List of Lentiviral Vector (LVV) Companies with Phase 1 Active Clinical Trial - 19

Founded in 2016, AffyImmune is a clinical-stage biotechnology company that optimizes CAR T cells for the treatment of solid tumors using our Tune & Track platform. AIC100, our first product, spares healthy cells through our unique affinity-tuning, enhancing its safety and efficacy. The initial indication for AIC100 is refractory thyroid cancer, which has a very poor prognosis and very few effective therapies. The antigen targeted by AIC100 is also overexpressed in a variety of additional cancers, including stomach, triple negative breast cancer and a number of others.

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/

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

At CERo, we are expanding the armamentarium of engineered cell therapies to create a whole new class of innovative medicines for oncology. Armed with an understanding of cellular clearance and molecular immunology, we engineer cells to engage new and complementary cell elimination pathways to enable deeper and more sustained clinical responses to maximize wellness.

Expression Therapeutics, Inc. is a fully integrated clinical stage gene and cell therapy company developing novel, potentially curative, therapeutics for hematology and oncology. The Company, based in Atlanta, Georgia, has a state-of-the-art 43,000 square foot manufacturing facility in West Chester, Ohio. Expression Therapeutics, Inc. also built a state-of-the-art scalable 43,000 square foot manufacturing facility in Cincinnati, Ohio.

Fate Therapeutics (Nasdaq: FATE) is a clinical-stage biopharmaceutical company dedicated to the development of next-generation cellular immunotherapies for cancer and immune disorders.

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.

Immusoft is a cell therapy company focused on developing novel therapies for rare diseases using sustained delivery of therapeutic proteins from a patient’s own cells. The company is developing a technology platform called Immune System Programming (ISP™), which modifies a patient’s B cells and instructs them to produce gene-encoded medicines. The B cells that are reprogrammed using ISP become miniature drug factories that are expected to survive in patients and provide therapeutic benefit for many years. The company is based in Seattle, WA.

Interius is an early-stage biotechnology company expanding the potential of cell and gene therapy by developing next-generation delivery technology based on inventions from the laboratory of Dr. Saar Gill at the University of Pennsylvania.

Legend Biotech Corporation, a clinical-stage biopharmaceutical company, engages in the discovery and development of novel cell therapies for oncology and other indications. Its lead product candidate, LCAR-B38M/JNJ-4528, is an autologous CAR-T cell therapy that targets the B-cell maturation antigen. The company is conducting multiple clinical trials to evaluate LCAR-B38M/JNJ-4528 as an earlier line of therapy for multiple myeloma (MM), as well as a comparison of the treatment with standard triplet therapy in Revlimid-refractory MM. It also has a broad portfolio of earlier-stage autologous product candidates targeting various cancers, including non-hodgkins lymphoma (NHL), acute myeloid leukemia, and T cell lymphoma. In addition, the company is developing an allogeneic CAR-T product candidate targeting CD20 for the treatment of NHL, which is currently in an investigator-initiated Phase 1 clinical trial in China. Further, it has various product candidates in early preclinical and clinical development for the treatment of solid tumors, as well as infectious diseases. The company was founded in 2014 and is based in Somerset, New Jersey. Legend Biotech Corporation is a subsidiary of Genscript Biotech Corporation.

Luminary is a clinical stage CAR-T therapy company with a novel allogeneic manufacturing platform where our CAR and or TCR development utilizes gamma delta cells. What makes our allogeneic therapeutics unique are that our final drug product includes the combinatory power of both the Vẟ1 and Vẟ2 gamma delta cells giving our therapies the power of both innate and adaptive cancer clearing power. We have multiple solid tumor targets under development with a proprietary signaling method to ensure T-Cell persistence. Additionally, our Ligand BAFF CAR for hematologic cancers is designed with 3 antigen receptors specifically designed to overcome antigen escape.

