Laurent Fischer, M.D., CEO

Redwood City. CA

(NASDAQ: ADVM)

Adverum Biotechnologies, Inc. is pioneering the use of gene therapy as a new standard of care for highly prevalent ocular diseases. Adverum’s lead program, Ixo-vec, is designed to be administered as a one-time IVT injection in the physician’s office, deliver long-term efficacy, reduce the burden of frequent anti-VEGF, optimize patient compliance, and improve vision outcomes for patients with wet AMD. At the 2025 Cell and Gene Meeting on the Med, we intend to present data from the ongoing LUNA Phase 2 trial and the OPTIC extension trial of Ixo-vec in wet AMD showing that Ixo-vec preserved vision and controlled CST while significantly reducing treatment burden in hard-to-treat patients with history of frequent anti-VEGF injections before receiving Ixo-vec, including a recently approved agent, and is preferred by patients as compared to the standard of care. This is the first time for Adverum to potentially participate at the Cell and Gene Meeting on the Med the intent is to help inform the community about Adverum’s clinical program results from the Ixo-vec program, the manageable safety profile for gene therapy in clinical trials, and to network with potential partners.

www.adverum.com

Harald Stover, Ph.D., Founder and CEO, Allarta Life Science Inc., Professor of Chemistry and Chemical Biology, McMaster University

Hamilton, Canada

(Private)

Allarta Life Science is a Canadian company, founded in 2019 by McMaster’s Chemistry Professor Dr. Harald Stover and Maria Antonakos. Allarta’s unique platform of synthetic hydrogel technology is based on 30+ years of foundational expertise and its modular design has the potential to advance next generation cell therapies. Allarta’s hydrogels allow transplanted cells to connect metabolically while protecting them from the hosts’ 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 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, Ph.D., Chief Development Officer

Stockholm, Sweden

(Private)

Anocca, a Swedish biotechnology company, 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. Based on programmable human cells, its proprietary technology platform recreates T-cell immunity and precisely maps HLA-peptide T-cell targets on cancer cells and identifies specific and potent TCRs that recognize those targets. Anocca uses its platform to generate TCR asset libraries that are matched to a patient’s tumor profile and deployed in personalized TCR-T cell therapies. Anocca’s pipeline comprises over 40 TCR assets that target driver mutations, dysregulated tumor-specific genes, and onco-viral antigens. Using a non-viral gene-editing approach, Anocca’s integrated in-house process development and cGMP manufacturing capabilities enable the clinical development of its TCR-T cell therapy products. VIDAR-1, a multi-asset TCR-T program in advanced pancreatic cancer with KRAS mutations, is the company’s first clinical program and, pending approval, is about to start.

www.anocca.com

Devyn Smith, Ph.D., CEO

Cambridge, MA

(Private)

At Arbor, we are discovering, developing and making available to all patients the next generation of genetic medicines. We are disrupting the field through our proprietary discovery engine to develop a portfolio of differentiated genetic editing and delivery capabilities. We have an unencumbered portfolio of multiple CRISPR based genetic modifiers that can be tailored to correct the underlying pathology of each genetic disease. Our pipeline of genetic medicines will focus on bringing cures to all patients with genetic disease.

www.arbor.bio

Daniel Rosan, Chief Business and Financial Officer

Boston, MA

(Private)

Ascidian’s first-of-its kind RNA exon editing platform is designed to expand the therapeutic possibilities of RNA medicine and treat diseases not addressed by today’s gene editing technologies. By rewriting RNA, Ascidian’s exon editing technology provides the durability of gene therapy and the benefits of gene editing, but without the risks of direct DNA editing, without using foreign proteins of any kind. Disease-causing genetic mutations are often located at different sites on the gene in different patients. Ascidian’s technological platform can replace multiple contiguous exons — not just single bases — providing a powerful, versatile RNA therapeutic approach. Ascidian is a 2024 Fierce 15 and Endpoints 11 winner.

www.ascidian-tx.com

Tamer Mohamed, Founder and CEO

Vancouver, 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

Richard Wilson, Senior Vice President, Primary Focus Lead, Genetic Regulation

Tokyo, Japan

(OTCMKTS: ALPMY)

