Jiwu Wang, Ph.D., CEO

San Diego, CA

(Private)

Allele Biotech is recognized as an industry leader in iPSC technology, offering the highest-quality mRNA-reprogrammed iPSC lines at the largest scale among commercial providers. Our bespoke lines are being integrated into clinical programs by partners worldwide. As an official resource partner of CRIM, we provide CDMO services for process development, CMC, and cGMP . Our advanced mRNA-directed iPSC differentiation platform is currently powering an ARPA-H-funded liver organ bioprinting project for transplantation. Other iPSC-derived cell types have been produced for NAM partners as assays of cells, organoids, and miniorgans . Our internal clinical program development team has established a best-in-class iPSC-beta islet production process, with convincing in vivo data and a proven path to clinical applications.

Allelebiotech.com

Casey McPherson, CEO and Founder

Austin, TX

(Private)

AlphaRose Therapeutics is a groundbreaking precision medicine company focused on advancing genetic treatments for ultra rare diseases, with our ASO and AI platform. Built upon the pioneering work of industry leaders like Henri Termeer and inspired by the incredible stories of Milasen and others. Our mission is to revolutionize the delivery of genetic therapies, speeding up their availability and expanding access, starting with children who need them the most. Our team of scientists, biotech professionals, and parents are united by a shared purpose: to create life-changing medicines for children suffering from genetic diseases.

www.alpharose.com

Mark Farmery, Ph.D., Chief Development Officer

Södertälje, Sweden

(Private)

Anocca is a clinical-stage biopharmaceutical company pioneering the development of next-generation T-cell receptor-engineered T cell (TCR-T) therapies to transform the treatment of solid tumor cancers. Anocca’s platform and product library unlocks the hurdles to cost-effective, industrial-scale development and deployment of personalized cell therapies, resulting in the largest pipeline of targeted TCR-T therapies in the world. Proprietary discovery technologies using programmable human cells and AI-acceleration, non-viral gene editing, and in-house GMP manufacturing give Anocca end-to-end control from disease gene-of-interest to developing and manufacturing products and executing clinical trials. The platform is designed for rapid iteration and lower per-asset development costs. The company’s pipeline includes 40+ assets covering high value shared cancer targets including dysregulated genes, driver mutations, and viral oncoproteins. Anocca’s lead assets are being evaluated in VIDAR-1, a phase I/II multi-site/product European umbrella clinical trial targeting KRAS oncogenic driver mutations in pancreatic ductal adenocarcinoma.

www.anocca.com

Devyn Smith, Ph.D., CEO

Cambridge, MA

(Private)

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

www.arbor.bio

Cristiana Pires, Ph.D., CEO

Lund, Sweden

(Private)

Asgard Therapeutics is a privately held biotech company pioneering in vivo direct cell reprogramming for cancer immunotherapy. Building on proprietary reprogramming technologies and backed by leading investors, Asgard is establishing a pipeline of off-the-shelf gene therapy products designed to induce efficient and personalized immune responses. Our lead program, AT-108, is a first-in-class, off-the-shelf gene therapy that directly reprograms tumor cells into a rare subset of dendritic cells critical for mounting efficient cytotoxic T cell responses, cDC1 cells (conventional dendritic cells type 1). Reprogramming forces the tumor cells to present their tumor antigens, leading to a personalized anti-tumor immune response. It is based on a replication-deficient adenoviral vector that delivers three proprietary reprogramming factors into tumor cells, rewiring them to become antigen-presenting cDC1-like cells. Asgard is currently preparing for clinical trials of AT-108 by progressing IND-enabling studies and CMC development. Key proof-of-concept data on AT-108 were published in the journal Science in 2024.

www.asgardthx.com

Tamer Mohamed, Founder and CEO

Vancouver, Canada

(Private)