Minerva Biotechnologies is a clinical stage biopharmaceutical company focused on developing immunotherapies for solid tumor cancers and cellular therapies for regenerative medicine. NCT04020575 is Minerva’s first-in-human trial of huMNC2-CAR44, a CAR T that targets the MUC1* growth factor receptor, for the treatment of metastatic breast cancers, 95% of which express a MUC1*. We are now expanding the current clinical trial to include our second CAR-T product that greatly increases persistence. The Company is rapidly accelerating its pipeline that includes next-gen CAR-T products, a straight antibody therapeutic and a small molecule therapeutic which are both for the treatment of cancer metastasis. Our track record of scientific breakthroughs is a testament to our visionary team and our collaborative culture. Join us in changing the world.

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.

OneChain Immunotherapeutics is a spin-off Company from the Josep Carreras Leukemia Research Institute and ICREA, that aims to develop CAR-T candidates against different antigens as a treatment for hematological malignancies based on the know-how of Dr. Pablo Menéndez.

Outpace is creating cell therapies with curative potential through protein design and cellular engineering. With an initial focus on solid tumors, Outpace is developing technologies that overcome the barriers to efficacy and safety by programming cells to make the right decisions inside patients. Outpace’s suite of technologies drive persistent activity of the cell therapy (OutLast™), recruit a productive immune response in the tumor microenvironment (OutSmart™), and achieve the potency (OutSpacers™), specificity (Co-LOCKR™), and safety (EGFRopt™) necessary to unlock diverse cancer indications. Outpace’s internal and partnered pipeline approach is optimized for rapid clinical validation of its technologies across diverse cell types and manufacturing processes. At Outpace Bio, we believe that the highest performing teams include people from a wide variety of backgrounds and experiences who respectfully challenge each other. We are committed to building an open, diverse, and inclusive culture for all employees.

OXB (LSE: OXB) is a global quality and innovation-led contract development and manufacturing organisation (CDMO) in cell and gene therapy with a mission to enable its clients to deliver life changing therapies to patients around the world. One of the original pioneers in cell and gene therapy, OXB has 30 years of experience in viral vectors; the driving force behind the majority of cell and gene therapies. OXB collaborates with some of the world’s most innovative pharmaceutical and biotechnology companies, providing viral vector development and manufacturing expertise in lentivirus, adeno-associated virus (AAV), adenovirus and other viral vector types. OXB's world-class capabilities span from early-stage development to commercialisation. These capabilities are supported by robust quality-assurance systems, analytical methods and depth of regulatory expertise. OXB offers a vast number of unique technologies for viral vector manufacturing, including a 4th generation lentiviral vector system (the TetraVecta™ system), dual plasmid system for AAV production, suspension and perfusion process using process enhancers and stable producer and packaging cell lines.

ReiThera is a company devoted to developing and producing biopharmaceutical products based on gene delivery technologies doe advanced therapies, in order to prevent and treat several serious or life-threatening diseases.

Sana Biotechnology is focused on utilizing engineered cells as medicines for patients. The ability to modify genes and use cells as medicines will be one of the most important advances in healthcare over the next several decades. Sana is building differentiated capabilities across the spectrum of cell and gene therapy. Three aspirations drive Sana as we look to discover treatments for patients with poor outcomes or currently untreatable diseases. The first is the ability to repair and control the genes in any cell in the body. We are advancing novel delivery technologies with the goal of being able to deliver any payload to any cell in a specific, predictable, and repeatable manner, paving the way for next-generation in vivo gene therapy. Next is the ability to differentiate pluripotent stem cells ex vivo into immune-cloaked functional cells with the aspiration of being able to replace any missing or damaged cells in the body. Last is a belief we can enable broader access to our therapies through focusing on scalable manufacturing solutions, the cost of manufacturing, and aligning with key stakeholders. Sana launched in early 2019 and has over 350 employees in Seattle, Cambridge, and South San Francisco. Sana was founded with the long view – bringing together great people and the best technologies to deliver on the challenging promise of using engineered cells to meaningfully change the outcome of many human diseases.

Umoja is developing a new approach to cancer therapy that retools a patient’s immune system in vivo, enhancing the body’s natural capacity to fight cancer. We envision a world where all patients with cancer have access to the most advanced immunotherapies and are free from the burdens of traditional cancer therapies.