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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.

Gritstone bio, Inc., a clinical-stage biotechnology company, engages in developing vaccine-based immunotherapy candidates against cancer and infectious diseases. Its primary product candidate is GRANITE, an individualized immunotherapy candidate, which is in Phase 2/3 clinical trials for the treatment of microsatellite stable colorectal cancers; and has completed Phase 1/2 clinical trials for treating solid tumors. The company is also developing SLATE, an off-the-shelf immunotherapy candidate, which is in Phase 2 clinical trials for the treatment of metastatic solid tumors. In addition, it develops CORAL, a COVID-19 vaccine program; and a therapeutic vaccine candidate that is in Phase 1 clinical trials designed to treat and cure human immunodeficiency virus (HIV) infection. Gritstone bio, Inc. has a strategic collaboration with bluebird bio, Inc.; collaboration agreement with Gilead Sciences, Inc.; and license agreement with Genevant Sciences GmbH. The company was formerly known as Gritstone Oncology, Inc. and changed its name to Gritstone bio, Inc. in May 2021. Gritstone bio, Inc. was incorporated in 2015 and is headquartered in Emeryville, California.

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.

Replimune Group, Inc., a clinical-stage biotechnology company, focuses on the development of oncolytic immunotherapies to treat cancer. The company's proprietary tumor-directed oncolytic immunotherapy product candidates are designed and intended to activate the immune system against cancer. Its lead product candidate is RP1, a selectively replicating version of HSV-1 that expresses GALV-GP R(-) and human GM-CSF, which is in Phase I/II clinical trials for a range of solid tumors; and that has completed Phase II clinical trials for treating cutaneous squamous cell carcinoma. The company is also developing RP2, which is in Phase I clinical trials to express an anti-CTLA-4 antibody-like protein to block the inhibition of the immune response otherwise caused by CTLA-4; and RP3 that is in Phase I clinical trial to express immune-activating proteins that stimulate T cells. Replimune Group, Inc. was founded in 2015 and is headquartered in Woburn, Massachusetts.

Scholar Rock Holding Corporation, a biopharmaceutical company, focuses on the discovery, development, and delivery of medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. The company develops Apitegromab, an inhibitor of the activation of myostatin that is in Phase 3 clinical trial for the treatment of spinal muscular atrophy; and SRK-181, which has completed Phase 1 clinical trials for the treatment of cancers that are resistant to checkpoint inhibitor therapies, such as anti-PD-1 or anti-PD-L1 antibody therapies. It is developing a pipeline of product candidates to deliver novel therapies to treat a range of serious diseases, including neuromuscular disorders, cardiometabolic disorders, cancer, fibrosis, and iron-restricted anemia. Scholar Rock Holding Corporation was founded in 2012 and is headquartered in Cambridge, Massachusetts.