David Kirn, Founder and CEO 

Oct. 11 | 11:30am | UBC Ballroom 

Emeryville, CA 

(NASDAQ: FDMT) 

In-person Presentation 

4D Molecular Therapeutics (4DMT) seeks to unlock the full potential of gene therapy using our platform, Therapeutic Vector Evolution (TVE). TVE combines the power of directed evolution with over one billion synthetic capsid sequences to invent evolved vectors for use in targeted gene therapy products. Our targeted and evolved AAV vectors are invented with the goals of being delivered through clinically routine, well-tolerated and minimally invasive routes of administration; of transducing diseased cells in target tissues efficiently; of having reduced immunogenicity; and, where relevant, of having resistance to pre-existing antibodies.  

www.4dmoleculartherapeutics.com 

Shelley Hartman, CEO

Oct. 11 | 11:45am | Oxford Biomedica Ballroom  

Woburn, MA 

(Private) 

In-person Presentation 

Aegle Therapeutics is a pioneer in the development of extracellular vesicle (EV) therapy. We are focused on rare diseases, including epidermolysis bullosa (EB), and burns. Aegle has a proprietary EV harvesting technology that is critical to the production of clinical grade product. Ours is a platform technology with potential indications including dermatology, immunology, and others. Aegle has been awarded Rare Pediatric Disease and Fast Track designation by the FDA for its EB indication. Aegle is seeking funding to launch its clinical trial in EB. 

www.aegletherapeutics.com 

Matt Britz, Chief Operating Officer 

Oct. 11 | 4:15pm | Oxford Biomedica Ballroom 

Natick, MA 

(Private) 

In-person Presentation 

AffyImmune is realizing the potential of cancer immunotherapy by extending the anti-cancer activity of CAR T cell therapy to solid tumors. AffyImmune’s proprietary technology allows fine-tuning of the affinity of CAR T cells to reduce toxicity, and a tracking system to allow for real-time localization of CAR T cells after administration to patients. By affinity-tuning CAR T cells to select those targeting overexpressed but non-unique tumor associated antigens, we avoid toxicity to normal cells expressing lower levels of the target. Lower affinity CARs result in activation of CAR T cells in a manner more similar to the natural activation observed in non-engineered T cells. This more physiological activation leads to better serial tumor cell killing, clonal expansion, and long-term persistence to result in greater effectiveness. The company was founded in 2016 and is currently enrolling patients in its Phase I trial to treat advanced thyroid cancer.  

www.affyimmune.com 

Phil Morton, Ph.D., Chief Technology Officer 

Oct. 12 | 3:15pm | Oxford Biomedica Ballroom 

Nottingham, UK 

(Private) 

In-person Presentation 

Albumedix is a science-driven company and recognized leader of best-in-class albumin-enabled solutions. Established in the UK in 1984, with a mission to empower excellence, Albumedix has supported its life science partners to deliver hundreds of million safe doses of clinical and marketed therapeutics globally. The company’s solutions include the world’s only USP-NF compliant recombinant human albumin (Recombumin®), client-centric development and compliance services, and drug-enhancing technologies (Veltis®). Recombumin® albumins have been used throughout the entire cell therapy development process, including improving culture, expansion, cell isolation, formulation, and cryopreservation. The effect is incredibly pronounced in high stress environments such as freeze and thaw as well as improving viral vector stability. By always striving for more and collaboratively challenging status quo, Albumedix enables the development and commercialization of advanced therapies and next-generation biopharmaceuticals to the benefit of people worldwide. 

www.albumedix.com 

Jim Williams, Ph.D., Vice President R&D and Chief Scientific Officer, Nature Technology, an Aldevron company

Oct. 12 | 9:45am | Oxford Biomedica Ballroom 

Fargo, ND 

(NASDAQ: DHR) 

In-person Presentation 

Aldevron serves the biotechnology industry with custom production of nucleic acids, proteins, and antibodies. Thousands of clients use Aldevron-produced plasmids, RNA, and gene editing enzymes for projects ranging from discovery research to clinical trials to commercial applications. These products are critical raw materials and key components in commercially available drugs and medical devices. Aldevron is known for inventing the GMP-Source® quality system and for specializing in cGMP manufacturing, operating the world’s largest facility at its company headquarters in Fargo, North Dakota, with additional facilities in Madison, Wisconsin, and Lincoln, Nebraska. 

www.aldevron.com 

Diana Brainard, M.D, CEO 

Oct. 12 | 2:30pm | UBC Ballroom 

Waltham, MA 

(NASDAQ: ALVR) 

In-person Presentation 

AlloVir is a late clinical-stage cell therapy company focused on restoring natural immunity against life-threatening viral diseases in immunocompromised patients. The company’s innovative, proprietary technology platforms leverage off-the-shelf, allogeneic, single- and multi-virus-specific T cells (VSTs) for patients with T cell deficiencies who are at risk from the life-threatening consequences of viral diseases. AlloVir’s technology and manufacturing process enables the potential for the treatment and prevention of a spectrum of devastating viruses with each single therapy. The company is advancing multiple mid- and late-stage clinical trials across its portfolio. AlloVir’s lead product, Posoleucel, is a multi-VST therapy targeting six viral pathogens: adenovirus, BK virus, cytomegalovirus, Epstein-Barr virus, human herpesvirus-6, and JC virus. Posoleucel is in Phase III development for three distinct indications and has received regulatory designations including FDA’s Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation (ODD), and EMA’s Priority Medicines (PRIME) and Orphan Medicinal Product (OMP). 

www.allovir.com 

Patricio Sepulveda, Ph.D., CEO 

Oct. 11 | 4:45pm | Oxford Biomedica Ballroom

Towson, MD

(Private) 

In-person Presentation 

Amplo Biotechnology is a late preclinical stage company developing AAV therapies to treat diseases of the neuromuscular junction, the connection between muscle and nerve.  Amplo’s lead asset, AMP-101, uses a muscle specific AAV construct expressing the Dok7 protein.  Patients who do not produce Dok7 develop Dok7 Congenital Myasthenic Syndrome, a severe rare disease of the neuromuscular junction. AMP-101 is able to cure the Dok7 mouse model with a dose that is ¼ the effective dose in other systemic muscle targeted AAV programs.  AMP-101 is further applicable to other forms of congenital myasthenic syndromes, Emery Dreifuss muscular dystrophy, ALS and sarcopenia, all for which there is animal data supporting AMP-101’s ability to stabilize the neuromuscular junction and improve muscle function. Amplo is currently conducting a dose finding pharmacology study and GLP tox studies on AMP-101, with the aim of a 2023 IND submission. 

www.amplobiotechnology.com

Sven Kili, CEO 

Oct. 12 | 1:45pm | Oxford Biomedica Ballroom 

Geneva, Switzerland  

(Private) 

In-person Presentation 

Antion Biosciences is the world leader in efficient, safe and cost effective Cell Multiplexing technologies for almost any cell type. We develop cutting-edge allogeneic immuno- cell therapies in indications of high unmet medical and technical engineering need for patients who need hope to become reality. We will start by changing the way oncology cell therapies are developed. 

www.antion.ch 

Devyn Smith, Ph.D., CEO

Oct. 11 | 2:30pm | Oxford Biomedica Ballroom 

Cambridge, MA 

(Private) 

In-person Presentation 

Arbor Biotechnologies is a life sciences company discovering and developing the next generation of genetic medicines based on wholly owned genomic editors discovered from its machine learning/AI driven discovery platform. Since its founding in 2016 by Feng Zhang, David Walt, David Scott, and Winston Yan, Arbor has built the most extensive toolbox of proprietary genomic editors in the industry. Using the discovery platform, Arbor can discover, screen, and engineer novel editing enzymes and effectors that can then be tailored to the underlying cause of disease to result in potentially curative medicines for patients. As Arbor continues to advance its pipeline toward the clinic with an initial focus in liver and CNS disease, the company is also working with multiple partners, including Vertex Pharmaceuticals, on several gene editing and ex vivo cell therapy programs to broaden the reach of its novel nuclease technology. 

www.arbor.bio 

Aileen Fernandes, Chief Business Officer 

Oct. 11 | 5:45pm | UBC Ballroom 

Gaithersburg, MD 

(NASDAQ: ACLX) 

In-person Presentation 

Arcellx is a clinical-stage biotechnology company reimagining cell therapy by engineering innovative immunotherapies for patients with cancer and other incurable diseases. Arcellx believes that cell therapies are one of the forward pillars of medicine and Arcellx’s mission is to advance humanity by developing cell therapies that are safer, more effective, and more broadly accessible. Arcellx’s lead product candidate, CART-ddBCMA, is being developed for the treatment of relapsed or refractory multiple myeloma (r/r MM) in an ongoing Phase I study. CART-ddBCMA has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy designations by the U.S. Food and Drug Administration. Arcellx is also advancing its dosable and controllable CAR T therapy, ARC-SparX, through two programs: a Phase I study of ACLX-001 for r/r MM, initiated in 2Q 2022; and ACLX-002 in relapsed or refractory acute myeloid leukemia and high-risk myelodysplastic syndrome, expected to enter the clinic in 2H 2022. 

