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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.

Aligos Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the development of novel therapeutics to address unmet medical needs in viral and liver diseases. Its drug candidate, ALG-055009, a small molecule THR-ß agonist that is in the Phase 2a clinical trial for the treatment of non-alcoholic steatohepatitis (NASH). The company also develops ALG-000184, a capsid assembly modulator, which is completed Phase 1b clinical trial to treat chronic hepatitis B (CHB); and ALG-125755, a siRNA drug candidate, which is in Phase I clinical trial for the treatment of CHB. In addition, it develops ALG-097558, which is in Phase 2 clinical trial for the treatment of coronavirus. The company has entered into license and research collaboration agreement with Merck to discover, research, optimize, and develop oligonucleotides directed against a NASH; license agreement with Emory University to provide hepatitis B virus capsid assembly modulator technology; license agreement with Luxna Biotech Co., Ltd. to develop and commercialize products containing oligonucleotides targeting hepatitis B virus genome; and research, licensing, and commercialization agreement with Katholieke Universiteit Leuven to develop coronavirus protease inhibitors. Aligos Therapeutics, Inc. was incorporated in 2018 and is headquartered in South San Francisco, California.

Cellectis S.A., a clinical stage biotechnological company, develops immuno-oncology products based on gene-edited T-cells that express chimeric antigen receptors to target and eradicate cancer cells. The company is developing UCART19, an allogeneic T-cell product candidate for the treatment of CD19-expressing hematologic malignancies, such as acute lymphoblastic leukemia; ALLO-501 and ALLO-501A to treat relapsed or refractory for non-hodgkin lymphoma (NHL); and ALLO-715 for the treatment of multiple myeloma. It also develops UCART22 to treat B-cell acute lymphoblastic leukemia; UCARTCS1 and ALLO-605 for the treatment of multiple myeloma; ALLO-316 for renal cell carcinoma; UCART123 for the treatment of acute myeloid leukemia; and UCART 20x22 for relapsed or refractory B-Cell NHL. The company has strategic alliances with Allogene Therapeutics, Inc. and Les Laboratoires Servier; research collaboration and exclusive license agreement with Iovance Biotherapeutics; and collaboration and license agreement with Cytovia, as well as a collaboration agreement with AstraZeneca to develop novel cell and gene therapy candidate products. Cellectis S.A. was founded in 1999 and is headquartered in Paris, France.

Inhibrx, Inc., a clinical-stage biopharmaceutical company, develops a pipeline of novel biologic therapeutic candidates. The company's therapeutic candidates include INBRX-109, a tetravalent therapeutic candidate targeting death receptor 5, which is in Phase 2 clinical trials to treat cancers, such as chondrosarcoma, mesothelioma, colorectal cancer, ewing sarcoma, and pancreatic adenocarcinoma; and INBRX-101, an alpha-1 antitrypsin (AAT)-Fc fusion protein therapeutic candidate, which is in Phase 1 clinical trials for use in the treatment of patients with AAT deficiency. It also develops INBRX-106, a hexavalent agonist of OX40 for a range of oncology indications. The company has license and collaboration agreements with 2seventy bio, Inc. and Bristol-Myers Squibb Company. Inhibrx, Inc. was incorporated in 2009 and is headquartered in La Jolla, California.

Krystal Biotech, Inc., a commercial-stage biotechnology company, discovers, develops, and commercializes genetic medicines for patients with rare diseases in the United States. It commercializes VYJUVEK (beremagene geperpavec-svdt, or B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB). The company also develops KB105, which is in Phase 1/2 clinical trials for treating patients with deficient autosomal recessive congenital ichthyosis; KB104 for treating netherton syndrome; KB407 that is in Phase 1 clinical trials for treating cystic fibrosis; KB707 that is in Phase 1 clinical trials for the treatment of anti-PD-1 relapsed/refractory; KB408, which is in Phase 1 clinical trials for treating Alpha-1 antitrypsin deficiency; and KB301 that is in Phase 2 clinical trials for treating aesthetic skin conditions, as well as in open label study with ophthalmic B-VEC for treating for ocular complications of deb. Krystal Biotech, Inc. was founded in 2016 and is headquartered in Pittsburgh, Pennsylvania.