for R&D, the technology involves adding nano sensitizers to cell culture and laser irradiation. For clinical use, Trince embeds nano sensitizers into polymer nanofibers or recommends biodegradable options. Recent studies demonstrate its efficacy in CAR T and iPSC cell therapy. Trince continues to advance genetic engineering research for therapeutic applications. 

www.trincebio.com 

David Fontana, Ph.D., Chief Operating Officer and Chief Business Officer

Oct. 8 | 1:15pm | FLW Ballroom G 

Seattle, WA

(Private)

Umoja is an in vivo gene delivery immunotherapy company creating off-the-shelf treatments that aim to extend the reach and effectiveness of CAR T cell therapies in oncology and autoimmunity.

www.Umoja-biopharma.com 

Tom Smith, Strategic Initiatives Director

Oct. 8 | 5:00pm | FLW Ballroom G 

Dublin, Ireland

(Private)

Uniphar is a trusted global partner to pharma, working to improve global patient access to medicines. Over the last 5 years, we have built unparalleled experience supporting our partners to accelerate patient access to cell and gene therapies around the globe.Uniphar’s CGT experts harness the capabilities, infrastructure and experience of a diversified pharmaceutical service provider with more than 55 years’ building success stories with 200+ multinational clients.Our understanding of the complex CGT requirements, market dynamics and barriers to access have allowed us to develop a fit-for-purpose CGT access platform which has delivered tailored solutions allowing more than 500 patients to access CGT treatments around the world. Uniphar Pharma comprises Uniphar | Access, Uniphar | Medical, and Uniphar | Commercial – together our combined capabilities make us your trusted partner to unlock access to innovative therapies in countries where you might think it unachievable.

www.uniphar.com/access

Jacques Galipeau, M.D., Associate Dean for Therapeutics Development

Oct. 7 | 9:45am | FLW Ballroom G 

Madison, WI

(Private)

We specialize in conducting first-in-human clinical trials to de-risk advanced cell therapy leveraging UW Health expertise in clinical domains of excellence. We empower our academic and industry partners to translate scientific discoveries to cell therapies through FDA CBER investigational new drug (IND) application-enabling and Phase I/II clinical studies. We have expertise in utilization and optimization of partially and fully automated closed systems for advanced cell manufacturing and enrichment. We maintain and continuously improve our operating procedures and quality system to meet cell therapy-specific FDA CBER guidance for Phase I/II clinical trials.

www.pact.wisc.edu