Aspect Biosystems is a biotechnology company pioneering the development of Bioprinted Tissue Therapeutics (BTTs) 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 AI-powered bioprinting technology, therapeutic cells, biomaterials, and computational design. Aspect’s BTTs 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, rare endocrine disorders, and liver diseases. Aspect has raised over US$260M in funding and is backed by leading tech-bio and strategic investors including Dimension, Novo Nordisk, and Radical Ventures. The company is advancing its pipeline through internal development and strategic collaborations, including its partnership with Novo Nordisk.

www.aspectbiosystems.com

Lisa Deschamps, CEO and Board Member

London, UK

(Private)

AviadoBio is relentlessly chasing cures and delivering hope to families living with neurodegenerative disease. We are developing one-time gene therapies that are built on scalable, targeted delivery technology designed to overcome the biological challenges of treating neurological diseases, prioritizing precision, efficacy and safety. AviadoBio’s pipeline includes programs for frontotemporal dementia (FTD), inherited retinal dystrophies such as retinitis pigmentosa, and tauopathies, including Alzheimer’s disease. The company is committed to advancing therapies that target complex neural systems across the brain, spinal cord, and retina using proprietary capsid engineering and our novel best in class vMiX RNA gene silencing platform. The company has raised over $160 million to date and last year signed an exclusive option and license agreement with Astellas Pharma for AVB-101. Under the terms of the agreement, Astellas provided a $50 million upfront and if exercised up to $2.2 billion in license fees and milestone payments plus royalties.

www.aviadobio.com

Robert Lin, Ph.D., CEO

Pittsburgh, PA

(Private)

Avista utilizes an AI-guided platform to engineer AAVs for tissue-specific delivery of genetic medicines. ATX002 is a best-in-class intravitreal capsid that outperforms the previous industry standard (AAV2.7m8) by up to 30x.  This increased performance can benefit patients through reduced dosing resulting in reduce inflammatory responses.  Our partner Roche is utilizing this capsid for larger ophthalmic indications, while Avista is pursuing rare inherited retinal disease treatments using ATX002. With our numerous high-quality datasets from non-human primate experiments, Avista has created a machine learning platform to further drive our engineered AAV platform.  We are currently testing further advances in the intravitreal capsid field.  Additionally, we are leveraging the AI-platform to revolutionalize AAV local delivery to the brain. By creating targeted capsids, Avista will enable delivery of genetic medicines with AAVs that can be delivered at much lower dose levels to reduce immunotoxicity and provide safer treatments for patients.

www.avistatx.com

Peter Keller, CEO

Philadelphia, PA

(Private)

BlueWhale Bio accelerates the adoption of cell-based therapies by modernizing cell manufacturing. The company is developing a portfolio of critical materials that enable higher-performing cell therapies to reach more patients faster and at lower cost. BlueWhale’s innovations stem from discoveries in cell activation by Dr. Carl June and Dr. Jim Riley at the University of Pennsylvania. Its Synecta™ platform uses cell-derived nanoparticles (CDNPs) that mimic the natural activation of immune cells, producing superior cell quality through a simplified, faster, and more scalable workflow. The first product, Synecta T1, mimics natural T-cell stimulation by delivering membrane-bound signals, cytokines, and adhesion molecules that enhance T-cell receptor engagement during CAR-T manufacturing.

www.bluewhale.bio

Giedrius Gasiunas, Ph.D., CEO

Vilnius, Lithuania

(Private)

Caszyme is a Lithuania-based gene editing company which combines its deep experience and dynamic platform to deliver world class CRISPR solutions. Caszyme was established in 2017 by scientists, who were first to demonstrate that CRISPR-Cas9 can be used to generate double strand DNA breaks. Over the last 8 years, Caszyme has rapidly advanced its excellence and capabilities as an enabling expert partner in CRISPR gene editing solutions, collaborating with multiple life science organizations aiming to enter the CRISPR field or expand their gene editing capabilities. Also, Caszyme has developed a platform of unique CRISPR nucleases, including novel Cas12l, available for licensing. This enables Caszyme partners across various industries effectively apply CRISPR gene editing technology and tools for their market-ready products. Currently, in the therapeutics field Caszyme aims to develop safer and more compact gene editing tools for treating genetic eye diseases in vivo.

