Krystal Biotech, Inc., a clinical stage biotechnology company, engages in the field of redosable gene therapy to treat serious rare diseases in the United States.

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases.

Iovance Biotherapeutics, Inc., a clinical-stage biotechnology company, focuses on developing and commercializing cancer immunotherapy products to harness the power of a patient's immune system to eradicate cancer cells.

Madrigal Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapeutic candidates for the treatment of cardiovascular, metabolic, and liver diseases.

Blueprint Medicines Corporation, a precision therapy company, develops medicines for genomically defined cancers and blood disorders in the United States and internationally.

Viking Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the development of novel therapies for metabolic and endocrine disorders. The company's lead drug candidate is VK2809, an orally available tissue and receptor-subtype selective agonist of the thyroid hormone receptor beta (TRß), which is in Phase IIb clinical trials to treat patients with biopsy-confirmed non-alcoholic steatohepatitis, as well as NAFLD.

Hepion Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development of drug therapy treatment for chronic liver diseases in the United States.