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Adverum Biotechnologies, Inc., a clinical-stage company, develops gene therapy product candidates to treat ocular diseases. Its lead product candidate is ixoberogene soroparvovec (ADVM-022), a single intravitreal injection gene therapy candidate used for the treatment of patients with wet age-related macular degeneration and diabetic macular edema which is in phase 2 clinical trials. The company is developing ADVM-062 (AAV.7m8-L-opsin), a novel gene therapy product candidate for the treatment of blue cone monochromacy via a single IVT injection. Adverum Biotechnologies, Inc. has license and collaboration agreements with University of California; GenSight Biologics; Lexeo Therapeutics; and Virovek, Inc. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was incorporated in 2006 and is headquartered in Redwood City, California.

Agenus Inc., a clinical-stage biotechnology company, discovers and develops immuno-oncology products in the United States and internationally. The company offers Retrocyte Display, an antibody expression platform for the identification of fully human and humanized monoclonal antibodies; and display technologies. It develops QS-21 Stimulon adjuvant, a saponin-based vaccine adjuvant. The company also develops Balstilimab, an anti-PD-1 antagonist that has completed Phase II clinical trial to treat second line cervical cancer; AGEN1181, an antigen 4 (CTLA-4) blocking antibody that is in Phase 2 clinical trial for the treatment of pancreatic cancer and and melanoma; AGEN2373, a CD137 monospecific antibody that is in Phase 1b clinical trial; AGEN1423, a CD73/TGFß TRAP antibody; AGEN1571, an ILT2 monospecific antibody that is in Phase 1 clinical trial; and BMS-986442, a TIGIT bispecific antibodies. In addition, it develops INCAGN1876, a GITR agonist; INCAGN2390, a TIM-3 monospecific antibody; INCAGN2385, a LAG-3 monospecific antibody; MK-4830, a monospecific antibody targeting ILT4 that is in Phase 2 clinical trial; UGN-301, a zalifrelimab intravesical solution for the treatment of cancers of the urinary tract that is in a Phase 1 clinical trial; and AGEN1884, a first-generation anti-CTLA-4 monospecific antibody. The company operates under Agenus, MiNK, Prophage, Retrocyte Display, and Stimulon trademarks. It has collaborations with Bristol-Myers Squibb Company, Betta Pharmaceuticals Co., Ltd., Incyte Corporation, Merck Sharpe & Dohme, and Gilead Sciences, Inc. The company was formerly known as Antigenics Inc. and changed its name to Agenus Inc. in January 2011. Agenus Inc. was founded in 1994 and is headquartered in Lexington, Massachusetts.

AVROBIO, Inc., a clinical-stage gene therapy company, develops ex vivo lentiviral-based gene therapies to treat rare diseases following a single dose worldwide. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The company's pipeline includes AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of Gaucher disease type 1, as well as to treat Gaucher disease type 3; AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease; AVR-RD-04, which is in phase 1/2 clinical trial for the treatment of cystinosis; and AVR-RD-05 that is in preclinical stage for the treatment of Hunter syndrome. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Precision BioSciences, Inc., a clinical stage gene editing company, develops ex vivo allogeneic chimeric antigen receptor (CAR) T immunotherapies and in vivo therapies for genetic and infectious diseases in the United States. The company offers ARCUS, a genome editing platform to cure genetic disorders. It also provides Ex vivo Allogeneic CAR T Immunotherapy, a form of immunotherapy in which T cell, a specific type of immune cell is genetically engineered to recognize and kill cancer cells; PBCAR0191, an azercabtagene zapreleucel, for the treatment of acute lymphoblastic leukemia and B-cell precursor acute lymphoblastic leukemia; PBCAR19B, an anti-CD19 CAR T candidate built on the stealth cell platform utilizing a single-step gene edit to minimize the risk of chromosome abnormalities; and PBCAR269A, an investigational allogeneic CAR T cell candidate targeting B-cell maturation antigen for relapsed or refractory (R/R) multiple myeloma in combination with nirogacestat, a gamma secretase inhibitor. The company has development and commercial license agreements with Les Laboratoires Servier to develop allogeneic CAR T cell therapies for antigen targets, hematological cancer targets beyond CD19, and solid tumor targets; Tiziana Life Sciences to evaluate foralumab, a fully human anti-CD3 monoclonal antibody as a lymphodepleting agent for the potential treatment of cancers; iECURE, Inc. to develop ARCUS-based gene-insertion therapies; and Novartis Pharma AG to discover and develop in vivo gene editing products. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.

Prevail Therapeutics Inc., a gene therapy company, focuses on developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company's lead product candidate is PR001, which is in Phase I/II clinical trial for the treatment of Parkinson's disease with GBA1 mutation and neuronopathic Gaucher disease. It is also developing PR006 for the treatment of frontotemporal dementia with GRN mutation; and PR004 for the treatment of synucleinopathies. Prevail Therapeutics Inc. was founded in 2017 and is based in New York, New York.