www.caszyme.com

Matthew Durdy, Ph.D., Chief Executive

London, UK

(Private)

The Cell and Gene Therapy Catapult is an independent innovation and technology organization committed to the advancement of the CGT industry with a vision of a thriving industry delivering life changing advanced therapies to the world. Its aim is to create powerful collaborations which overcome challenges to the advancement of the sector. With around 300 experts covering all aspects of advanced therapies, it applies its unique capabilities and assets, collaborates with academia, industry and healthcare providers to develop new technology and innovation.

ct.catapult.org.uk

Jason Jones, Global Head of Business Development

Cambridgeshire, UK

(Private)

Cellular Origins is enabling the manufacture of cell therapies at scale. With a flexible, scalable, factory-led approach, Cellular Origins has overcome the unique challenges of cell therapy manufacturing, unlocking access to the same advanced automation that has driven scale in logistics, automotive, and silicon fabrication industries. Cellular Origins’ platform, Constellation, combines mobile robotics and AI with proven bioprocess instruments, providing end-to-end manufacturing, eliminating therapy redevelopment, and speeding up clinical-to-commercial timelines. Cellular Origins is trusted by market leaders such as J&J, Cytiva, Fresenius Kabi, Thermo Fisher Scientific, and Wilson Wolf. Constellation orchestrates seamless automation of existing workflows, reliably scaling manufacture to enable the production of 100,000s of doses of transformative therapies.

www.cellularorigins.com

Larry Forman, CEO

San Francisco, CA

(Private)

CHO Plus is driving down the cost of manufacturing AAV viral vectors while also making it possible to produce unlimited doses for treating common diseases. We have a dual approach: (a) We engineer HEK-293 cells for up to 20-fold higher productivity (4 x 10^16 vg/liter) and up to 2-fold higher full capsids (up to 55% full) before purification.  Further advances are anticipated. (b) We have developed non-leaky, inducible promotors that allow for normal growth before induction, and full production with high percent-full capsids after induction.  Scale-up to 10,000 liters is possible, using small molecule inducers.  This innovation eliminates the need for transient transfection (and the costly DNA), and it allows for the manufacture of millions of gene therapy doses. We will present our work, and discuss its value—to pharmaceutical companies, and to the patients who need these therapies.

www.cho-plus.com

Rodolphe Clerval, CEO

Paris, France

(Private)

Coave Therapeutics is tackling the main hurdle in gene therapy: delivery. The company has developed the world’s first ligand-conjugated AAVs, creating Precision Vectors that are highly tissue-specific, accurately delivered, and safer than traditional methods. This targeted approach enables a first-in-class precision suprachoroidal vectorcapsid aimed at transforming retinal vascular diseases such as neovascular AMD and diabetic macular edema. Coave is advancing its lead program, CoTx-101, toward clinical development with the aim of delivering a best-in-class, durable, and more convenient treatment that reduces the need for frequent injections. Its plug-and-play technology can be applied to any capsid/vector, regardless of administration route or target tissue, unlocking exploration of indications previously deemed unfeasible with standard gene therapy. Founded on pioneering science from the University of Nantes and supported by rigorous quantitative analysis, Coave is led by a team of gene therapy and ophthalmology experts.

www.coavetx.com

Daniel Getts, Ph.D., CEO

Cambridge, MA,

(Private)

CREATE Medicines is a clinical-stage biotech pioneering in vivo multi-immune programming. Its proprietary mRNA-LNP platform directly programs immune cells inside the body to deliver scalable, repeat-dose, off-the-shelf immunotherapies. With proven human validation and next-generation RNA technology, CREATE is advancing a pipeline of in vivo CAR therapies to transform outcomes in cancer and autoimmunity.

createmedicines.com

Daniela Marino, Ph.D., CEO

Schlieren, Switzerland

(Private)

