List of Lentiviral Vector (LVV) Companies with Phase 2 Active Clinical Trial - 28

Adaptimmune is a fully integrated cell therapy company, designed and built from the ground up with four U.K.- and U.S.-based biotechnology hub locations. Our comprehensive capabilities and teams include preclinical research, clinical development, translational sciences, autologous and allogeneic manufacturing, and in-house commercial and corporate operations. For more information about Adaptimmune, please visit www.adaptimmune.com. If interested in joining our fast growing team, click on the careers tab of this page.

At Adicet (Nasdaq: ACET), we are defined by our mission to deliver best-in-class gamma-delta T cell therapies for patients fighting autoimmune diseases and cancer. Every one of our team members, whether in the lab, manufacturing, or general business role, comes to work every day with the patient in mind. As pioneers in the cell therapy space, we are honored and humbled by the opportunity to put our talents to bear in developing transformative therapies to defeat autoimmune diseases and cancer. We believe that the best way to do this is through discovering and developing allogeneic gamma-delta T cell therapies, a novel type of cell therapy. Adicet has chosen the gamma-delta T cell because it: • Preferentially traffics to tissues and provides B cell depletion, potentially leading to an immune reset for patients with autoimmune diseases. • Has multiple ways to target and kill cancer cells including solid tumors. We also chose to use cells from donors (allogeneic) to enable our potential therapies to be readily available when needed (taken “off-the-shelf” or available to the patient on demand). We’re not just blazing new trails in cell therapy, we’re having fun along the way. With regular team gatherings, many shared meals, larger company events, and a consistent commitment to our values, we have a strong culture! We are proud to attract and retain some of the greatest talent in the biopharma and cell therapy space, and we’re consistently giving back to our employees. The Adicet benefits package includes generous financial compensation, comprehensive health plans, generous paid time-off, educational reimbursement, and access to many amenities in our state-of-the-art facility. To learn more or inquire about joining our team in Boston, MA or Redwood City, CA, please visit http://www.adicetbio.com

Affini-T is a leading precision immunotherapy company targeting core oncogenic driver mutations to develop potentially curative therapies for patients with solid tumors. Our differentiated cell therapy platforms harness state-of-the-art engineering, synthetic biology, and gene editing capabilities to target even the most devastating cancer-driving mutations, beginning with KRAS. We leverage these tools to optimize T cell functions and rewrite the rules of the solid tumor microenvironment, enabling the potential for sustained clinical outcomes in patients. Building on the world-class innovation inherent in our leadership team, founders and technologies, we are powered to develop transformational medicines that last.

Allogene Therapeutics, Inc., a clinical stage immuno-oncology company, develops and commercializes genetically engineered allogeneic T cell therapies for the treatment of cancer. The company is developing UCART19, an allogeneic chimeric antigen receptor (CAR) T cell product candidate, which is in Phase I clinical trials for the treatment of pediatric and adult patients with R/R CD19 positive B-cell ALL; ALLO-501, an anti-CD19 allogeneic CAR T cell product candidate that is in Phase I clinical trial for the treatment of R/R non-Hodgkin lymphoma; and ALLO-501A for the treatment R/R large B-cell lymphoma or transformed follicular lymphoma. It is also developing ALLO-715, an allogeneic CAR T cell product candidate that is in a Phase 1 clinical trial for treating R/R multiple myeloma; ALLO-819, an allogeneic CAR T cell product candidates for the treatment of acute myeloid leukemia; ALLO-647, an anti-CD52 monoclonal antibody; CD70 to treat renal cell cancer; and DLL3 for the treatment of small cell lung cancer and other aggressive neuroendocrine tumors. The company has license and collaboration agreements with Pfizer Inc.; Servier; Cellectis S.A.; and Notch Therapeutics Inc., as well as clinical trial collaboration agreement with SpringWorks Therapeutics, Inc. Allogene Therapeutics, Inc. also has a strategic collaboration agreement with The University of Texas MD Anderson Cancer Center for the preclinical and clinical investigation of AlloCAR T candidates across a portfolio of hematologic and solid tumors. The company was founded in 2017 and is headquartered in South San Francisco, California.