www.arcellx.com 

Xiaokui Zhang, Ph.D., Chief Scientific Officer 

Oct. 11 | 9:15am | Oxford Biomedica Ballroom 

San Diego, CA 

(Private) 

In-person Presentation 

Aspen Neuroscience is a development-stage, private biotechnology company focused on personalized cell therapies. We are developing autologous (the patient’s own) induced-pluripotent stem cells (iPSCs) to address diseases with high unmet medical need, beginning with autologous neuron replacement for both sporadic and genetic forms of Parkinson’s disease (PD). As a leading iPSC platform company, we combine stem cell biology with the latest artificial intelligence and genomic approaches to investigate patient-specific, restorative treatments. We have developed a best-in-class platform to create and characterize pluripotent-derived cell medicines, including in-house bioinformatics, manufacturing, and QC. 

www.aspenneuroscience.com 

Pascal Touchon, President and CEO 

Oct. 12 | 4:30pm | UBC Ballroom 

San Franciso, CA 

(NASDAQ: ATRA) 

In-person Presentation 

Atara Biotherapeutics is a pioneer in T cell immunotherapy, leveraging its novel allogeneic EBV T cell platform to develop transformative therapies for patients with serious diseases. With our lead program in Phase III clinical development and currently under review to support registration in Europe, Atara is the most advanced allogeneic T cell immunotherapy company and intends to rapidly deliver off-the-shelf treatments to patients with high unmet medical need. Our platform leverages the unique biology of EBV T cells and has the capability to treat a wide range of EBV-associated diseases, or other serious diseases through incorporation of engineered CARs or TCRs. Atara is applying this one platform, which does not require TCR or HLA gene editing, to create a robust pipeline including: tab-cel® (tabelecleucel) for EBV+ PTLD; ATA188, a T cell immunotherapy targeting EBV antigens as a potential treatment for multiple sclerosis; and next-generation CAR T immunotherapies for both solid tumors and hematologic malignancies. 

www.atarabio.com 

RW Mays, Ph.D., Vice President of Regenerative Medicine 

Oct. 11 | 4:00pm | Oxford Biomedica Ballroom 

Cleveland, OH 

(NASDAQ: ATHX) 

In-person Presentation 

Athersys is a clinical-stage biotechnology company developing novel and proprietary best-in-class therapies designed to extend and enhance the quality of human life. Our focus is on the treatment of medical conditions where there is significant clinical need, and we are particularly focused on developing therapies in the regenerative medicine area. We are developing MultiStem®, a patented, adult-derived off-the-shelf stem cell therapy platform, for disease indications in areas of neurological, inflammatory and immune, and cardiovascular disease areas, as well as other critical care indications where there is substantial unmet medical need due to the limitations in standard of care. 

www.athersys.com 

Christopher Vann, Chief Operating Officer 

Oct. 12 | 4:45pm | UBC Ballroom 

London, UK 

(NASDAQ: AUTL) 

In-person Presentation 

Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the company is engineering precisely targeted, controlled, and highly active T cell therapies that are designed to better recognize cancer 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 and solid tumors. 

www.autolus.com 

Rick Fair, President and CEO 

Oct. 11 | 4:30pm | Oxford Biomedica Ballroom 

Houston, TX 

(NASDAQ: BLCM) 

In-person Presentation 

Bellicum Pharmaceuticals is a clinical-stage biopharmaceutical company striving to deliver cures through controllable cell therapies. The company’s next-generation product candidates are differentiated by powerful cell signaling technologies designed to produce more effective CAR T cell therapies. Bellicum’s GoCAR-T® product candidates, BPX-601 and BPX-603, are designed to be more efficacious CAR T cell products capable of overriding key immune inhibitory mechanisms. 

www.bellicum.com 

Haro Hartounian, Ph.D., CEO 

Oct. 11 | 9:30am | Oxford Biomedica Ballroom 

Newark, NJ 

(Private) 

In-person Presentation 

BioCentriq is a premier cell and gene therapy development and manufacturing center located in New Jersey. The company operates multiple GMP and process development facilities and has in-house analytical method development capabilities. BioCentriq currently supports Phase I-III clinical trials with commercial manufacturing capabilities planned in the near future. Through its Hybrid Team Model, BioCentriq offers a collaborative environment in which clients are welcomed and encouraged to participate in development and manufacturing activities under the guidance of experienced staff. BioCentriq’s expertise spans across autologous and allogeneic cell therapies such as T cells, CAR T, MSC/iPSC, NK cells, dendritic cells, HEK 293(T), Sf9, and in viral vectors such as AAV, adenovirus, retrovirus, and lentiviral vectors with a maximum production capacity of 200L. 

www.biocentriq.com 

Seth Ettenberg, Ph.D., CEO 

Oct. 11 | 10:00am | Oxford Biomedica Ballroom 

Cambridge, MA 

(Private) 

In-person Presentation 

BlueRock Therapeutics is a leading engineered cell therapy company with a mission to create authentic cellular medicines to reverse devastating diseases, with the vision of improving the human condition. The company’s cell+gene platform harnesses the power of cells to create new medicines for neurology, cardiology, immunology, and ophthalmology indications. BlueRock Therapeutics’ cell differentiation technology recapitulates the cell’s developmental biology to produce authentic cells, some of which are engineered for additional therapeutic function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore lost function. 

www.bluerocktx.com 

Steven Nichtberger, M.D., President and CEO 

Oct. 11 | 10:45am | Oxford Biomedica Ballroom 

Philadelphia, PA 

(NASDAQ: CABA) 

In-person Presentation 

Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies that have the potential to provide a deep and durable, perhaps curative, treatment for patients with autoimmune diseases. The CABA™ platform, in combination with Cabaletta Bio’s proprietary technology, has advanced a growing pipeline that currently includes potential treatments for patients with mucosal pemphigus vulgaris, MuSK-associated myasthenia gravis, PLA2R-associated membranous nephropathy, mucocutaneous pemphigus vulgaris, and hemophilia A with FVIII alloantibodies. 

www.cabalettabio.com 

Xavier Avat, Chief Business Officer 

Oct. 12 | 12:00pm | Oxford Biomedica Ballroom 

San Diego, CA 

(NASDAQ: CAPR) 

In-person Presentation 

Capricor Therapeutics is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of first-in-class cell and exosome-based therapeutics for the treatment and prevention of diseases. Our innovative technology builds upon a large body of scientific research and enables us to approach the treatment of diseases in novel ways. Capricor’s lead candidate, CAP-1002, is an off-the-shelf cardiac cell therapy that is currently in late-stage clinical development for the treatment of Duchenne muscular dystrophy (DMD).CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that have been shown to exert potent immunomodulatory activity, which alters the immune system’s activity to stimulate cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials. 

www.capricor.com 

Kelly Kester, Director of Advanced Therapy Solutions 

Oct. 12 | 4:15pm | Oxford Biomedica Ballroom 

Dublin, OH 

(NASDAQ: CAH) 

In-person Presentation 

Cardinal Health Advanced Therapy Solutions brings a unique understanding of the challenges of launching and commercializing cell and gene therapies (CGTs) — and how to overcome them effectively. Our early involvement, starting with assisting biopharma in establishing the first CAR-T products in 2017, has enabled us to develop tailored solutions to simplify and streamline the complexities of CGTs. Our clinical and commercial capabilities across the product lifecycle, from regulatory consulting to cryopreservation and shipping, help biopharma companies manage more variables so they can focus on bringing their advanced therapy to patients. 

www.cardinalhealth.com 

Steven Kelly, President and CEO 

Oct. 12 | 10:45am | Oxford Biomedica Ballroom 

Philadelphia, PA 

(Private) 

In-person Presentation 

Carisma Therapeutics’ innovative approach has the potential to create life-changing cellular therapies for patients with cancer and other serious disorders. 

www.carismatx.com 

Fabian Gerlinghaus, CEO 

Oct. 13 | 10:30am | Oxford Biomedica Ballroo

San Francisco, CA 

(Private) 

In-Person Presentation 

Cellares is creating the future of cell therapy manufacturing and accelerating access to life-saving cell therapies. The company is developing the Cell Shuttle, a scalable solution to make cell therapies more affordable and widely available to patients in need by enabling end-to-end, automated manufacturing of these highly individualized treatments. While CAR T cell therapies have proven their near curative ability, the error prone, expensive, and lengthy manufacturing processes used today limit the number of cancer patients who can be treated. Cellares’ Cell Shuttle enables biopharmaceutical companies to meet patient demand and is capable of producing more than 10 patient doses in parallel. Importantly, this will reduce the per-patient manufacturing cost by up to 70 percent for most processes. The Cell Shuttle has the potential to accelerate therapy entry into the market by more than a year and could make developers’ cell therapies accessible to large patient populations quickly, safely, and cost-effectively. 