We are a Swiss biotech leader in skin regenerative medicine. Our first focus is to provide patients who suffer from large skin injuries and defects with personalized skin tissue therapy in a safe, effective and accessible manner via a proprietary automation platform. We are in phase 3 clinical trials. 50 FTE, 100m $ raised to date.

www.cutiss.com

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

Paris, France

(Private)

EG 427 is the global leader in non-replicating HSV-1 (nrHSV-1) vectors in neurology. EG 427 has started a phase1/2 study in the US with its lead DNA medicine candidate, EG110A, in patients with neurogenic detrusor overactivity-related incontinence. This is the first human study with an nrHSV-1 vector targeting sensory neuron-based diseases. EG110A is being developed to addresses multiple severe bladder diseases and has the potential to be a major improvement over existing therapies, resulting in better care for patients and lower costs for healthcare systems. The company’s unique HERMES platform delivers pinpoint neurotherapeutics to treat prevalent diseases of the peripheral and central nervous system. Its vectors can achieve focal transduction in specific regions and selective transgene expression in targeted subsets of neurons. With demonstrated clinical safety and possible repeated dosing, the large payload capacity of nrHSV-1 vectors allows for versatile DNA delivery for smarter DNA medicine.

www.eg427.com

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

Hamburg, Germany

(NASDAQ : EVT)

Evotec is a leading life sciences company dedicated to delivering highly effective new therapeutics to patients. Leveraging multimodality platforms, we drive proprietary projects and co-create pipelines with partners across pharma, biotech, academia, and other healthcare stakeholders. Our flexible business model spans from service-based support to strategic partnerships. In cell therapy, Evotec offers one of the industry’s most comprehensive end-to-end platforms for advancing iPSC-based projects from inception to clinic. This includes in-house GMP manufacturing and GMP gene-editing capabilities. We focus on indications requiring long-term cell engraftment, such as regenerative therapies (beta cells, cardiomyocytes, photoreceptors) and immune-modulation therapies (macrophages). Additionally, Evotec provides integrated drug development solutions, covering safety assessment, CMC, process and analytical development, regulatory strategy, and clinical services for both autologous and allogeneic cell therapy candidates.

www.evotec.com

Taleen Barsoumian, Senior Vice President, Client Development

Grove City, OH

(Private)

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

www.forgebiologics.com

David Curry, CEO

Philadelphia, PA

(Private)

GE2P2 Global was founded in 2016 as a non-profit [501(c)3] spin-off from the NYU School of Medicine’s Division of Medical Ethics. Our public benefit corporation affiliate provides medical ethics/bioethics advisory services to selected global, public sector, INGO, academic, and commercial organizations. A key focus of our advisory work is genomic medicine, gene editing, and the wider ecology of convergent biosciences. We support organizations facing complex, real-world challenges across the discovery-development-translation life cycle — helping develop and implement evidence-grounded, operationally-sound, and ethically-resilient solutions. Ge2p2.org GeneScape Karen Malone, Ph.D., CEO Leiden, Holland (Private) Genetically defined diseases, including rare disease populations are challenging to quantify, leading to prevalence underestimations. This weakens business cases, leads to unreliable forecasts, causes promising projects to be halted prematurely, and misaligns commercial strategies. Additionally, misdiagnosis further drives inefficient trial design, slow patient recruitment, and increases development costs. GeneScape provides prevalence estimates based on population genetics modelling, as well as literature based meta-analysis. We offer diagnosis-independent patient estimates with high genetic and geographic resolution, delivering more robust incidence and prevalence data than classical epidemiology by integrating 1.2 million genomes globally. This is further supported by our proprietary atlases in key therapeutic areas. Innovative companies that partner with GeneScape , enable stronger decision-making, optimized trial design, and more effective strategic planning through robust, genetics-driven target patient population insights.

ge2p2.org

Karen Malone, Ph.D., CEO

Leiden, Netherlands

(Private)