Astellas is a global life sciences company committed to turning innovative science into VALUE for patients. Through our research and development programs, we are pioneering new healthcare solutions for diseases with high unmet medical need. This includes our focus on delivering breakthrough gene therapies with life-changing potential for patients across many disease areas. We are building one of the world’s most robust pipelines of gene therapies for genetic disorders and an end-to-end gene therapy infrastructure with industry-leading discovery, development, manufacturing and commercial capabilities. 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. 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

Evry, 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. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD), with two clinical-stage programs targeting respectively LGMD-R9 and LGMD-R5. Initial positive clinical results with ATA-100 gene therapy in LGMD-R9 were presented in October 2024. Additional programs target LGMD-R1 and cardiomyopathies.

www.atamyo.com

Christopher Vann, Chief Operating Officer

London, United Kingdom

(NASDAQ GS: AUTL)

Autolus Therapeutics is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer and autoimmune disease. Using a broad suite of proprietary and modular T cell programming technologies, Autolus is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize target cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies, solid tumors and autoimmune diseases.

www.autolus.com

Graeme Fielder, Ph.D., Chief Operating Officer

London, United Kingdom

(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

Jason Dowd, Ph.D., Global Lead Cell Therapy Platform Process

Whippany, NJ

(OTCMKTS: BAYRY)

At Bayer, we are committed to advancing the industrialization of cell therapy manufacturing through innovative platform technologies, embodying our mission of “health for all, hunger for none.” By harnessing cutting-edge advancements in bioprocessing, automation, and data analytics, we aim to streamline the production of cell therapies, ensuring scalability and accessibility for patients worldwide. Our approach not only enhances the efficiency and consistency of manufacturing processes but also aligns with our vision of delivering transformative therapies that address unmet medical needs. At the Alliance of Regenerative Medicine Meeting on the Med in 2025, we will showcase our pioneering efforts in establishing robust, sustainable manufacturing solutions that empower healthcare systems and improve patient outcomes. Together, we can redefine the future of regenerative medicine and contribute to a healthier, more equitable world.

www.bayer.com

Kathryn Penkus Corzo, President and Chief Operating Officer

Cambridge, UK

(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 new possibilities in research and drug discovery.

www.bit.bio

Steven Nichtberger, M.D., CEO

Philadelphia, PA

(NASDAQ: CABA)

Cabaletta Bio is a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases. The CABA™ platform encompasses two complementary strategies which aim to advance the discovery and development of engineered T cell therapies with the potential to become deep and durable, perhaps curative, treatments for a broad range of autoimmune diseases. The lead CARTA (Chimeric Antigen Receptor T cells for Autoimmunity) strategy is prioritizing the development of CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy. CABA-201 is currently being evaluated in the RESET™ (REstoring SElf-Tolerance) clinical development program spanning multiple therapeutic areas, including rheumatology, neurology and dermatology.

www.cabalettabio.com

Tom Wilton, Chief Business Officer

Philadelphia, PA

(NASDAQ: CARM)

Carisma Therapeutics Inc. is a clinical stage biopharmaceutical company focused on utilizing our proprietary macrophage and monocyte cell engineering platform to develop transformative immunotherapies to treat cancer and other serious diseases. We have created a comprehensive, differentiated proprietary cell therapy platform focused on engineered macrophages and monocytes, cells that play a crucial role in both the innate and adaptive immune response. Carisma is headquartered in Philadelphia, PA.

www.carismatx.com

Rodolphe Clerval, CEO

Paris, France

(Private)

Coave Therapeutics is a genetic medicine company pioneering the development of innovative solutions to enhance the precision, safety, efficacy and manufacturability of genetic medicines. With its proprietary ALIGATER™ platform, Coave is at the forefront of addressing challenges in gene therapy delivery to extra-hepatic tissues, creating a robust pipeline targeting CNS, neuromuscular and eye diseases.

www.coavetx.com

Timothy Schroeder, Chairman and CEO

Covington, KY

(Private)

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

www.ctifacts.com

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

Paris, France

(Private)

EG 427 is developing DNA medicines based on non-replicative HSV-1 vectors for prevalent indications.

