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Akouos, Inc., a precision genetic medicine company, developing gene therapies to restore, improve, and preserve physiologic hearing for individuals. The company's precision genetic medicine platform incorporates a proprietary adeno-associated viral vector library and a novel delivery approach. Its lead product candidate is AK-OTOF, a gene therapy for the treatment of hearing loss due to mutations in the OTOF gene. The company is also developing AK-CLRN1 for the auditory manifestations of Usher syndrome 3A, or USH3A; and AK-antiVEGF for vestibular schwannoma. In addition, its precision genetic medicine platform addresses hearing loss related to genes needed for supporting cell function. The company was incorporated in 2016 and is based in Boston, Massachusetts.

ESSA Pharma Inc., a clinical stage pharmaceutical company, focuses the development of small molecule drugs for the treatment of prostate cancer. The company's lead candidate EPI-7386, an androgen receptor based resistance mechanisms that develop in patients with castration-resistant prostate cancer and metastatic castration-resistant prostate cancer. It has collaboration agreements with Bayer Consumer Care AG; Janssen Research & Development, LLC; and Astellas Pharma Inc. ESSA Pharma Inc. was incorporated in 2009 and is headquartered in Vancouver, Canada.

Homology Medicines, Inc., a genetic medicines company, focuses on transforming the lives of patients suffering from rare genetic diseases in the United States. The company offers proprietary platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines in vivo either through a nuclease-free gene editing mobility, gene therapy or for various genetic disorders. Its earlier stage product candidates comprising HMI-202, an investigational gene therapy which completed IND-enabling studies for the treatment of metachromatic leukodystrophy; and HMI-104 which completed IND-enabling studies. Homology Medicines, Inc. was incorporated in 2015 and is headquartered in Bedford, Massachusetts.

Inozyme Pharma, Inc., a clinical-stage rare disease biopharmaceutical company, focuses on developing novel therapeutics for the treatment of vasculature, soft tissue, and skeleton diseases. The company's lead product candidate is INZ-701, a soluble, recombinant, genetically engineered, and fusion protein for the treatment of ENPP1 and ABCC6 deficiencies, as well as patients with end-stage kidney disease receiving hemodialysis. It has a license agreement with Yale University for specified therapeutic and prophylactic products. The company was incorporated in 2015 and is headquartered in Boston, Massachusetts.

Rigel Pharmaceuticals, Inc., a biotechnology company, engages in discovering, developing, and providing therapies that enhance the lives of patients with hematologic disorders and cancer. The company's commercialized products include Tavalisse, an oral spleen tyrosine kinase inhibitor for the treatment of adult patients with chronic immune thrombocytopenia; Rezlidhia, a non-intensive monotherapy for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test; and GAVRETO, a once daily, small molecule, oral, kinase inhibitor for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer, as well as for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer. It also develops R289, an oral IRAK1/4 Inhibitor, which is in Phase 1b clinical trials for the treatment of hematology-oncology, autoimmune, and inflammatory diseases; and a receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitor program in clinical development with partner Eli Lilly and Company. In addition, the company has product candidates in clinical development with partners BerGenBio ASA and Daiichi Sankyo. The company has strategic development collaboration with The University of Texas MD Anderson Cancer Center for the development of REZLIDHIA (Olutasidenib) in acute myeloid leukemia (AML) and other hematologic cancers. The company was incorporated in 1996 and is headquartered in South San Francisco, California.