Genetically defined diseases, including rare disease populations are challenging to quantify, leading to prevalence underestimations. This weakens business cases, leads to unreliable forecasts, causes promising projects to be halted prematurely, and misaligns commercial strategies. Additionally, misdiagnosis further drives inefficient trial design, slow patient recruitment, and increases development costs. GeneScape provides prevalence estimates based on population genetics modelling, as well as literature based meta-analysis. We offer diagnosis-independent patient estimates with high genetic and geographic resolution, delivering more robust incidence and prevalence data than classical epidemiology by integrating 1.2 million genomes globally. This is further supported by our proprietary atlases in key therapeutic areas. Innovative companies that partner with GeneScape, enable stronger decision-making, optimized trial design, and more effective strategic planning through robust, genetics-driven target patient population insights.

www.genescape.nl

Julianne Smith, Ph.D., Chief Development Officer

Milano, Italy

(Private)

Genespire is a biotechnology company, developing off-the-shelf 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. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini and Dr. Alessio Cantore, the 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

Alessandro Padova, Ph.D., CEO

Modena, Italy

(Private)

Holostem srl is a commercial stage biotech company delivering life changing advanced therapeutic medicinal products for patients affected by orphan and rare diseases. Holostem srl is 100% owned by Fondazione ENEA Tech e Biomedical, a private foundation supervised by the Ministry of Business and Made in Italy and the Ministry of Health. Holostem pioneered GMP manufacturing for Advanced Therapeutic Medicinal Products. Holoclar® is the first cell therapy to receive conditional marketing authorization by EMA and MHRA in 2015. Holoclar has received full marketing approval in 2024 following a positive Phase III trial. Holostem has been recognized by the European Commission as a leader in the field of cell and gene therapy for orphan and rare therapeutic indications. Holostem project entitled “Integrated Cell and Gene Therapy Platforms” has been selected as one of the 13 EU programs to be funded under the IPCEI (Important Projects of Common European Interest).

www.holostem.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 early 2026 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 powering the development of disease-responsive advanced therapies through our unique, programmable gene control technology. Our innovative platform harnesses the cell’s natural regulatory mechanisms to deliver programmable and tunable gene control through recoded miRNAs. This enables the development of highly effective medicines with enhanced precision and improved safety profiles. Utilizing our platform technologies, we are working to develop the next-generation of advanced therapies, both through our own pipeline – targeting oncology and genetic medicine – and through partnerships.

www.laverocktx.com

Jeff Liter, CEO

Minneapolis, MN

(Private)

Luminary Therapeutics is a clinical-stage biotechnology company developing next-generation CAR T-cell therapies that integrate advanced CAR engineering, novel targets and proprietary manufacturing innovations to create durable, transformative treatments that could become broadly accessible to patients with cancer and autoimmune diseases. Luminary’s lead program features a multi-antigen targeting BAFF ligand CAR designed to overcome tumor escape pathways and achieve robust B-cell depletion, including long-lived plasma B cells. Lead product, LMY-920 is an autologous BAFF-CAR T-cell therapy currently in Phase 1 clinical trials for NHL and MM, with planned expansion into CLL and lupus and rheumatoid arthritis. Second product, LMY-932, is an allogeneic BAFF-CAR T-cell therapy, engineered on a gamma-delta chassis for improved tumor homing and safety, expected to enter clinical trials in hematologic cancers and autoimmune diseases in 2026. Additionally, Luminary’s second program is developing gamma-delta T cells engineered with dual-targeting CARs for enhanced safety and broad applicability across solid tumors.

www.luminarytx.com

Yael Weiss, M.D., Ph.D., CEO

San Francisco, CA

(Private)

Mahzi specializes in developing genetic therapies for neurogenetic disorders that present clinically with seizures, autism and intellectual disabilities. Our lead program an AAV9 gene therapy for Pitt Hopkins Syndrome is now in a P1/2 study, with additional AAV gene therapy and ASO programs close behind. Mahzi works closely with PAtient Advocacy Groups to advance gene specific disease modifying therapies from bench to bedside.

www.mahzi.com

Ali Afshar, Ph.D., CEO and Co-Founder

London, UK

(Private)