www.cellares.com 

Ramin Baghirzade, Ph.D., Global Head Commercial, Gene Therapy CDMO Services 

Oct. 12 | 2:00pm | Oxford Biomedica Ballroom 

Wilmington, MA 

(NASDAQ: CRL) 

In-person Presentation 

A CRO/CDMO with 75 years experience bringing therapeutics to market. Charles River now has a concept-to-commercial portfolio in cell and gene therapy enabling partners to reduce the number of complex external relationships which need to be managed. This serves to simplify the CRO/CDMO relationship and speed time to clinic and commercial. 

www.criver.com 

Rodolphe Clerval, CEO

Oct. 12 | 1:30pm | Oxford Biomedica Ballroom  

Paris, France 

(Private) 

In-person Presentation 

Coave Therapeutics develops gene therapy products in CNS and ophthalmology based on a unique AAV-ligand platform for improved delivery of payloads in deep structures of the brain and the eye. We have three preclinical CNS programs with a focus on enhancing the autophagy-lysosomal pathway in neurodegenerative diseases. Our legacy program, CTx-PDE6b for PDE6b mutated inherited retinal dystrophies, has achieved enrollment of a Phase I/II study with read-out expected in the next two years. 

www.coavetx.com 

Ryan Pruchnic, Managing Vice President  

Oct. 13 | 9:00am | Oxford Biomedica Ballroom  

Pittsburgh, PA 

(Private) 

In-person Presentation 

In 2002 Cook MyoSite was formed to guide the Cook Group organization into the expanding world of cellular technologies. Today, Cook MyoSite is dedicated to the development and subsequent commercialization of technology related to the collection, selection, and expansion of human skeletal muscle cells for the treatment of various disorders. 

www.cookmyosite.com 

Mark Sawicki, Ph.D., President and CEO

Oct. 12 | 5:30pm | Oxford Biomedica Ballroom  

Brentwood, TN 

(NASDAQ: CYRX) 

In-person Presentation 

Cryoport Systems is a market leader in globally integrated temperature-controlled supply chain support for critical, irreplaceable products and materials in the life sciences industry. We are already trusted to support over 600 active regenerative medicine clinical trials and leading commercial products worldwide – delivering unparalleled, integrated supply chain services for products, therapies, and treatments requiring unique, specialized temperature-controlled management. And as life sciences constantly evolve and advance, so do we. 

www.cryoport.com 

Timothy Schroeder, Chairman and CEO

Oct. 11 | 1:15pm | Oxford Biomedica Ballroom 

Covington, KY 

(Private) 

In-person Presentation 

CTI is a global research service provider delivering a full suite of services to the pharma and biotech industry. Offering manufacturing, regulatory development and strategy, clinical operations, research site, laboratory, and real-world evidence services, CTI is the only global research service provider with a history of success in regenerative medicine spanning decades, and the ability to support cell and gene therapy programs throughout the entire clinical development lifecycle. 

www.ctifacts.com 

Tony Ward, Chief Technology Officer

Oct. 12 | 11:45am | Oxford Biomedica Ballroom  

Carlsbad, CA 

(Private) 

In-person Presentation 

Curate Biosciences (formerly GPB Scientific) enables cell processing that radically changes the outcomes and economics of cell therapy manufacturing. The Curate® Cell Processing System applies Deterministic Cell Separation™ (DCS) technology through a benchtop system and single-use cassettes optimized for T cell isolation. With virtually no cell loss, and designed for both manufacturing and clinical application, the Curate System enables production of CAR T and other cell therapies beyond today’s limitations. The Curate System is an easy to use, functionally closed wash, separation, and concentration device designed primarily for integration within the cell therapy workflow, processing standard leukopaks within approximately 40 minutes. The system’s core DCS technology is uniquely gentle on cells, circumvents the need for cell labeling or Ficoll processing for WBC isolation, and enables substantially better recovery (>2-fold vs Ficoll-based commercial methods) and purity (99% Platelet and 92% RBC removal). 

www.curatebio.com 

Ross Macdonald, Ph.D., CEO 

Oct. 13 | 9:15am | Oxford Biomedica Ballroom 

Melbourne, Australia 

(ASX: CYP) 

In-person Presentation 

Cynata Therapeutics is a clinical-stage stem cell and regenerative medicine company developing a therapeutic mesenchymal stem cell (MSC) platform technology, Cymerus™, utilizing induced pluripotent stem cells (iPSCs) as starting material. A successful clinical trial in GvHD has been completed, meeting all safety and efficacy endpoints. The company is undertaking a Phase III clinical trial in osteoarthritis with further clinical trials underway in respiratory distress and diabetic foot ulcers. A Phase II clinical trial in GvHD is expected to commence in late 2022 following IND clearance from the FDA. The Cymerus technology addresses a critical shortcoming in conventional methods of production of MSCs for therapeutic use, which is the ability to achieve consistent, economic manufacture at commercial scale. Cymerus utilizes a proprietary process that is independent of donor limitations providing an off-the-shelf production platform for therapeutic product manufacture. 

www.cynata.com 

Kareem Reda, Chief Business Officer

Oct. 12 | 4:30pm | Oxford Biomedica Ballroom  

Waltham, MA 

(Private) 

In-person Presentation 

ElevateBio is a technology-driven company built to power transformative cell and gene therapies today and for decades to come. The company has assembled industry-leading talent, built state-of-the-art facilities, and integrated diverse technology platforms, including gene editing, induced pluripotent stem cells, and protein, vector, and cellular engineering, necessary to drive innovation and commercialization of cellular and genetic medicines. At the center of the business model is BaseCamp, a purpose-built facility offering process development, analytics, automation, and current GMP manufacturing capabilities. Through BaseCamp and its enabling technologies, ElevateBio enables a wide range of partners from global biopharmaceuticals to early-stage biotechs to bring innovation from concept through commercialization while also developing its own portfolio of cell and gene therapies. Our team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare. 

www.elevate.bio 

David McNinch, Chief Business Officer 

Oct. 11 | 9:45am | Oxford Biomedica Ballroom 

San Francisco, CA 

(Private) 

In-person Presentation 

Encoded is a biotech company that engineers gene therapies to tackle challenging targets and diseases. Our research platform, driven on machine learning, targets cell specificity for greater selectivity and potency across many tissue types. We endeavor to create gene therapies with optimal on and off target expression. Encoded has its own burgeoning pipeline focusing on pediatric CNS disorders and is open to partnering in new cell and tissue types. 

www.encoded.com 

Emile Nuwaysir, Ph.D., President and CEO 

Oct. 11 | 12:00pm | Oxford Biomedica Ballroom 

Boston, MA 

(Private) 

In-person Presentation 

Ensoma is a genomic medicines company developing one-time, in vivo treatments that precisely engineer any cell of the hematopoietic system to cure diseases from within. The company is poised to create a new therapeutic category addressing complex diseases such as immune-oncology and autoimmunity through multiplex cellular and genetic programming. Ensoma’s platform can uniquely address these disorders by leveraging its unprecedented payload capacity and exquisite specificity to harness powerful HSC biology. Ensoma also has the potential to leapfrog therapies in well-validated indications such as hemoglobinopathies and dramatically expand patient eligibility through its portable in vivo solution. Ensoma launched in 2021 with a $70 million Series A led by 5AM Ventures, with participation from a syndicate of top-tier investors. The company also announced a strategic collaboration with Takeda in rare disease, with the potential for $100 million in upfront and near-term research payments, and total milestones up to $1.25 billion. 

www.ensoma.com 

Bill Symonds, Ph.D., CEO

Oct. 12 | 5:30pm | UBC Ballroom  

Cambridge, MA 

(Private) 

In-person Presentation 

Enzyvant is a commercial-stage biotechnology company with a focus on regenerative medicines for rare diseases. The company’s first commercial product is FDA-approved RETHYMIC® (allogeneic processed thymus tissue-agdc), a tissue-based regenerative therapy for an ultra-rare and life-threatening pediatric immunodeficiency. Enzyvant has distinctive capabilities in expedited development of regenerative therapies for rare diseases. The company has obtained and leveraged multiple regulatory designations including Regenerative Medicine Advanced Therapy, Breakthrough, Fast Track, Rare Pediatric Disease, Orphan Drug, and Advanced Therapies Medicinal Product. Enzyvant is wholly owned by Sumitovant Biopharma (wholly owned by Sumitomo Dainippon Pharma Co.). 

www.enzyvant.com 

David Millette, CEO and Chief Operating Officer 

Oct. 11 | 11:00am | Oxford Biomedica Ballroom 

Montreal, Canada 

(Private) 

In-person Presentation 

ExCellThera is a clinical-stage cell expansion and engineering company delivering molecules and bioengineering solutions to expand and engineer various cell lines for use in novel one-time curative therapies for patients with hematologic malignancies and other diseases. ExCellThera’s most advanced technology, ECT-001 Cell Therapy, combines a proprietary molecule, UM171, and an optimized culture system. In pursuit of better treatments for patients, the company is building out its cell expansion and engineering platform, as well as supporting best-in-class clinical trials. 