Altheia Science is a pioneering start-up founded by two world renowned Italian scientists, Prof. Alessandra Biffi, MD and Prof. Paolo Fiorina, MD, PhD, together with AurorA-TT. Altheia Science’s pipeline exploits pioneering therapeutic tools for autoimmune diseases and cancer. The modulation of PD-L1 expression at the molecular and protein level is key to devise advanced treatments. This can be achieved by first-in-class molecules controlling the PD-L1 pathway and/or by lentiviral vector-based engineering of patients’ hematopoietic stem cells. Altheia Science’s approach intends to drastically modify the natural history of diseases with high clinical impact in autoimmunity and cancer by modulating PD-L1 expression, achieving tangible and durable benefit for each patient.

American Gene Technologies® International Inc. (AGT™) is a private biotechnology company pursuing cures and treatments for infectious diseases, cancers, and monogenic disorders. AGT™'s emerging gene and cell therapeutics programs are designed to: ✅ Create immunotherapy for HIV disease with gene-modified T cells ✅ Activate innate T cell immunity capable of killing all tumor cells with its vector used in immuno-oncology ✅ Treat PKU by bringing a functional gene into cells that express a dysfunctional gene due to mutation ✅ Advance clinically to provide human proof of concept For over a decade, AGT™ has developed, tested, and banked thousands of lentiviral vectors that can be adapted to the specific needs of new target diseases. AGT™ has developed gene therapy for diseases using this rapid gene delivery platform. GENE THERAPIES AGT™’s platform allows it to pursue clinical “cures” for complex diseases. The company has developed patent-protected gene therapeutics that are breakthroughs in medicine. *** See our extensive award winning patents on our website *** HIV/AIDS Immunotherapy for HIV disease with gene-modified T cells IMMUNO-ONCOLOGY Activates innate T cell immunity capable of killing all tumor cells with its vector used in immuno-oncology PHENYLKETONURIA (PKU) Treats PKU by bringing a functional gene into cells that express a dysfunctional gene due to mutation AGT™'s Core Objectives ✔️ Leverage our lentivirus platform to create highly-effective targeted genetic medicines ✔️ Create solutions for patients across a spectrum of infectious diseases, cancers and inherited disorders ✔️ Collaborate with leading medical researchers from around the globe To learn more and get in touch with American Gene Technologies® Inc., contact us today at https://www.americangene.com/contact-us/

At Artiva, our mission is to deliver to cancer patients highly effective cellular immunotherapies that are also safe and immediately accessible. Towards this end, Artiva is advancing a pipeline of off-the-shelf, allogeneic NK cell therapies, including CAR-NK cell therapies, that leverage the company’s unique manufacturing and engineering capabilities. Artiva is headquartered in San Diego.

Beam Therapeutics Inc., a biotechnology company, engages in developing precision genetic medicines for patients suffering from serious diseases in the United States. The company is developing therapies for the development of sickle cell disease and beta-thalassemia; CAR-T cell therapies for pediatric T-cell acute lymphoblastic leukemia and pediatric acute myeloid leukemia; therapies for alpha-1 antitrypsin deficiency and glycogen storage disorder 1A; and therapies for ocular and central nervous system disorders. It has a research and clinical collaboration agreement with Magenta Therapeutics, Inc. Beam Therapeutics Inc. was founded in 2017 and is based in Cambridge, Massachusetts.

BlueSphere Bio was founded to unlock the potential of personalized T cell therapy for the treatment of cancer by harnessing the unique specificity of each patient’s own T cells for the particular molecular fingerprint of each patient’s own cancer through our novel TCXpress technology. Our proprietary TCXpress platform is an elegant, rapid approach that exploits the patient’s own immune defenses to attack their cancer specifically, and is designed to leave normal tissues untouched, thereby reducing harmful side effects. The platform and implementation allows us to deliver rapid, personalized T cell treatments for a variety of cancer indications.

