List of Lentiviral Vector (LVV) Companies with Phase 3 Active Clinical Trial - 18

Our name, 2seventy bio, reflects why we do what we do - TIME. Cancer rips time away, and our goal is to work at the maximum speed of translating human thought into action – 270 miles per hour – to give the people we serve more time. We are building the leading immuno-oncology cell therapy company, focused on discovering and developing new therapies that truly disrupt the cancer treatment landscape With a deep understanding of the human body’s immune response to tumor cells and how to translate cell therapies into practice, we’re applying this knowledge to deliver next generation cellular therapies that focus on a broad range of hematologic malignancies, including the first FDA-approved CAR T cell therapy for multiple myeloma, as well as solid tumors. Our research and development is focused on delivering therapies that are designed with the goal to “think” smarter and faster than the disease. Importantly, we remain focused on accomplishing these goals by staying genuine and authentic to our “why” and keeping our people and culture top of mind every day.

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/

Arcellx, Inc., a clinical-stage biotechnology company, engages in the development of various immunotherapies for patients with cancer and other incurable diseases. The company’s lead ddCAR product candidate is CART-ddBCMA, which is in phase 1 clinical trial for the treatment of patients with relapsed or refractory (r/r) multiple myeloma (MM). It is also developing ACLX-001, an immunotherapeutic combination composed of ARC-T cells and bi-valent SparX proteins targeting BCMA to treat r/r MM; ACLX-002 and ACLX-003 for treating r/r acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS); and other AML/MDS product candidates, as well as solid tumor programs. The company was formerly known as Encarta Therapeutics, Inc. and changed its name to Arcellx, Inc. in January 2016. Arcellx, Inc. was incorporated in 2014 and is headquartered in Gaithersburg, Maryland.

BASF Corporation is the largest affiliate of BASF SE and the second largest producer and marketer of chemicals and related products in North America. BASF creates chemistry for a sustainable future. They combine economic success with environmental protection and social responsibility.BASF Corporation operates more than 100 production and research and development sites throughout North America and operates Verbund sites in Geismar, Louisiana and Freeport, Texas. At Verbund sites, production plants, energy and material flows, logistics, and site infrastructure are all integrated.

BioNTech is a global next generation immunotherapy company aspiring to translate science into survival. Around 6.100 pioneers currently work with us, employing a wide array of computational discovery and therapeutic drug platforms for the rapid development of novel biopharmaceuticals. Our broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor (CAR) T cells, several protein-based therapeutics, including bispecific immune checkpoint modulators, targeted cancer antibodies and antibody-drug conjugate (ADC) therapeutics, as well as small molecules.   Based on our deep expertise in mRNA vaccine development and in-house manufacturing capabilities, we are developing multiple mRNA vaccine candidates for a range of infectious diseases alongside its diverse oncology pipeline. To learn more about us, please visit www.BioNTech.com. Our privacy statement can be found here:  https://www.biontech.com/int/en/home/data-privacy-statement.html Our imprint can be found: https://www.biontech.com/int/en/home/imprint.html

bluebird bio, Inc., a biotechnology company, engages in researching, developing, and commercializing of transformative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin for the treatment of ß-thalassemia and sickle cell disease; and Lenti-D for the treatment of cerebral adrenoleukodystrophy. The company’s product candidates in oncology include bb2121 and bb21217, which are chimeric antigen receptor T (CAR T) cell product candidates for the treatment of multiple myeloma. It has a strategic collaboration with Bristol-Myers Squibb to discover, develop, and commercialize disease-altering gene therapies in oncology; and Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize various immune cell therapies for cancer. The company also has collaborations with Medigene AG to discover T cell receptor (TCR) product candidates in the field of cancer; Gritstone Oncology, Inc. to discover TCR product candidates in the field of cancer; TC BioPharm Limited to research and develop gamma delta CAR T cells directed at hematologic and solid tumor targets; Forty Seven, Inc. to pursue clinical proof-of-concept for an antibody-based conditioning regimen; and Novo Nordisk A/S to develop in vivo genome editing treatments for genetic diseases, as well as clinical trial collaboration with Magenta Therapeutics. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was founded in 1992 and is headquartered in Cambridge, Massachusetts.