www.excellthera.com 

Daniel Dornbusch, CEO 

Oct. 13 | 11:30am | Oxford Biomedica Ballroom 

San Francisco, CA 

(Private) 

In-person Presentation 

Excision BioTherapeutics is a biotechnology company developing CRISPR-based therapies as potential cures for viral infectious diseases. Excision unites next-generation CRISPR nucleases with a novel gene editing approach and has become the first and only company in history to cure HIV with a therapeutic in an animal model. EBT-101, the company’s lead program intended to cure HIV with a single IV infusion, is being evaluated in clinical trials. Excision’s pipeline includes one-time potential cures for herpes virus, JC virus, which causes PML, and hepatitis B virus. The company’s foundational technologies were developed in the laboratories of Dr. Kamel Khalili at Temple University and Dr. Jennifer Doudna at the University of California, Berkeley. 

www.excision.bio 

Tim Miller, Ph.D., CEO, President, and Co-Founder, Forge Biologics 

Oct. 12 | 9:15am | Oxford Biomedica Ballroom 

Grove City, OH  

(Private) 

In-person Presentation 

Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company. Forge’s mission is to enable access to life changing gene therapies and help bring them from idea to reality. Forge has a 200,000 square foot facility in Columbus, Ohio, the Hearth, to serve as its headquarters. The Hearth is a custom-designed cGMP facility dedicated to AAV vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.  

www.forgebiologics.com 

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

Oct. 13 | 9:30am | Oxford Biomedica Ballroom 

Evry, France 

(Private) 

In-person Presentation 

Genethon has been pioneering gene therapy for 30 years. Genethon’s teams have originated multiple gene therapy successes that are now bringing tremendous benefits for patients. Dedicated to tackling rare diseases, Genethon has accumulated world-class expertise in the identification of gene targets, product design, and manufacturing as well as preclinical and clinical development of gene therapies. With 200+ scientists and professionals, Genethon is pursuing its mission to bring life-changing therapies to patients suffering from rare genetic diseases and eradicate these pathologies. Genethon is a not-for-profit organization, created in 1990 by the French Muscular Dystrophy Association (AFM-Telethon) 

www.genethon.com 

Steven Engle, CEO

Oct. 11 | 5:30pm | Oxford Biomedica Ballroom  

Dallas, TX 

(Private) 

In-person Presentation 

Gradalis is a late-stage biotechnology company preparing to initiate a Phase III registration study in patients with ovarian cancer using a breakthrough immunotherapy platform. Based on multiple clinical studies, Gradalis has demonstrated that its immunotherapy platform decloaks the full repertoire of a patient’s tumor antigens, reactivates the immune system, and summons key effector cells to deliver a durable clinical response. When combined, these form a powerful trifecta of anti-cancer activities, potentially eliminating even the elusive metastatic cells, and as shown in Phase II clinical studies in ovarian cancer, a potential game changer in oncology. In addition, multiple clinical trials have demonstrated that Gradalis’ platform is better tolerated compared to standard cancer treatments. This is not a surprise since the platform uses the patient’s immune system operating with its natural state of balance rather than in an artificial overdrive as with some technologies 

www.gradalisinc.com 

Richard Kincaid, Executive and Chief Financial Officer

Oct. 11 | 5:00pm | UBC Ballroom  

Tokyo, Japan 

(TSE: 4593) 

In-person Presentation 

Healios K.K. is a biotechnology venture leading the field of regenerative medicine and cell therapy. It was founded in 2011 and listed on the Tokyo Stock Exchange in 2015. It aims to offer new therapies for patients suffering from diseases without effective treatment options. Healios K.K. is a pioneer in the development of regenerative medicines in Japan, and has established a proprietary, gene-edited universal donor induced pluripotent stem cell (iPSC) line to develop next generation regenerative treatments in immuno-oncology, ophthalmology, liver diseases, and other areas of severe unmet medical need. The company’s lead iPSC-derived cell therapy candidate is a next generation NK cell treatment for solid tumors that has been functionally enhanced through gene editing. 

www.healios.co.jp 

Sunyoung Kim, CEO

Oct. 11 | 2:45pm | UBC Ballroom  

Seoul, Korea 

(084990.KQ) 

In-person Presentation 

Introducing Helixmith’s ALS-Focused Program DART*: A novel, multimodal parallel approach exploiting HGF/c-Met Signaling. (*DART: Defeating ALS through Regenerative Therapeutics)Helixmith has established the ALS-Focused Program called DART. Our first platform capitalizes on the HGF/c-Met signaling pathway. Three modalities (plasmid DNA, AAV, and humanized antibodies) are under investigation in both clinical and pre-clinical stages. Helixmith recently completed Phase 1 and phase 2a studies using Plasmid DNA-encoding HGF modality (Engensis or VM202), and a small-scale phase 1/2 is planned in which plasmid DNA will be injected directly into the muscles involved in respiration, including the diaphragm. Having obtained encouraging data on survival rates, motor neuron function, and motor function in animal models, an optimized AAV vector modality has been developed for clinical studies. Additionally, a fully humanized antibody modality that binds to c-Met receptors and activates signaling has shown its effectiveness in a variety of disease models. AAV and antibody modalities will be tested in phase 1 studies between 2024 and 2026. In this presentation, we provide an overview of Helixmith’s DART program, its scientific rationale, status, and future direction.

www.helixmith.com 

Albert Seymour, Ph.D., President and CEO 

Oct. 11 | 3:15pm | UBC Ballroom 

Bedford, MA 

(NASDAQ: FIXX) 

In-person Presentation 

Homology Medicines is a clinical-stage genetic medicines company dedicated to transforming the lives of patients suffering from rare diseases by addressing the underlying cause of the disease. Homology’s clinical programs include HMI-102, an investigational gene therapy for adults with PKU; HMI-103, a gene editing candidate for PKU; and HMI-203, an investigational gene therapy for Hunter syndrome. Additional programs focus on metachromatic leukodystrophy, paroxysmal nocturnal hemoglobinuria, and other diseases. Homology’s proprietary platform is designed to utilize its family of 15 human hematopoietic stem cell-derived adeno-associated virus vectors to precisely and efficiently deliver genetic medicines in vivo through a gene therapy or nuclease-free gene editing modality, as well as to deliver one-time gene therapy to produce antibodies throughout the body through the GTx-mAb platform. Homology believes its initial clinical data and compelling preclinical data, scientific and product development expertise, and broad intellectual property position the company as a leader in genetic medicines. 

www.homologymedicines.com 

Sean Ainsworth, Chief Executive Officer and Chairman of the Board 

Oct. 11 | 2:15pm | Oxford Biomedica Ballroom   

Seattle, WA 

(Private) 

In-person Presentation 

Our mission at Immusoft is to develop novel cell therapies by programming B cells to produce therapeutic proteins that improve the lives of patients with rare diseases. 

www.immusoft.com 

Xiaofang Wang, M.D., Ph.D., Chief Technology Officer 

Oct. 12 | 9:30am | Oxford Biomedica Ballroom 

Farmington, CT 

(Private) 

In-person Presentation 

ImStem Biotechnology is an innovative biopharmaceutical company targeting areas in autoimmune, degenerative, and rare orphan diseases. With our state-of-the-art technology, we aspire to improve patient treatment outcomes and quality of health, as well as advance the knowledge of regenerative medicine globally. Our proprietary T-MSC technology (trophoblast-derived mesenchymal stem cells) has opened a new chapter in the field of pluripotent stem cell therapy. Through our proprietary differentiation method, we derive mesenchymal stem cells (MSCs) from human embryonic stem cells (hESCs) via a trophoblast-like intermediate stage. In 2020, T-MSC cell therapy was accepted by the FDA to proceed to clinical trial in multiple sclerosis patients, which we believe became the first hESC based allogenic stem cell therapy to be administered intravenously for this potential indication. Furthermore, our T-MSC technology has been granted 11 patents worldwide. In Q3 2021, ImStem initiated a Phase I clinical trial in the U.S. 

www.imstem.com 

Kenneth LaMontagne, Ph.D., Senior Vice President, Business Development

Oct. 11 | 5:15pm | Oxford Biomedica Ballroom 

New York, NY 

(NASDAQ: INAB) 

In-person Presentation 

In8Bio is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of gamma-delta T cell therapies for the treatment of cancer. Our platform employs allogeneic, autologous, and genetically modified approaches to develop novel cell therapies, which are designed to effectively identify and eradicate tumor cells. We are one of the most advanced gamma-delta T cell companies and the first to bring genetically modified gamma-delta T cells into the clinic. Our lead product candidates are currently in Phase I clinical trials: INB-200, for the treatment of newly diagnosed glioblastoma, or GBM, and INB-100, for the treatment of patients with leukemia that are undergoing hematopoietic stem cell transplantation, or HSCT. Our platform has yielded a broad portfolio of preclinical and discovery-stage programs, focused on addressing solid tumors. 

