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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.

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.

Eiger BioPharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the development and commercialization of targeted therapies for rare and ultra-rare diseases in the United States and internationally. Its lead product candidate is Lonafarnib, an orally bioavailable, small molecule, which is in Phase III clinical trials to treat hepatitis delta virus infection. The company's product candidate also include Lambda, which targets type III interferon receptors that has completed Phase II clinical trials; Lonafarnib for the treatment of progeria and progeroid laminopathies; and Avexitide for the treatment of congenital hyperinsulinism, as well as has completed Phase II clinical trials to treat post-bariatric hypoglycemia. Eiger BioPharmaceuticals, Inc. was founded in 2008 and is headquartered in Palo Alto, California.

Fennec Pharmaceuticals Inc., a biopharmaceutical company, develops product candidates for use in the treatment of cancer in the United States. Its lead product candidate is the Sodium Thiosulfate, which has completed the Phase III clinical trial for the prevention of cisplatin induced hearing loss or ototoxicity in children. The company was formerly known as Adherex Technologies Inc. and changed its name to Fennec Pharmaceuticals Inc. in September 2014. Fennec Pharmaceuticals Inc. was founded in 1996 and is based in Research Triangle Park, North Carolina.

Gamida Cell Ltd., a clinical-stage biopharmaceutical company, develops cell therapies to cure blood cancers and serious blood diseases. The company's lead product candidate is omidubicel, a cell therapy that has completed Phase III clinical trial in patients with hematologic malignancies, as well as in Phase II clinical trials in patients with severe aplastic anemia. It is also developing GDA-201, an investigational NK cell-based cancer immunotherapy, which is in Phase I/II studies for the treatment of relapsed or refractory non-Hodgkin lymphoma and multiple myeloma. The company was incorporated in 1998 and is headquartered in Jerusalem, Israel.