Cabaletta Bio, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases. Its proprietary technology utilizes chimeric autoantibody receptor (CAAR) T cells that are designed to selectively bind and eliminate B cells, which produce disease-causing autoantibodies or pathogenic B cells. The company’s lead product candidate is DSG3-CAART, which is in Phase I clinical trial for the treatment of mucosal pemphigus vulgaris, an autoimmune blistering skin disease, and Hemophilia A with Factor VIII alloantibodies. Its product candidate pipeline also include MuSK-CAART, a preclinical stage product to treat a subset of patients with myasthenia gravis; FVIII-CAART, a discovery stage product to treat a subset of patients with Hemophilia A; and DSG3/1-CAART, a discovery stage product for the treatment of mucocutaneous pemphigus vulgaris. Cabaletta Bio, Inc. has a collaboration with the University of Pennsylvania; and research agreement with The Regents of the University of California. The company was formerly known as Tycho Therapeutics, Inc. and changed its name to Cabaletta Bio, Inc. in August 2018. Cabaletta Bio, Inc. was incorporated in 2017 and is headquartered in Philadelphia, Pennsylvania.

Carina Biotech An Australian clinical stage immunotherapy company established to research and develop chimeric antigen receptor T cell (CAR-T) therapies to treat solid cancers. Carina are working towards producing broad-spectrum CAR-T therapies that can be used to treat multiple solid cancers yet are patient-specific and result in little, if any, off-cancer damage. Using its proprietary platforms, Carina is also developing technologies to improve access to, and infiltration of, solid cancers, and to enhance CAR-T cell manufacturing. We are working to… 1. Expand the clinical indications for T cell therapies 2. Improve the commercial viability of T cell therapies by developing supporting technologies that make T cell therapies more effective or economically viable Headquartered in Adelaide, South Australia, Carina has strong T cell R&D capability across a network of research providers and collaborators including leading scientists at the UniSA, the Women's & Children's Hospital in Adelaide, the University of Adelaide, the Royal Prince Alfred Hospital in NSW and the Seattle Children's Hospital in the United States.

CARsgen is a biopharmaceutical company with operations in China and the U.S. focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors. The company has built an integrated cell therapy platform with in-house capabilities that span target discovery, antibody development, clinical trials, and commercial-scale manufacturing. CARsgen has internally developed novel technologies and a product pipeline with global rights to address major challenges of CAR T-cell therapies, such as improving the safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs. Our vision is to become a global biopharmaceutical leader that brings innovative and differentiated cell therapies to cancer patients worldwide and makes cancer curable.

Cellectis is a global clinical-stage biopharmaceutical company. Pioneers and innovators in our field, our mission is to develop innovative treatments for patients with unmet medical needs. With 25 years of expertise, we have the best-in-class gene editing platform focusing on immuno-oncology, and gene therapy. Through our efficient and precise TALEN® technology, we create allogeneic CAR-T cells capable of recognizing and combating cancer cells. Today, our three clinical programs target patients with B-cell acute lymphoblastic leukemia (B-ALL), non-Hodgkin lymphoma (NHL) and acute myeloid leukemia (AML). We are fully integrated and a leader in end-to-end gene editing, allogeneic CAR T-cell companies. With our in-house manufacturing, we control our gene and cell therapy process from start to finish with starting materials produced in Paris (France) and CAR-T therapy products created in Raleigh, NC (USA). We also have several ongoing strong collaborations, based on our TALEN® technology, with leading cell & gene therapy companies, including our recent partnership with AstraZeneca, to develop new product candidates in oncology, immunology, and treatment of rare diseases. At Cellectis, we are committed to a cure. Cellectis headquarters are in Paris, France, with additional locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more about us, visit our website: www.cellectis.com. To learn more about our community guidelines, visit: http://www.cellectis.com/en/social-media-guidelines/ Follow our other social media accounts: @cellectis on LinkedIn on X (formerly Twitter) . TALEN® is a registered trademark owned by the Cellectis Group.