www.in8bio.com 

Zachary Robert, M.D., Ph.D., Chief Medical Officer

Oct. 12 | 5:45pm | UBC Ballroom  

Dallas, TX 

(NASDAQ: TIL) 

In-person Presentation 

Instil Bio is a clinical-stage biopharmaceutical company focused on developing an innovative cell therapy pipeline of autologous TIL therapies for the treatment of patients with cancer (solid tumors). Using an optimized and scalable manufacturing process, Instil is advancing its lead TIL product candidate, ITIL-168, for the treatment of advanced melanoma. TIL therapy is derived from lymphocytes extracted from a patient’s tumor tissue. These lymphocytes are expanded ex vivo to create a polyclonal T cell therapy that can recognize a broad set of antigens unique to each patient’s cancer. In 2022, Instil received clearance of an IND and plans to initiate a Phase I trial of ITIL-168 in additional indications targeting areas of unmet medical need, including non-small cell lung cancer (NSCLC), head and neck squamous cell carcinoma (HNSCC), and cervical cancer. Instil is also advancing ITIL-306, a genetically engineered Costimulatory Antigen Receptor TIL (CoStAR-TIL) therapy designed to enhance the activity of TILs.  

www.instilbio.com 

Derek Hicks, Chief Business Officer

Oct. 11 | 11:15am | UBC Ballroom  

Cambridge, MA 

(NASDAQ: NTLA) 

In-person Presentation 

Intellia Therapeutics is a leading clinical-stage genome editing company, focused on developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. We are building a full-spectrum genome editing company, by leveraging our modular platform, to advance in vivo and ex vivo therapies for diseases with high unmet need. For our in vivo programs to address genetic diseases, we use intravenously administered CRISPR as the therapy, in which our proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. For our ex vivo programs to address immuno-oncology and autoimmune diseases, we use CRISPR to create the therapy by engineering cells outside of the body. Our deep scientific, technical, and clinical development experience, along with our robust intellectual property portfolio, enables us to unlock broad therapeutic applications of CRISPR/Cas9 and related technologies to create new classes of genetic medicine. 

www.intelliatx.com

Karen Walker, Chief Technology Offiicer 

Oct. 11 | 11:15am | Oxford Biomedica Ballroom 

Emeryville, CA 

(Private) 

In-person Presentation 

Kyverna Therapeutics is a cell therapy company engineering a new class of therapies for autoimmune and inflammatory diseases. We combine advanced T cell engineering and synthetic biology to target the underpinnings of autoimmunity: chimeric antigen receptor T cells (CAR T) with properties uniquely suited for use in autoimmunity, and synReg T cells (synthetic regulatory T cells) to suppress pathogenic immune cells in specific tissues. Our lead program, KYV-101, is a novel clinical-stage anti-CD19 chimeric antigen receptor (CAR) T cell construct with properties uniquely suited for use in autoimmune diseases. It is designed to improve clinical tolerability, through a fully human anti-CD19 CAR with rationally assembled costimulatory domains. In a 20-patient Phase I/II study in oncology, expected anti-lymphoma activity was associated with a significant reduction of cytokines released. KYV-101 will enter clinical development in 2022. Additionally, Kyverna is developing an allogeneic version (KYV-201), using Intellia’s proprietary ex vivo CRISPR/Cas9-based allogeneic platform. 

www.kyvernatx.com 

Brian Culley, CEO 

Oct. 12 | 2:15pm | UBC Ballroom 

Carlsbad, CA 

(NASDAQ: LCTX) 

In-person Presentation 

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. Lineage develops and manufactures specialized, terminally differentiated human cells developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs include five allogeneic product candidates: (i) OpRegen, a retinal pigment epithelial cell therapy, being developed under a worldwide collaboration with Roche and Genentech, a member of the Roche Group; (ii) OPC1, an oligodendrocyte progenitor cell therapy for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced for immuno-oncology and infectious disease; (iv) ANP1, an auditory neuronal progenitor cell therapy; and (v) PNC1, a photoreceptor neural cell therapy. 

www.lineagecell.com 

Jeff Liter, President and CEO 

Oct. 12 | 11:00am | Oxford Biomedica Ballroom 

Minneapolis, MN 

(Private) 

In-person Presentation 

Luminary Therapeutics is a clinical-stage cell therapy company focused on combining advanced receptor design with superior cell engineering to overcome antigen escape and T cell dysfunction. Our novel ligand-based CAR is designed to bind three targets and is set to enter the clinic in Q3 2022. Additionally, we have an allogeneic platform based on gamma delta cells. Luminary was founded by the team from B-MoGen that achieved a successful 5x exit in only three years. We are seeking Series A financing with venture firms or strategic partners to support our first clinical trial and development of our disruptive U-Receptor that can modulate antigen specificity. 

www.luminarytx.com 

Patrick Lucy, President and CEO

Oct. 11 | 5:45pm | Oxford Biomedica Ballroom  

Hopkinton, MA 

(Private) 

In-person Presentation 

Lykan Bioscience is an innovative contract development and manufacturing services organization (CDMO) focused on cell-based therapies. With decades of biopharmaceutical industry experience, Lykan offers a full range of development and manufacturing services. The state-of-the-art, purpose-built facility offering eight independent manufacturing suites is uniquely designed to fully integrate cGMP principles and advanced software solutions to enable real-time testing and release of product. Located in Hopkinton, Massachusetts, 25 miles southwest of downtown Boston and in the proximity of four international airports, Lykan Bioscience is ideally situated to deliver life-saving cell therapy treatments to patients on behalf of their partners. 

www.lykanbio.com 

Julia Tarasenko, Senior Vice President, Global Commercial Operation, Sales, and Pricing 

Oct. 12 | 10:00am | Oxford Biomedica Ballroom 

Durham, NC 

(Private) 

In-person Presentation 

Marken is a wholly owned subsidiary of UPS and is a critical part of UPS Healthcare. With Polar Speed and Marken included, the UPS Healthcare division staffs 128 locations with 5,500 employees worldwide. Marken offers a state-of-the-art GMP-compliant depot network and logistic hubs for clinical drug product storage and distribution in 59 locations worldwide, while maintaining the leading position for direct-to-patient and home healthcare services, biological sample shipments, and biological kit production. Marken’s dedicated 2,250+ staff members manage 150,000 drug product and biological sample shipments every month at all temperature ranges in more than 220 countries and territories and have orchestrated 14,000+ home healthcare visits. Additional services such as cell and gene supply chain services, ancillary material sourcing, storage and distribution, shipment lane verification and qualifications, as well as GDP, regulatory, and compliance consultancy add to Marken’s unique position in the pharma and logistics industry. 

www.marken.com 

Cenk Sumen, Chief Technology Officer 

Oct. 12 | 4:45pm | Oxford Biomedica Ballroom 

Gaithersburg, MD 

(NASDAQ: MXCT) 

In-person Presentation 

MaxCyte® is a leading provider of cell-engineering platform technologies and is responsible for helping to bring next‐generation cell-based therapies to life. The company’s technology is employed by leading drug developers worldwide, including 20 of the top 25 global biopharmaceutical companies. Our Flow Electroporation® technology and next-generation ExPERT™ platform enable our partners to accelerate, streamline, and improve the drug development process from the early stages of research to commercialization. 

www.maxcyte.com 

Antonio Lee, Ph.D., CEO 

Oct. 13 | 9:45am | Oxford Biomedica Ballroom 

Vienna, VA 

(Private) 

In-person Presentation 

MEDIPOST is a Korean-based company founded in 2000 which became a publicly traded company in 2005, listed on KOSDAQ with a $350 million market cap and 300 employees. The U.S. subsidiary is located in Virginia. MEDIPOST operates the largest private cord blood bank, CELLTREE®, in South Korea with over 280,000 units of private cord blood bank under storage. Each year, over 20,000 private cord blood units are collected and stored at CELLTREE®. MEDIPOST’s research and development is focused on novel off-the-shelf, allogeneic cell therapeutics using human Umbilical Cord Blood-derived Mesenchymal Stromal Cells (hUCB-MSCs) with clinical-stage assets in the disease areas of osteoarthritis (OA), broncho-pulmonary dysplasia (BPD), and Alzheimer’s disease (AD). The company’s preclinical-stage pipeline includes Acute Respiratory Distress Syndrome (ARDS), alopecia, and diabetic nephropathy. In addition, MEDIPOST is exploring commercialization of an allogeneic umbilical cord blood-derived regulatory T cell therapeutics pipeline for unmet medical needs, through its subsidiary IMMUNIQUE Therapeutics. 

www.medi-post.com 

Jeff Vassallo, Senior Director, Clinical Operations

Oct. 12 | 5:00pm | Oxford Biomedica Ballroom  

Cincinnati, OH 

(NASDAQ: MEDP) 