EG110A is our lead program currently in Ph1b/2a trial in neurogenic detrusor overactivity (NDO) delivering safety and efficacy read-out in this high-value indication(s) in early ’26. Our HERpes-based Modular Expression System (HERMES) platform is the foundation for a pipeline in multiple highly prevalent indications. nrHSV-1 vectors enable precision delivery of DNA medicines to specific groups of cells, have a strong track-record of success and safety in the clinic, can be re-administered, and have a cost per dose consistent with treatment of common diseases. The HERMES platform provides stable nrHSV-1 vectors to safety deliver large and complex DNA medicines to predefined specific targets with superior control and safety. We have early-stage programs in pain, neurodegenerative disease, neuromuscular disease, and oncology.

www.eg427.com

Jonathan Garen, Chief Corporate Development Officer

Boston, MA

(Private)

Ensoma is a in vivo HSC (hemopoietic stem cell) engineering company. We have programs in rare disease, sickle cell disease and solid tumor immuno-oncology. Our lead program is expected to enter the clinic by mid-2025. Our platform is unique able to safely insert large genes and edit genes in a single product, enable lineage cell specific expression and multiplexing, and enables control functions to accurately titrate up and down levels of modified HSC cells. Ensoma has a base editor that allows efficiently edits without any DNA cuts.

www.ensoma.com

Alireza Shahin, Ph.D., CEO

Ontario, Canada

(Private)

Evolved.Bio is a pre-clinical stage tissue therapeutics company creating the most in vivo-like tissues in a completely scaffold/biomaterial-free way using its proprietary Anchored Cell Sheet Engineering platform. Evolved’s first product is focused on skeletal muscle regeneration in the case of traumatic injury, but partnerships with multiple commercial groups are expanding the pipeline to include hepatic, pancreatic, and cardiac tissues. Scaffold-based solutions have limited efficacy because the exogenous biomaterials do not properly integrate with host tissues, interfere with natural cellular processes, have significant variability, and cause severe immune responses, all impeding regeneration. Evolved solves these issues by creating tissues composed of only properly differentiated cells and the mature, tissue-specific ECM that they produce. This enables Evolved’s tissues to properly engraft with the host tissue and regenerate the target. Evolved’s ultimate goal is to heal every damaged tissue.

www.evolved.bio

Christine Guenther, M.D., Ph.D., Executive Vice President, Head of Global Cell Therapy Translation, Managing Director Evotec Modena

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

Per Lundin, Ph.D., CEO and Co-Founder

Oxford, UK

(Private)

Evox Therapeutics is a privately owned biotechnology company headquartered in Oxford, UK. We specialize in developing exosome-based therapeutics for severe, life-threatening rare diseases. Our proprietary ExoEdit™ platform allows us to engineer exosomes—natural extracellular vesicles—to deliver genome editing tools to targeted cells and tissues, addressing challenges associated with traditional therapies. We are advancing a pipeline aimed at treating CNS conditions such as amyotrophic lateral sclerosis and spinocerebellar ataxia type 2. We have a track record of impactful partnerships, including with Eli Lilly and Takeda, to develop exosome-based therapies for neurological disorders, showcasing the therapeutic potential of our platform. Over the years, we have attracted substantial investment, including a $95 million Series C funding round in 2021, to support the development of our innovative therapies.

www.evoxtherapeutics.com

Brianna Barrett, Ph.D., Director of Technical Sales and Scientific Advisory

Grove City, OH

(Private)

Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company, 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

Karen Nicole Pignet Aiach, CEO

Milano, Italy

(Private)

Genespire is a biotechnology company, developing gene therapies based on immune shielded lentiviral vectors (ISLVs) for pediatric patients affected by genetic diseases. ISLVs are designed to be used intravenously and allow the life-long production of the therapy directly from the patient’s liver. Genespire is initially advancing therapeutic programs in inherited metabolic diseases with high unmet medical need. Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini and Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. Genespire is a spin-out of SR-Tiget, a world leading cell and gene therapy research institute.

