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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.

Mirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.

Tricida, Inc. is a pharmaceutical company. It focuses on the development and commercialization of its product, TRC101, a non-absorbed, orally-administered polymer drug designed to treat metabolic acidosis in patients with chronic kidney disease. The company was founded by Gerrit Klaerner and Craig Jon Hawker on May 22, 2013 and is headquartered in South San Francisco, CA.

Vanda Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization of therapies to address high unmet medical needs worldwide. The company's marketed products include HETLIOZ to treat non-24-hour sleep-wake disorders; and Fanapt oral tablets for the treatment of schizophrenia. Its pipeline products include HETLIOZ (tasimelteon) to treat jet lag disorder, insomnia, delayed sleep phase disorder, sleep disturbances in autism spectrum disorder, and pediatric Non-24; Fanapt (iloperidone) for the treatment of bipolar I disorder and Parkinson's disease psychosis, as well as a long acting injectable (LAI) formulation to treat schizophrenia; and Tradipitant (VLY-686), a small molecule neurokinin-1 receptor (NK-1R) antagonist to treat gastroparesis, motion sickness, atopic dermatitis, and COVID-19 pneumonia. The company's pipeline products also include VTR-297, a small molecule histone deacetylase inhibitor to treat hematologic malignancies and with potential use as a treatment for various oncology indications; VQW-765, a small molecule nicotinic acetylcholine receptor partial agonist to treat performance anxiety and psychiatric disorders; VHX-896, an active metabolite of iloperidone; and antisense oligonucleotide molecules. In addition, it offers a portfolio of cystic fibrosis transmembrane conductance regulator activators and inhibitors that include VSJ-110 for the treatment of dry eye and ocular inflammation; and VPO-227 for the treatment of secretory diarrhea disorders comprising cholera. Vanda Pharmaceuticals Inc. was incorporated in 2002 and is headquartered in Washington, the District of Columbia.

ZIOPHARM Oncology, Inc. is a biopharmaceutical company, which engages in the development, acquisition, and commercialization of immuno-oncology platforms that leverage cell- and gene-based therapies to treat patients with cancer. Its pipeline includes Sleeping Beauty TCR-T Targeting neoantigens; Ad-RTS-hlL-12 + veledimex; and Sleeping Beauty CAR-T. The company was founded on September 9, 2003 and is headquartered in Boston, MA.