In-person Presentation 

Medpace is a scientifically-driven, global, full service clinical contract research organization (CRO) providing Phase I-IV clinical development services to the biotechnology, pharmaceutical, and medical device industries. Medpace’s mission is to accelerate the global development of safe and effective medical therapeutics through its high-science and disciplined operating approach that leverages local regulatory and deep therapeutic expertise across all major areas including oncology, cardiology, metabolic disease, endocrinology, neuroscience, and anti-viral and anti-infective. 

www.medpace.com 

Nina Bauer, Ph.D., Global Head of Commercial 

Oct. 12 | 9:00am | Oxford Biomedica Ballroom 

Burlington, MA 

(OTCMKTS: MKKGY) 

In-person Presentation 

MilliporeSigma is the U.S. and Canada Life Science business of Merck KGaA, Darmstadt, Germany.  From solving your unique upstream and downstream challenges, to meeting urgent manufacturing timelines, and navigating uncertain regulatory guidelines, our 30+ years of global expertise integrating leading manufacturing technologies with process development, scale-up, product characterization and safety testing can help you accelerate your path to cures. Draw on our experience to bring your cell and gene therapies to life. 

www.sigmaaldrich.com

Anna Wijatyk, M.D., Chief Medical Officer

Oct. 12 | 2:30pm | Oxford Biomedica Ballroom  

Waltham, MA 

(Private) 

In-person Presentation 

Miltenyi Biomedicine is a newly established biopharmaceutical company that develops innovative cell and gene therapies. We are committed to bringing cancer treatments and regenerative therapies to patients worldwide. 

www.miltenyibiomedicine.com 

Jeff Ross, Ph.D., CEO 

Oct. 12 | 3:30pm | Oxford Biomedica Ballroom 

Eden Prairie, MN 

(NASDAQ: MIRO) 

In-person Presentation 

Miromatrix is a life sciences company pioneering a novel technology for bioengineering fully transplantable human organs to help save and improve patients’ lives. The company has developed a proprietary perfusion technology platform for bioengineering organs that it believes will efficiently scale to address the shortage of available human organs. Miromatrix’s initial development focus is on human livers and kidneys. 

www.miromatrix.com 

Yan Zhang, Ph.D., CEO

Oct. 11 | 1:30pm | Oxford Biomedica Ballroom  

San Francisco, CA 

(Private) 

In-person Presentation 

Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure, characterize, and predict our resistance and response to new therapies. Mission Bio’s multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others. The company’s Tapestri Platform gives researchers around the globe the power to interrogate genotype and immunophenotype together in individual cells, providing a comprehensive understanding of activity from a single sample. Tapestri is the only commercialized multi-omics platform capable of analyzing DNA and protein simultaneously at single-cell resolution. The Tapestri Platform is being utilized by customers at leading research centers, pharmaceutical, and diagnostics companies worldwide. 

www.missionbio.com 

Kristi Jones, Pharm.D., CEO

Oct. 11 | 5:00pm | Oxford Biomedica Ballroom  

Gaithersburg, MD 

(NASDAQ: NEXI) 

In-person Presentation 

NexImmune is a rapidly growing leader in the emerging field of antigen-directed immunotherapies. We are developing a novel technology that uses natural biology to orchestrate a targeted cell-mediated immune response across a broad range of diseases. We can customize our technology to intensify a targeted immune response to treat cancer or infectious disease. Conversely, we can also customize it to dampen an overactive immune response to treat autoimmune disorders. 

www.neximmune.com 

Dr. Gregory Block, Senior Vice President, Corporate Development 

Oct. 12 | 11:15am | Oxford Biomedica Ballroom 

Vancouver, Canada 

(Private) 

In-person Presentation 

Notch Therapeutics is a biotechnology company developing renewable, induced pluripotent stem cell (iPSC)-derived immunotherapies, with an initial focus on cancer where cell therapies offer transformational benefits over existing standards of care. Notch is working to maximize the benefit of cell therapies through a proprietary T cell-production platform which combines sophisticated product design with commercial-compatible processes to enhance patient outcomes. Notch’s iPSC-based technology platform allows for precision control of notch signaling, which removes several critical limiting factors in the development of cell therapies, delivering the ability to design and manufacture a uniform and unlimited supply of therapeutic T cells. 

www.notchtx.com 

Shankar Musunuri, Ph.D., Co-founder, Chairman and CEO

Oct. 12 | 2:00pm | UBC Ballroom  

Malvern, PA 

(NASDAQ: OCGN) 

In-person Presentation 

Ocugen is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologicals, and vaccines that improve health and offer hope for people and global communities. We are making an impact through courageous innovation, taking science in new directions in service of patients. Our breakthrough modifier gene therapy platform has the potential to treat multiple diseases with one drug and we are advancing research in other therapeutic areas to offer new options for people with unmet medical needs. 

www.ocugen.com 

Leslie Meltzer, Ph.D., Chief Medical Officer 

Oct. 12 | 4:15pm | UBC Ballroom 

London, UK 

(NASDAQ: ORTX) 

In-person Presentation 

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing, and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment. In 2018, the company acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard is advancing a pipeline spanning preclinical, clinical, and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families, and society and current treatment options are limited or do not exist. 

www.orchard-tx.com 

Kevin Caldwell, Co-founder and CEO

Oct. 11 | 2:00pm | Oxford Biomedica Ballroom  

San Francisco, CA 

(Private) 

In-person Presentation 

Ossium Health has developed a proprietary technology to recover bone marrow stem cells from deceased organ donors. With this platform, we treat hematologic malignancies: Ossium is working to enable transplants for the thousands of patients each year who die because they cannot find a matched donor. The FDA has already cleared Ossium’s product for market use, and we are doing a single-arm study with Be The Match to enter the registry. This platform also prevents organ rejection: the lifelong immunosuppression regimens after organ transplant cause chronic infections and poor QoL. By providing infusions of bone marrow stem cells from organ donors into the same patients who receive their organs, Ossium’s technology enables transplant patients to live without immunosuppression. Finally, the platform treats diseases of inflammation: Ossium’s interferon-gamma primed mesenchymal cell product is engineered to regulate inflammation. This proprietary manufacturing platform enables on-demand delivery of optimized doses and positions the company well to access this growing market. 

www.ossiumhealth.com 

Brian Lee, Ph.D., President and CEO

Oct. 12 | 3:45pm | Oxford Biomedica Ballroom  

Camarillo, CA 

(Private) 

In-person Presentation 

PBS Biotech offers innovative single-use bioreactors as well as contract process development services for the global cell therapy market. Our unique Vertical-Wheel® bioreactor provides ideal fluid mixing conditions for sensitive human cells such as induced pluripotent stem cells grown as aggregates or mesenchymal stem cells grown on the surface of microcarriers. Optimized hydrodynamic conditions can also be consistently reproduced across various sizes of vertical-wheel bioreactors, enabling robust scale up of cell culture processes for clinical and commercial scale manufacturing. Furthermore, our bioprocess R&D team possesses unparalleled knowledge regarding cell culture process development, optimization, and scale up for a variety of cell types and applications. PBS Biotech is committed to providing scalable solutions and meeting customer requirements for all stages of cell therapy manufacturing, from R&D to clinical studies and eventually commercial production. 

www.pbsbiotech.com 

Brent Warner, President, Gene Therapy  

Oct. 12 | 5:00pm | UBC Ballroom  

San Diego, CA 

(NASDAQ: PSTX) 

In-person Presentation 

Poseida Therapeutics is a clinical-stage biopharmaceutical company dedicated to utilizing its proprietary gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure. The company has discovered and is developing a broad portfolio of product candidates in a variety of indications based on its core proprietary platforms, including its non-viral piggyBac DNA Modification System, Cas-CLOVER site-specific gene editing system, and nanoparticle- and AAV-based gene delivery technologies. Poseida’s core platform technologies have utility, either alone or in combination, across many cell and gene therapeutic modalities and enable the company to engineer its portfolio of product candidates that are designed to overcome the primary limitations of current generation cell and gene therapeutics. 

www.poseida.com 

Cindy Atwell, Senior Vice President, Business Development

Oct. 11 | 5:15pm | UBC Ballroom 

Durham, NC 

(NASDAQ: DTIL) 

In-person Presentation  

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

www.precisionbiosciences.com 

Steven Yatomi-Clarke, CEO

Oct. 12 | 11:20am | Oxford Biomedica Ballroom  

Melbourne, Australia 

(ASX: PTX) 

In-person Presentation 

Prescient Therapeutics has two novel cell therapy platform technologies. OmniCAR is a universal immune receptor platform enabling controllable T cell activity and multi-antigen targeting with a single cell product. OmniCAR is based on technologies licensed from UPenn and Oxford. OmniCAR provides a method for single-vector, single cell product targeting multiple antigens simultaneously or sequentially, whilst allowing post infusion control and sustained T cell response. CellPryme-M is a minimal intervention manufacturing enhancement that produces more favorable cell phenotype, including 50% more memory T cells; doubling helper T cells and significantly greater chemokine receptor expression for greater tumor trafficking and penetrance. CellPryme-M doubled the performance of CAR T in tumor control and survival in immunocompetent mouse models with aggressive and resistant breast cancer. Cell therapies that could benefit from additional productivity in manufacturing or increased potency and durability in-vivo would be good candidates for CellPryme. 