BrainStorm Cell Therapeutics Inc. (NASDAQ) develops autologous stem cell therapies for neurodegenerative disorders such as ALS, MS, and Parkinson’s disease. These diseases have limited treatment options and represent unmet medical needs. NurOwn® is BrainStorm’s proprietary process for differentiating autologous mesenchymal stem cells (MSC) into neurotrophic factor (NTF)-secreting cells, transplanted at or near the affected tissue site. Our technology converts MSCs into a living drug delivery system for NTFs. We have proof-of-concept in various animal models of neurodegenerative diseases, including Parkinson's, Huntington's, ALS, MS, and peripheral nerve injury. We have completed two single-arm clinical trials in ALS patients in Israel, demonstrating good safety and tolerability profiles and strong efficacy signals. We also finalized a Phase 3 randomized, double-blind, placebo-controlled clinical trial at multiple US sites, supported by a $16 million USD grant from the California Institute for Regenerative Medicine (CIRM) and another grant from the ALS Association. Currently, we are planning a Phase 3b clinical trial of NurOwn® in ALS under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA). For more information, visit www.brainstorm-cell.com

BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX: BCT) is a clinical-stage biotechnology company specializing in targeted immunotherapies for cancer. BriaCell achieved positive proof-of-concept in a Phase I/IIa clinical trial for Bria-IMT™ in patients with advanced breast cancer. BriaCell is conducting a Phase I/IIa clinical trial of Bria-IMT™ in combination with a PD-1 inhibitor, retifanlimab (provided by Incyte under a collaboration agreement). Additionally, BriaCell is developing Bria-OTS™, the first “off-the-shelf” personalized immunotherapy for advanced breast cancer.

Castle Creek Biosciences, Inc., a late-stage cell and gene therapy company, is using two lentiviral platforms to develop and commercialize therapies for genetic diseases.

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom.

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.

Immatics N.V., a clinical-stage biopharmaceutical company, focuses on the discovery and development of T cell receptor (TCR) based immunotherapies for the treatment of cancer in the United States. The company is developing targeted immunotherapies with a focus on treating solid tumors through two distinct therapeutic modalities, such as adoptive cell therapies (ACT) and antibody-like TCR Bispecifics. Its ACTengine product candidates include IMA201 that targets melanoma-associated antigen 4 or 8 in patients with solid tumors; IMA202 that targets melanoma-associated antigen 1 in patients with various solid tumors, including squamous non-small cell lung carcinoma and hepatocellular carcinoma; IMA203 that targets preferentially expressed antigen in melanoma in adult patients with relapsed and/or refractory solid tumors; and IMA204, an anti-tumor therapy that targets the malignant tumor cell. Its TCR Bispecifics product candidates include IMA401, a cancer testis antigen for the treatment of solid tumor; and IMA402 for the treatment of solid and hematological malignancies. The company also develops IMA101, a multi-target precision immunotherapy; and IMA301, an off-the-shelf ACT. It has a strategic collaboration agreement with GlaxoSmithKline plc to develop novel adoptive cell therapies targeting multiple cancer indications; MD Anderson Cancer Center to develop multiple T cell and TCR-based adoptive cellular therapies; Celgene Switzerland LLC to develop novel adoptive cell therapies targeting multiple cancers; Genmab A/S to develop T cell engaging bispecific immunotherapies targeting multiple cancer indications; Amgen Inc.; and MorphoSys to develop novel antibody-based therapies against various cancer antigens that are recognized by T cells. Immatics N.V. is headquartered in Tubingen, Germany.

Iovance Biotherapeutics (NASDAQ: IOVA) aims to be the global leader in innovating, developing and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer. We are adding to our talented team as we execute our commercial launch, advance our research and development efforts, and build our internal manufacturing capabilities. Iovance is headquartered in the San Francisco Bay Area. Our Iovance Cell Therapy Center (iCTC), a state-of-the-art manufacturing facility, is located in Philadelphia. We also have a research site in Tampa, Florida. Any information expressed by Iovance is subject to the risk factors and information on forward-looking statements contained in its filings with the Securities and Exchange Commission and available here: https://ir.iovance.com/sec-filings

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.

Orchard Therapeutics, a Kyowa Kirin company, is a global gene therapy leader focused on ending the devastation caused by genetic and other severe diseases 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 with a single treatment. Founded in 2015, Orchard’s roots go back to some of the first research and clinical developments involving HSC gene therapy. Our team has played a central role in the evolution of this technology from a promising scientific idea to a potentially life-transforming reality. Today, Orchard is advancing a pipeline of 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. For more information, please visit www.orchard-tx.com.

Sequirus is one the largest cell-based influenza vaccine producer in the world. The company also makes antivenoms, and Q fever vaccine.

Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $40 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, increasing productivity in their laboratories, improving patient health through diagnostics or the development and manufacture of life-changing therapies, we are here to support them. Our global team delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services, Patheon and PPD.