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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 hematopoietic stem cell gene therapies to treat rare diseases in the United States. 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 and 3; AVR-RD-01, which is in phase 2 clinical trials for the treatment of Fabry disease; and AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Harpoon Therapeutics, Inc., a clinical-stage immunotherapy company, engages in the development of a novel class of T cell engagers that harness the power of the body's immune system to treat patients suffering from cancer and other diseases in the United States. The company develops tri-specific T cell activating construct (TriTAC) product candidate, including HPN328, which is in Phase I/II clinical trials for the treatment of small cell lung cancer and other Delta-like canonical Notch ligand 3-expressing tumors; and HPN217 that is in Phase I clinical trials for the treatment of multiple myeloma. Its preclinical stage product is HPN601 for the treatment of multiple solid tumor indications. It has a discovery collaboration and license agreement with AbbVie Biotechnology Ltd. to develop and commercialize products that incorporate its proprietary TriTAC platform technology together with soluble T cell receptors. The company was incorporated in 2015 and is headquartered in South San Francisco, California. As of March 11, 2024, Harpoon Therapeutics, Inc. operates as a subsidiary of Merck Sharp & Dohme LLC.

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.