At Editas Medicine, we are pioneering the possible. Our mission and commitment is to harness the power and potential of CRISPR gene editing to develop a robust pipeline of medicines for people living with serious diseases around the world. Our goal is to discover, develop, manufacture, and commercialize transformative, durable genomic medicines for many diseases.

Genenta (Nasdaq: GNTA) is a clinical stage immuno-oncology company developing a cell-based platform harnessing the power of hematopoietic stem cells to provide durable and safe treatments for solid tumors. Our platform is not tumor type nor target antigen restricted and provides sustained targeted expression of therapeutic payload(s) inside the tumor micro environment. Genenta’s lead product candidate, Temferon™, precisely targets the delivery of interferon-alpha to the tumor micro-environment, minimizing systemic toxicity while breaking tumor-induced immune tolerance. Our treatments are designed as one-time monotherapies, but with the additional potential, when used in combination, to significantly enhance the efficacy of other approved therapeutics.

Glycostem Therapeutics is a Dutch biotech company established in 2007. Glycostem Therapeutics has developed the world's first GMP compliant NK cell platform that is ready for industrial scale-up.

IN8bio, Inc. is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of gamma-delta T cell therapies for the treatment of cancer. Gamma-delta T cells are naturally occurring immune cells that embody properties of both the innate and adaptive immune systems, and can intrinsically differentiate between healthy and diseased tissue. IN8bio develops ex vivo-expanded and activated gamma-delta T cells based upon its deep expertise in gamma-delta T cell biology, proprietary genetic engineering, and cell-type specific manufacturing capabilities, which we refer to collectively as our DeltEx platform. IN8bio’s platform employs allogeneic, autologous, and genetically modified approaches to develop novel cell therapies, which are designed to effectively identify and eradicate tumor cells. IN8bio is currently the most clinically advanced gamma-delta T cell company and the first company to bring genetically modified gamma-delta T cells into the clinic. For more information about the Company and its programs, visit www.IN8bio.com.

Kyverna Therapeutics is a clinical-stage cell therapy company with the mission of engineering a new class of therapies for autoimmune and inflammatory diseases. The Kyverna therapeutic platform combines advanced T cell engineering and synthetic biology technologies to suppress and eliminate the autoreactive immune cells at the origin of autoimmune and inflammatory diseases. Kyverna’s pipeline includes KYV-101, a fully human, autologous chimeric antigen receptor (CAR) T-cell therapy with properties well suited for use in B cell-driven autoimmune diseases and KYV-201, a fully human, allogeneic CAR T-cell therapy. By offering more than one mechanism for taming autoimmunity, and with patients currently in treatment for multiple indications in the U.S. and Europe, Kyverna is positioned to act on its mission of transforming how autoimmune diseases are treated.

Lyell Immunopharma, Inc., a T cell reprogramming company, engages in developing T cell therapies for patients with solid tumors. The company develops therapies using technology platforms, such as Gen-R, an ex vivo genetic reprogramming technology to overcome T cell exhaustion; and Epi-R, an ex vivo epigenetic reprogramming technology to generate population of T cells with durable stemness. Its pipeline includes LYL797, a T cell product candidate for the treatment of non-small cell lung cancer and triple negative breast cancers; LYL845 for multiple solid tumors; and NY-ESO-1 for synovial sarcoma and other solid tumor indications. Lyell Immunopharma, Inc. was incorporated in 2018 and is headquartered in South San Francisco, California.

OUR MISSION: Deliver Innovation. Empower Discovery. Improve Life. For over 25 years, Mirus Bio has pioneered the development of transfection reagents and proprietary technologies for nucleic acid delivery applications. Our work is both technical and cutting-edge, and we promote a culture of creativity, responsibility, and respect. Our team members are engaged and motivated by the desire to support our customers and partners to achieve strong results. Mirus, together with Gamma Biosciences, is establishing the future of science and medicine by developing products that enable product developers within the cell and gene therapy space. As we approach a new era of addressing and conquering life-altering diseases, we will continue to expand our expertise and leadership in transfection to be integral in the future of advanced therapies.