www.ptxtherapeutics.com 

Matthew Klein, M.D., Chief Operating Officer

Oct. 11 | 12:00pm | UBC Ballroom  

South Plainfield, NJ 

(NASDAQ: PTCT) 

In-person Presentation 

PTC Therapeutics is a science-driven, global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC’s mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC’s strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. 

www.ptcbio.com 

Luke Henry, Chief Business Officer 

Oct. 11 | 11:30am | Oxford Biomedica Ballroom 

London, UK 

(Private) 

In-person Presentation 

Quell Therapeutics is leading the emerging field of using engineered T-regulatory (Treg) cell therapies that aim to harness, direct, and optimize their immune suppressive properties to address serious medical conditions driven by the immune system. Quell is leveraging its pioneering phenotype lock technology, unique multi-modular engineering platform, and integrated manufacturing capabilities to design and develop a pipeline of highly engineered Treg cell therapies with greater potential for persistence, potency, and stability than earlier generations of Treg cell therapy approaches. Quell’s lead clinical candidate QEL-001 is being developed to induce operational tolerance following liver transplantation, with the potential to protect the post-transplant liver without the need for chronic immunosuppressive medications. Quell is also advancing additional programs in neuroinflammatory and autoimmune diseases. 

www.quell-tx.com 

Peter Francis, Chief Scientific and Medical Officer  

Oct. 12 | 1:15pm | Oxford Biomedica Ballroom 

San Francisco, CA 

(Private) 

In-person Presentation 

Ray Therapeutics is a late-stage preclinical biotechnology company pioneering next generation optogenetic therapies for vision restoration to individuals with blindness. The company is based in the San Francisco Bay Area. Ray Therapeutics recently received a translational grant from CIRM which will accelerate progress towards a clinical trial of RAY-001, an intravitreally-delivered AAV-based gene therapy carrying our proprietary highly sensitive optogenetic transgene. Our preclinical studies have shown best-in-class vision restoration in animal models pointing to potentially transformative vision improvement in patients. The initial indication for our lead product is retinitis pigmentosa, a heterogeneous group of genetic disorders that cause near-or-complete blindness for most patients. No effective treatment is available to restore vision once the retina has degenerated. Unlike current RP gene therapies in development, which are targeted to specific genetic mutations or individuals with remaining photoreceptors that only address a small patient population, RAY-001 is mutation-independent. 

www.raytherapeutics.com 

Jane Lebkowski, Ph.D., President 

Oct. 12 | 2:00pm | Oxford Biomedica Ballroom 

Menlo Park, CA 

(Private) 

In-person Presentation 

Regenerative Patch Technologies (RPT) is a biotechnology company focused on the development of cell-based implant technology for the treatment of retinal diseases. RPT has developed a composite subretinal implant, termed the California Project to Cure Blindness-Retinal Pigment Epithelium 1 (CPCB-RPE1), consisting of a polarized monolayer of human embryonic stem cell-derived RPE (hESC-RPE) on an ultrathin, synthetic parylene substrate designed to mimic Bruch’s membrane. The company has successfully filed an IND on the product and completed enrollment and three-year follow-up of subjects in a Phase I/IIa clinical trial with advanced dry age-related macular degeneration. RPT is actively planning for a Phase IIb clinical trial, the design of which has been cleared by FDA. 

www.regenerativepatch.com 

Nina Hunter, Ph.D., Vice President, Corporate Strategy 

Oct. 12 | 1:45pm | UBC Ballroom 

Rockville, MD 

(NASDAQ: RGNX) 

In-person Presentation 

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a “5x’25” strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage trials or commercial products by 2025. 

www.regenxbio.com 

Noah Davidsohn, Ph.D., Chief Scientific Officer 

Oct. 11 | 2:45pm | Oxford Biomedica Ballroom 

San Diego, CA 

(Private) 

In-person Presentation 

Rejuvenate Bio is reversing aging and eliminating age related disease. Aging is caused by a change in gene expression in the body that manifests itself in age related disease. Aging represents a $1.5T opportunity in the USA healthcare system, which has unsustainably quadrupled spending on healthcare as a % of GDP since 1960. Rejuvenate Bio utilizes proprietary gene therapy, targets, and tools to target these changes in gene expression. Its first efforts have demonstrated that re-regulating even two genes can simultaneously treat multiple age-related diseases. Rejuvenate Bio is also developing the key targets, delivery, control and manufacturing technologies necessary to make full age reversal possible through Epigenetic Reprogramming. In 2023, Rejuvenate Bio will launch its first human clinical trial and bring its first product to market for the treatment of heart failure in dogs through a partnership with Phibro, a large animal health company. 

www.Rejuvenatebio.com 

Raj Prabhakar, Senior Vice President and Chief Business Officer 

Oct. 11 | 2:30pm | UBC Ballroom 

New York, NY 

(NASDAQ: RCKT) 

In-person Presentation 

Rocket Pharmaceuticals is a clinical-stage multi-platform gene therapy company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. 

www.rocketpharma.com 

Timothy Lu, M.D., Ph.D., Co-founder and CEO

Oct. 11 | 5:30pm | UBC Ballroom  

San Francisco, CA 

(NASDAQ: SNTI) 

In-person Presentation 

Senti Biosciences’ mission is to create a new generation of smarter medicines that outmaneuver complex diseases using novel and unprecedented approaches. To accomplish this, we are building a synthetic biology platform that may enable us to program next-generation cell and gene therapies with what we refer to as Gene Circuits. These Gene Circuits, which are created from novel and proprietary combinations of DNA sequences, are designed to reprogram cells with biological logic to sense inputs, compute decisions, and respond to their cellular environments. Our synthetic biology platform utilizes off-the-shelf CAR-NK cells, outfitted with these Gene Circuit technologies, to target particularly challenging oncology indications. Our lead programs include SENTI-202, a Logic Gated OR+NOT off-the-shelf CAR-NK cell therapy designed to target and eliminate acute myeloid leukemia (AML) cells while sparing the healthy bone marrow, and SENTI-301, a multi-armed off-the-shelf CAR-NK cell therapy designed for the treatment of Hepatocellular Carcinoma (HCC). 

www.sentibio.com 

Philip Toleikis, Ph.D., President and CEO

Oct. 11 | 3:00pm | UBC Ballroom  

Ontario, Canada 

(TSXV: SVA) 

In-person Presentation 

Sernova is a clinical-stage biotechnology company developing regenerative therapeutic technologies to deliver a functional cure for chronic diseases, including insulin-dependent diabetes, thyroid disease, and rare diseases like Hemophilia A. An ongoing Phase I/II study at the University of Chicago using Sernova’s Cell Pouch implantable device and human donor islets has provided positive interim data. The first two subjects in the study have achieved long term insulin independence. To provide an unlimited supply of cells to treat type 1 diabetes, Sernova and Evotec recently entered into an option and licensing partnership developing an off-the-shelf iPSC-based beta cell replacement therapy. 

www.sernova.com

Rogerio Vivaldi, M.D., President and CEO

Oct. 11 | 1:45pm | Oxford Biomedica Ballroom  

Cambridge, MA
(NASDAQ: SGTX) 

In-person Presentation 

Sigilon Therapeutics seeks to develop functional cures for patients with a broad range of chronic diseases by harnessing the power of the human cell through its Shielded Living Therapeutics™ platform. Sigilon’s product candidates are non-viral engineered cell-based therapies designed to produce crucial proteins, enzymes, or other therapeutic molecules needed by patients living with chronic diseases such as lysosomal diseases and diabetes. The engineered cells are encapsulated by Sigilon’s Afibromer™ biomaterials matrix, which is designed to shield them from immune rejection. Sigilon was founded by Flagship Pioneering in conjunction with Daniel Anderson and Robert Langer of the Massachusetts Institute of Technology. 

www.sigilon.com 

Stephane Boissel, President and CEO

Oct. 12 | 2:15pm | Oxford Biomedica Ballroom  

Paris, France 

(Private) 

In-person Presentation 

SparingVision is a genomic medicines company with a mission to translate pioneering science into vision saving treatments. Leveraging its unparalleled understanding of retinal diseases, SparingVision has built the world’s most compelling portfolio of synergistic cutting-edge gene therapy and genome editing treatments for inherited retinal diseases (IRDs). Both of its most advanced products, SPVN06 and SPVN20 look to go beyond single gene correction therapies to deliver new mutation agnostic treatments for Retinitis Pigmentosa (RP), a group of IRDs which are the leading cause of blindness globally. The company also has a strategic collaboration with Intellia Therapeutics to develop novel genome editing-based treatments for ocular diseases utilizing CRISPR-Cas9 technology. 