www.genespire.com

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

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

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

Paris, France

(Private)

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

www.kiji-tx.com

David Venables, Ph.D., CEO

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 of choice in a way which is programmable, tunable, stable and specific. This opens a path to more effective, safer and accessible advanced therapies. Laverock’s platform exemplification encompasses ex vivo cell therapy including:

• iPSC derived macrophages engineered to be resistant to the tumour-associated macrophage phenotype and to exclusively polarise to a pro-inflammatory state in the tumour microenvironment.

• CAR-T cells for hard-to-treat cancers where multiple check-point pathways can be conditionally silenced providing improved efficacy without negative affects on effector cell fitness.

• iPSC derived hypoimmune pancreatic islets to treat type 1 diabetes.

www.laverocktx.com

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

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

Jeff Liter, Founder and CEO

Minneapolis, MN

(Private)

Luminary Therapeutics is revolutionizing allogeneic CAR-T therapy by pioneering a triple receptor CAR design aimed at surpassing the efficacy of conventional single receptor CD19 treatments for autoimmune diseases. Additionally, our innovative split co-stimulatory CAR design is poised to propel the industry forward in addressing solid tumors. Currently, Luminary is conducting three Phase I First-in-Human trials utilizing an autologous format to establish safety and detect early indications of efficacy. Encouragingly, our second treated patient has already achieved a complete response (CR) without experiencing cytokine release syndrome (CRS). Our Autoimmune trial is expected to begin in early 2025. As we embark on the early stages of a Series A fundraising round, Luminary is committed to advancing our assets through the clinical trial pipeline, bringing hope and transformative therapies to patients in need.

www.luminarytx.com

TBD

Rockville, MD

(NASDAQ: MXCT)

MaxCyte is a leading cell-engineering company providing enabling platform technologies to advance the discovery, development and commercialization of next-generation cell therapeutics and to support innovative, cell-based research. MaxCyte builds best-in-class technology designed to facilitate complex engineering of a wide variety of cells and to drive the rapidly expanding cell therapy market forward. For over 20 years, MaxCyte has been a leader in developing transfection workflows and ventures beyond today’s processes to innovate tomorrow’s solutions.

www.maxcyte.com

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

Boston, MA

(Private)

Nanite, Inc. 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. Nanite is headquartered in Boston, MA.

www.nanitebio.com

Eric Halioua, Ph.D., CEO

Belgium

(Private)

PDC*line Pharma is a Belgian-French biotech company that is developing a novel class of off-the-shelf cancer immunotherapies based on a proprietary Plasmacytoid Dendritic Cell line (PDC*line) pre-loaded with peptides that are derived from target tumor antigens. Based on a robust preclinical package and a first-in-human phase Ib feasibility study in melanoma, PDC*line Pharma is focusing on lung cancer with a new candidate in phase I/II clinical trial (PDC*lung) and neoantigens (PDC*Neo). PDC*line Pharma comprises a team of 42 highly skilled professionals based in Liège (Belgium) and Grenoble (France) and has raised more than €61M in equity and non-dilutive funding.

www.pdc-line-pharma.com

Helen Sabzevari, Ph.D., President and CEO

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

Brian Freed, Ph.D., Chief Science Officer

Aurora, Colorado

(Private)

RheumaGen is an engineering cure for rheumatoid arthritis (RA), multiple sclerosis (MS), type 1 diabetes (T1D), and other major autoimmune diseases with HLA gene editing. Our approach builds upon clinically proven methods to collect and edit hematopoietic stem cells, which allow us to safely transform harmful HLA alleles into healthy ones. RheumaGen has received excellent initial FDA feedback and is preparing an IND submission to begin Phase I with refractory RA patients in Q1 2026.

www.rheumagen.com

Kinnari Patel, President, Head of R&D and Chief Operating Officer

Cranbury, N.J.