Mustang Bio, Inc., a clinical-stage biopharmaceutical company, focuses on translating medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors, and rare genetic diseases. Its pipeline focuses on gene therapy programs for rare genetic disorders, chimeric antigen receptor (CAR) engineered T cell (CAR T) therapies for hematologic malignancies, and CAR T therapies for solid tumors. The company develop MB-107, a gene therapy program, potential curative treatment for X-linked severe combined immunodeficiency, a rare genetic immune system condition in which affected patients do not live beyond infancy without treatment. Its ex vivo lentiviral gene therapy is in two Phase 1/2 clinical trials.

Oncternal Therapeutics is a clinical-stage oncology company developing first-in-class, novel therapies that target a broad spectrum of cancers with large unmet medical need. By leveraging pathways absent or minimally expressed in normal cells or tissue, we aim to inhibit cancer cell growth while reducing potential harm to healthy cells. We are pursuing trials in chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), breast cancer, and Ewing sarcoma. Looking forward, we intend to rapidly broaden our pipeline and invest in the development of our innovative therapies across multiple cancers.

Rocket Pharmaceuticals (NASDAQ: RCKT), Ltd is a biotechnology company focused on seeking cures through molecular and gene therapy. We are developing first-in-class treatments for patients with rare and often devastating orphan diseases using a lentiviral-based and adeno-associated virus (AAV) therapy approach. We have developed rewarding partnerships with a number of leading international research institutions as well as with centers of manufacturing and cell processing expertise, while building an internal team to drive the research, development, clinical and regulatory programs. Ultimately we aim to launch and globally commercialize innovative gene therapies in disease areas with high unmet need.

Takara Bio Inc. is committed to improving the human condition through biotechnology. Starting as the biomedical business of Takara Shuzo, a fermentation and beverage company founded in 1925 (now Takara Holdings Company, Inc.), Takara Bio has focused on developing research tools for the life sciences, cell and gene therapy technologies, and nutraceuticals since its launch in 1967. The three business units of TAKARA BIO INC are: • Genetic Engineering Research - manufactures and sells research reagents and scientific instruments used by biotechnology researchers worldwide, as well as contract research services. • Agribio - produces and sells health food products whose functionality have been proven by biotechnology, and conducts a mushroom business based on technologies for large-scale mushroom production. • Gene Medicine - conducts clinical development projects to work toward commercializing cell and gene therapies centered on a highly efficient gene transduction method and a lymphocyte expansion-culture system, both using RetroNectin® reagent.

TC BioPharm is a publicly traded, clinical-stage cell therapy company developing advanced allogeneic CAR-T cell therapy products for the treatment of cancer, as well as developing gamma delta T cell therapies for the treatment of infectious disease. We have a growing team based in our Glasgow office, alongside a wide range of scientific advisors, and partner organisations.

At Triumvira Immunologics, Inc., our team is dedicated to a vision of finding a cure and providing new hope for patients with life-threatening disease. Co-founded in 2015 by licensing technology out of McMaster University and Bloom Burton & Co., Triumvira is working to achieve this vision by developing a proprietary T cell Antigen Coupler (TAC) technology, thought to be safer and more efficacious than current cancer treatments including chimeric antigen receptor (CAR) and engineered T cell receptor (TCR) therapies. We are an emergent US company with operations in the United States and Canada. We bring together talent from a wide range of disciplines including immunobiology, drug development, fundraising, commercialization and finance, with decades of experience across big pharma and small biotech, multiple industries and academia. We are passionate about science that pushes the limits, the patients that drive the heart of our work, and the wellness of our employees. We embrace flexibility. If you are looking for a career where you are encouraged to be innovative, challenge the status quo, and have some fun while doing it, Triumvira is the place for you. For more information about Triumvira career opportunities, please see our Careers web page at: https://triumvira.com/contact-us/careers/

We are a cell therapy company that combines a novel patient engineering approach with targeted therapies to provide a solution for patients suffering from hematological malignancies.

Vyriad is a biotechnology company that develops targeted genetic therapies using engineered viruses and viral vectors for oncolytic virotherapy and gene editing applications.