Mytos runs fully-automated stem cell manufacturing sites powered by our iDEM automation platform – to scale stem cell-derived therapies to millions of patients rapidly at lower cost. Manual manufacturing at CDMOs has held back Regenerative Medicine due to high costs and high failure rates. With Mytos ’ automated CDMO, these therapies can now be scaled globally at low cost. We’ve announced partnerships across multiple therapies so far ranging from Parkinson’s, to hearing loss, to bone repair. Within the next 12- 18 months , cells grown on our machines will be going into patients – and our first site will be licensed for releasing patient batches by September.

www.mytos.bio

Sean Kevlahan, Ph.D., Cofounder and CEO

Boston, MA

(Private)

Nanite is a AI-native materials science company focused on first principles design of novel polymeric materials for nucleic acid delivery. We have developed polymer nanoparticles that enable DNA delivery via subcutaneous (SubQ) injection, turning adipocytes (human fat cells) into factories for proteins and peptides expression as well as IV for tissue specific DNA delivery.

www.nanitebio.com

Helen Sabzevari, Ph.D., President and CEO

Germantown, MD

(NASDAQ: PGEN)

Precigen is a commercial-stage biopharmaceutical company specializing in the advancement of innovative precision medicines to address difficult-to-treat diseases with high unmet patient need. Precigen is dedicated to advancing scientific breakthroughs from proof-of-concept through commercialization. With a strong commitment to innovation, Precigen is developing a robust pipeline of differentiated therapies across its core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases.

www.Precigen.com

Gaurav Shah, M.D., CEO

Cranbury, N.J.

(NASDAQ: RCKT)

Rocket Pharmaceuticals, Inc. 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 innovative multi-platform approach 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. Rocket’s adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage clinical program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, and an early-stage clinical program for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket’s lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia.

www.rocketpharma.com

Charlotte Guzzo, Ph.D., Co-Founder and Chief Operating Officer

Cambridge, UK

(Private)

For cell and gene therapy developers who rely on precise, genotype-driven patient identification, Sano Genetics is the precision patient platform that unifies recruitment, eligibility screening, at-home genetic testing, and long-term engagement in one compliant, modular ecosystem. Unlike CROs or point solutions that only address isolated steps, Sano’s 360° platform reduces screen failures, accelerates enrollment, and builds reusable, consented, genotype-tagged cohorts that support both clinical development and post-approval evidence generation. We create the missing patient data layer for modern drug development, supporting early natural history insights, recontact across programs, and real-world follow-up for durability and safety. By maintaining continuous connection from first touchpoint through long-term engagement, Sano helps gene therapy and rare disease sponsors lower operational risk, shorten timelines, and ensure valuable data and patient relationships carry forward across studies and programs.

www.sanogenetics.com

Ricardo Baptista, Ph.D., Chief Technology Officer

Stockholm, Sweden

(Private)

SmartCella is a Sweden-based biopharma company developing next-generation regenerative and molecular therapeutics supported by advanced targeted-delivery technology. At the core is the Extroducer®, a first-in-class endovascular infusion catheter designed to overcome limitations in accessing hard-to-reach tissues and organs. The Extroducer enables precise intraparenchymal administration of cells, genes, biologics, or small molecules through a controlled micro-needle that safely crosses the vessel wall while maintaining vascular integrity. This minimally invasive approach improves targeting accuracy, reduces systemic exposure, and may lower effective doses through localized delivery. By avoiding open surgery, it reduces patient burden, shortens recovery, and supports repeat or multi-site dosing with high reproducibility. Preclinical studies across multiple organs demonstrate localized biodistribution, favorable safety, and consistent procedural performance. This presentation outlines SmartCella’s vision, the engineering principles behind the Extroducer, and key translational data supporting its clinical advancement, positioning the technology as an enabling platform for precise targeted delivery.

www.smartcella.com

Laura Koivusalo, CEO and Founder

Tampere, Finland

(Private)