www.sparingvision.com 

Stephen Gunstream, President and CEO

Oct. 12 | 5:45pm | Oxford Biomedica Ballroom  

Hollister, CA 

(NASDAQ: TKNO) 

In-person Presentation 

Teknova expedites breakthroughs in modern medicine by providing critical reagents for the production of molecular diagnostics, novel vaccines, and next-generation therapies. With a focus on quality, speed, and service, we manufacture custom solutions that are used at every stage of the product development workflow, from basic research to product commercialization. To meet the special demands of custom manufacturing, we built our production facilities with flexibility in mind, allowing us to quickly adapt our production setup to satisfy unique requests. Our R&D team, which focuses on increasing efficiency in gene therapy bioproduction, works with customers to define their needs, offering expertise from experimental design to guidance on manufacturing of custom reagents. With over 25 years of formulation, dispense, and QC expertise, we can deliver custom research and GMP products with exceptionally fast turnaround times, in formats that allow seamless integration into customer-specific workflows.  

www.teknova.com 

Faraz Ali, CEO

Oct. 11 | 11:45am | UBC Ballroom  

San Francisco, CA 

(NASDAQ: TYNA) 

In-person Presentation 

Tenaya Therapeutics is a biotechnology company committed to a bold mission: to discover, develop, and deliver curative therapies that address the underlying drivers of heart disease. Founded by leading cardiovascular scientists from Gladstone Institutes and the University of Texas Southwestern Medical Center, Tenaya is developing therapies for rare genetic cardiovascular disorders, as well as for more prevalent heart conditions, through three distinct but interrelated product platforms: gene therapy, cellular regeneration, and precision medicine. 

www.tenayatherapeutics.com 

Delara Motlagh, Ph.D., General Manager, Cell Therapy Technologies

Oct. 12 | 5:15pm | Oxford Biomedica Ballroom  

Lakewood, CO 

(NASDAQ: TRUMF) 

In-person Presentation 

Cell and gene therapy developers work with Terumo Blood and Cell Technologies when they want more than just our portfolio of automated devices. Not only do we enable unparalleled excellence in cell collections, but we also bring the expertise in automating and closing the manufacturing process of biological products. Our team of experts are dedicated to working alongside customers to help them achieve their goals from early-stage development to commercialization. 

www.terumobct.com 

Matthew Lakelin, Ph.D., Co-founder and Vice President, Scientific Affairs and Product Development

Oct. 13 | 10:15am | Oxford Biomedica Ballroom 

Cardiff, UK 

(Private) 

In-person Presentation 

TrakCel is the first provider of integrated cellular orchestration solutions for the precise management, control, and tracking of cell and gene therapy products. With the largest number of live deployments supporting the broadest number of therapy classes, TrakCel has built a deep understanding of the unique challenges faced by advanced therapy developers globally. OCELLOS by TrakCel is a powerful, integrated cellular orchestration solution for the precise management, control, and tracking of advanced therapy supply chains, patient by patient. OCELLOS supports autologous, allogeneic, and personalized cancer vaccine therapies at all clinical and commercial stages, whilst brand new OCELLOS Lite has been developed for quick deployment for early-stage clinical therapies. The platforms help to establish and safeguard the therapy chain of identity and chain of custody whilst generating reportable, electronic audit trails in real-time. Dashboards unite therapy developers with healthcare providers and supply chain partners, sharing the information needed to guide decisions. 

www.trakcel.com 

Richard Porter, Ph.D., Chief Business Officer 

Oct. 11 | 2:15pm | UBC Ballroom 

Lexington, MA 

(NASDAQ: QURE) 

In-person Presentation 

uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe diseases. 

www.uniqure.com 

Barbara Sanders, Ph.D., Vice President, Vector Development

Oct. 13 | 11:45am | Oxford Biomedica Ballroom 

Amsterdam, Netherlands  

(Private) 

In-person Presentation 

VectorY combines the therapeutic potential of antibodies and gene therapy to develop long-lasting therapeutic solutions for large disease areas of high unmet medical need. Founded in August 2020, and based in the Amsterdam Science Park, VectorY is a fully integrated gene therapy company focused on the development of innovative therapeutics such as vectorized antibodies for both CNS and somatic disorders, with a special focus on muscle diseases.

www.vectorytx.com

Petter Bjorquist, Ph.D., CEO 

Oct. 12 | 4:00pm | Oxford Biomedica Ballroom 

Gothenburg, Sweden 

(Private) 

In-person Presentation 

VERIGRAFT is a Swedish biotechnology company run by experienced entrepreneurs and mastering a unique, breakthrough technology in the field of advanced therapies and regenerative medicine. We make transplantation possible without the severe risks of immunosuppression, and develop advanced therapies and tissue engineered products that will be able to help millions of patients with serious diseases. Our first product, the Personalized Tissue Engineered Vein (P-TEV) is a human vein segment graft for use in transplantation to replace a defective or missing part of a patient’s vein. A P-TEV with functioning valves is implanted to replace a non-functioning venous valve in the femoral vein of a patient suffering from severe chronic venous insufficiency (CVI). The first-in-man clinical trial is ongoing, and results are very positive. CVI is a multi-billion USD market and these patients do not receive any curative treatment today. 

www.verigraft.com 

Gitesh Dubal, Executive Vice President and Chief Marketing Officer

Oct. 13 | 11:00am | Oxford Biomedica Ballroom  

Milwaukee, WI 

(Private) 

In-person Presentation 

Versiti is proud to support clinical research through seamlessly integrated clinical trial capabilities, Leukopak and other human blood product provision, and central laboratory services. With over 70 years of industry-leading blood health experience, our depth of expertise in blood collection, laboratory medicine, logistics, and clinical research lends unparalleled support for trial and research partners, every step of the way. 

www.versiticlinicaltrials.org 

Luisa Amoedo, Chief Operating Officer

Oct. 11 | 3:15pm | Oxford Biomedica Ballroom 

San Sebastian, Spain 

(Private) 

In-person Presentation 

Viralgen is a CDMO born as a joint venture between AskBio and Columbus Venture Partners, combining decades of technology and drug development experience in multiple platforms to support best-in-class service offerings to the gene therapy market. We specialize in the production of rAAV viral vectors. Our optimized facility in San Sebastian, Spain maximizes throughput and efficiency of our proprietary Pro10™ suspension manufacturing platform. This enables industry-leading scalability, reproducibility, and speed to market. Through our superior technology platform, we deliver industry-leading titers and cGMP-certified quality for all AAV serotypes to our client partners, optimize the cost-of-goods and accelerate clinical development and commercialization of life-saving genetic medicines. 

www.viralgenvc.com 

Robert Ang, President and CEO

Oct. 12 | 5:15pm | UBC Ballroom  

Cambridge, MA 

(NASDAQ: VOR) 

In-person Presentation 

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 

Dr. Jason King, Associate Director Business Development 

Oct. 11 | 3:00pm | Oxford Biomedica Ballroom  

Philadelphia, PA 

(Private) 

In-person Presentation 

WuXi Advanced Therapies, a global Contract Testing Development and Manufacturing Organization (CTDMO), is the advanced therapies business unit of WuXi AppTec and offers integrated platforms to transform the discovery, development, testing, manufacturing, and commercialization of cell and gene therapies for customers worldwide. WuXi ATU’s technologies, automation platforms, and service solutions advance preclinical research and accelerate the timeline to GMP manufacture, while integrated GMP manufacturing and testing platforms reduce time to market, maintaining high titres, high levels of quality assurance, and full regulatory compliance. Ultimately, WuXi ATU’s complete end-to-end solutions support pioneering companies to deliver breakthrough cell and gene therapies to the patients who need them.  

www.advancedtherapies.com 

Evy Lundgren Åkerlund, Ph.D., CEO 

Oct. 13 | 10:00am | Oxford Biomedica Ballroom 

Lund, Sweden 

(NASDAQ: XINT) 

In-person Presentation 

Xintela is a clinical-stage biopharmaceutical company developing allogeneic mesenchymal stem cell (MSC)-based therapies with a current focus on osteoarthritis (OA) and difficult-to-heal venous leg ulcers (VLU). The company’s proprietary product platform XSTEM® is based on integrin α10β1-selected MSCs which assures homogenous and high quality MSC preparations and consistency between donors. XSTEM is manufactured in Xintela’s own GMP-approved facility. Xintela has started a Phase I/IIa clinical study with XSTEM for the treatment of knee OA and will start a Phase I/IIa clinical study for the treatment of VLU in Q3 2022. The goal is to show that XSTEM is safe and to investigate preliminary efficacy. Preclinical studies in large animals have demonstrated both safety and regenerative properties of integrin α10β1-selected MSCs and support the disease-modifying effect of XSTEM. Xintela’s current XSTEM pipeline also includes treatment of ARDS as well as MSC products for the treatment of degenerative joint diseases in animals. 

www.xintela.se