(NASDAQ: RCKT)

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

www.rocketpharma.com

Ricardo Baptista, Ph.D., Chief Technical Officer

Tullinge, Sweden

(Private)

SmartCella is a world-leading biotech company pioneering the future of targeted therapies through delivery solutions and advanced therapy development. Founded in 2014, the company is built on globally renowned science and research from Karolinska Institutet in Sweden. SmartCella combines novel delivery platforms, such as the Extroducer® (an endovascular delivery device that enables direct injection to hard-to-reach organs and tumors), with cutting-edge development and manufacturing of cell-mediated therapies. The company operates in two business segments: Targeted Deliveries and Cell-Mediated Therapies. The international team consists of scientists, visionary innovators, and experienced business leaders, all dedicated to shaping the future of targeted therapies and delivering life-changing treatments to patients.

www.smartcella.com

Dagmara Szkolnicka, Ph.D., Chief Scientific Officer

Copenhagen, Denmark

(Private)

The company is developing a scalable and effective treatment for liver diseases using liver tissue implants developed from pluripotent stem cells (PSCs). Stimuliver has developed revolutionary proof-of-concept data which demonstrates that human liver tissue can be administered under the skin, providing long-term host liver support. For chronic liver disease patients, there are benefits of implanting cells subcutaneously, as the liver is highly inflamed making it a hostile environment for cell-based therapies. By utilizing PSCs as a source of tissue, the technology Stimuliver has developed is not dependent on donor organ availability. This may in the future provide a treatment which is available without waiting time and has the potential to reverse liver disease and prevent prolonged or recurrent hospitalizations. The research is based on technology developed at the University of Edinburgh’s Centre of Regenerative Medicine, is incubated at the BioInnovation Institute and was funded by the Novo Nordisk Foundation.

www.stimuliver.bio

Frederic Desdouits, Ph.D., CEO

Pessac, France

(Private)

TreeFrog Therapeutics is a regenerative medicine biotech 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 business model that includes its own therapeutic programs and partnerships with leading biotech and industry players based on their proprietary platform technology, C-Stem™. In the last 3 years, the company has raised $82 million to advance a pipeline of stem cell-based therapies in regenerative medicine. Headquartered in Pessac, France, the company has a technological hub in Boston, USA to drive the adoption of the platform and establish strategic alliances with leading academic, biotech and industry players.

www.treefrog.fr

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

Seattle, WA

(Private)

Private biotech focused on bringing the transformational activity of CAR T therapy to all patients in need. We are the leaders in in vivo cart delivery with two products in the clinic, our own GMP manufacturing facility and strong Pharma partnerships. Umoja also has an iPSC platform and a targeting approach for solid tumors in development. We are committed to share our learning along the development path with the ARM membership

www.umoja-biopharma.com

Benjamin Gaub, Ph.D., Business Development Director

Amsterdam, Netherlands

(NASDAQ: QURE)

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

www.uniqure.com

Petter Bjorquist, Ph.D., CEO

Gothenburg, Sweden

(Private)

VERIGRAFT AB is a Swedish biotech company revolutionizing regenerative medicine with its cutting-edge tissue therapy solutions. At the core of its innovation is a proprietary technology that transforms donor tissues into personalized grafts, ensuring compatibility with the recipient’s immune system. This breakthrough 100% biocompatibility and eliminates the risk of rejection, setting a new standard for safety and efficacy in tissue transplants. VERIGRAFT’s current focus is on its flagship product, the Personalized Tissue Engineered Vein (P-TEV), designed to treat chronic venous insufficiency – a debilitating condition affecting millions worldwide. Backed by strong scientific expertise and ongoing clinical studies, VERIGRAFT is positioned to address unmet medical needs and deliver transformative therapies. With its commitment to advancing the future of regenerative medicine, VERIGRAFT aims to redefine patient care and improve outcomes for individuals who previously had limited or no treatment options.

www.verigraft.com

Jason Politi, Chief Technical Operations Officer

Boston, Massachusetts

(NASDAQ: VERV)