StemSight develops off-the-shelf stem cell therapies for curing blindness. Based out of Tampere, Finland, StemSIght is backed by 20+ years of academic research on pluripotent stem cells for ocular applications. StemSight’s go-to-market is a rare disease called limbal stem cell deficiency (LSCD), a severe and currently untreatable corneal disease, with an orphan drug fast-track to market and a well-established clinical rationale for the cell therapy approach. StemSight’s lead asset is currently in preclinical stage, with first-in-human trials planned for 2028.

www.stemsight.fi

Andries Zijlstra, Ph.D., Chief Technology Officer

Houston, TX,

(Private)

StimulusBio accelerates the development of cell and gene therapies through innovative product solutions that reduce the time and cost of therapeutic development. Our scalable, easy-to-integrate technologies drive consistency, enhance cellular expansion, and streamline complex workflows empowering researchers and manufacturers to more efficiently translate discoveries into clinical solutions. The StimulusBio product pipeline centers around a programmable cellular interface ( sbPCI ) that enables precise control over cellular environments, supporting reproducible outcomes and faster iteration in both research and clinical translation. In addition to tools development, our scientists are working to realize applications of sbPCI in therapeutic solutions. By combining commercial expertise and scientific innovation, StimulusBio is committed to ensuring that our foundational sbPCI technology successfully enables the leap from the bench to the bedside.

www.stimulusbio.com

David James, Ph.D., Chief Scientific Officer

Sheffield, United Kingdom

(Private)

SynGenSys designs synthetic promoter systems and signal peptides to enable precise control of gene expression for cell and gene therapy applications and biopharmaceutical manufacturing. Using its proprietary bioinformatics platform and integrated testing workflows, SynGenSys develops compact, tunable promoters that deliver tissue-specific targeting and accurate dosing for DNA-based gene therapies and gene-modified cell therapies including CAR-T and CAR-NK. Promoter systems are independent of delivery vector , improving targeting for viral vectors such as AAV and non-viral approaches. SynGenSys’s signal peptides are engineered to optimize protein translocation and expression for secreted and membrane-anchored transgenes such as CARs and clotting factor VIII. SynGenSys also supports biopharmaceutical manufacturing with off-the-shelf and custom promoter systems that improve yield and turnaround time, even for multi- component or difficult-to-express proteins. By combining computational transcriptional analysis with rational design, SynGenSys delivers promoter and signal peptide solutions that enhance safety, efficacy, and manufacturability for next-generation medicines.

www.syngensys.com

Jason Slingsby, Ph.D., CEO

Sharnbrook, UK

(Private)

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

www.tozaro.com

Petter Bjorquist, Ph.D., CEO

Gothenburg, Sweden

(Private)

VERIGRAFT is at the forefront of personalized tissue therapies, bringing a paradigm shift to the treatment of serious disease. By harnessing natural, tissue-engineered grafts, VERIGRAFT offers a groundbreaking approach to restore and regenerate damaged vascular and other structures. This innovation provides patients with severe and debilitating vascular conditions access to customized, life-changing treatment options that were previously unattainable, transforming the possibilities in personalized medicine and paving the way for a new era in advanced therapeutic solutions.

www.verigraft.com

Brian Feth, CEO

San Francisco, CA

(Private)

Xcellbio is a venture-backed (Viking, Casdin , HBM, Labcorp ) San Francisco-based cell therapy technology company with a novel GMP manufacturing platform designed to enhance metabolic fitness leading to more persistent and potent cell therapies. The Company’s proprietary approach to modifying immunometabolism, protected by over a dozen patent families, drives improvements in cell therapy potency and enables AI-driven optimization of therapeutic products during manufacturing. The Company offers platforms for both small and large scale process development and manufacturing of immune cell therapies, as well as platforms to assess the functional potency of cell therapies. The core Avatar technology was approved for clinical use in 2023 and is used for every patient that receives Lyfgenia , a curative gene therapies for sickle cell anemia. In addition, the Company, along with its partners, is advancing several immune cell therapies to IND filings with plans for first-in-human use in 2026.

www.xcellbio.com