At Verve, we are striving to build the preeminent company developing gene editing medicines to treat atherosclerotic cardiovascular disease (ASCVD). We are leveraging the expertise and capabilities of our team whose singular focus is on addressing the root causes of this condition that touches so many individuals worldwide, whether personally or through a loved one. Our goal is to disrupt the chronic care model for treating ASCVD by providing a new therapeutic approach with single-course gene editing medicines.

www.vervetx.com

Jean -Philippe Combal, Ph.D., Chairman and CEO

Paris, France

(Private)

Vivet Therapeutics is a private, clinical-stage biotech company developing novel and long-lasting gene therapies for rare inherited metabolic conditions, including Wilson’s Disease. Vivet’s gene therapy platform uses recombinant adeno-associated viruses (rAAVs) as vectors and has initiated two clinical programs and four pre-clinical assets to date. Its most advanced program VTX-PID, is aimed at depleting Neutralizing Antibodies (NAbs) levels for a given AAV serotype and is advancing in a Phase 1 dose escalating study in Nabs+ subjects. Its Leading pre-clinical program VTX-806, for the treatment of Cerebrotendinous Xanthomatosis (CTX), received ODD by the EC & US FDA in 2024 and with its lead candidate, demonstrating its potential as an alternative treatment option for patients affected by the burden of CTX.Vivet Therapeutics was founded in 2016 by CEO Dr Jean-Philippe Combal and CSO Dr Gloria Gonzalez-Aseguinolaza and is led by a highly experienced management team with deepexpertise developing gene therapies and orphan drugs.Vivet Therapeutics is backed by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer Inc., Columbus Venture Partners, Ysios Capital, Kurma Partners and Eurazeo. In 2019, key investor Pfizer contributed a €45M investment to collaborate with Vivet in recognition of its scientific expertise and innovative technology platforms.

www.vivet-therapeutics.com

Robert Ang, M.D., President and CEO

Cambridge, MA

(NASDAQ: VOR)

Vor Bio is a clinical-stage cell and genome engineering company that aims to change the standard of care for patients with blood cancers by engineering hematopoietic stem cells to enable targeted therapies post-transplant.

www.vorbio.com

David Del Bourgo, CEO and Co-founder

Paris, France

(Private)

WhiteLab Genomics is a French-American TechBio company founded in 2019, leveraging Artificial Intelligence to in silico accelerate and mitigate risks in early-stage R&D within the field of genomic medicine. We’re using biological data guided rational approaches to improve these cell and gene therapies, DNA or RNA-based, addressing both viral and non-viral delivery. We are actively engaged in partnerships with renowned academic institutions (INSERM, Vision Institute of Paris, Brain Institute of Paris, Genethon), biotech and pharmaceutical companies (Sanofi, Debiopharm, SIREN Biotechnology), leveraging our ecosytem (YCombinator, Gingko Bioworks, Co.Lab Bayer). Our tools and approaches are aimed to address critical challenges in the biopharma industry through accelerating target discovery, vector (either viral and non-viral) and payload design, and bioproduction. By bringing together the power of computer sciences and biology, we help our partners develop more genomic medicines, faster and make those revolutionary drugs more accessible, affordable, and safer to patients in need.

www.whitelabgx.com

Evy Lundgren Akerlund Ph.D., CEO

Lund, Sweden

(NASDAQ: XINT)

Xintela is a clinical-stage biopharma company developing novel allogeneic stem cell therapies based on its proprietary stem cell marker technology. Xintelas product platform XSTEM® consists of homogenous integrin α10β1-selected Mesenchymal stem cells (MSCs) from adipose tissue, with superior potency and consistency. In a Phase I/IIa study on knee osteoarthritis (OA) patients, safety has been demonstrated with three different dose levels in 24 patients and patients report reduced pain and improved function of the knee joint. An interim analysis of safety and efficacy, 18 months after dosing, will be performed in Q1 2025. XSTEM is also investigated in a Phase I/IIa clinical study for the treatment of difficult-to-heal venous leg ulcers (VLU). Extensive preclinical studies in large animals, support disease modifying effects in OA and VLU.

www.xintela.se