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.

Akero Therapeutics, Inc., a cardio-metabolic nonalcoholic steatohepatitis (NASH) company, engages in the development of medicines designed to restore metabolic balance and improve overall health.

Apellis Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases.

Day One Biopharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops and commercializes targeted therapies for patients with genetically defined cancers. Its lead product candidate is DAY101, an oral brain-penetrant type II pan-rapidly accelerated fibrosarcoma kinase inhibitor that is in Phase II clinical trial for pediatric patients with relapsed/progressive low-grade glioma.

Phathom Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on developing and commercializing treatments for gastrointestinal diseases. The company has the rights in the United States, Europe, and Canada to vonoprazan, a potassium-competitive acid blocker (P-CAB) that blocks acid secretion in the stomach.

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

Lyra Therapeutics, Inc., a clinical-stage therapeutics company, focuses on the development and commercialization of novel integrated drug and delivery solutions for the localized treatment of patients with ear, nose, and throat diseases.

Cullinan Oncology Inc., a biopharmaceutical company, focuses on developing a pipeline of targeted oncology and immuno-oncology therapies for cancer patients in the United States.

Ascendis Pharma A/S, a biopharmaceutical company, focuses on developing therapeutics for unmet medical needs. The company offers SKYTROFA for treating patients with growth hormone deficiency (GHD).

Pliant Therapeutics, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes novel therapies for the treatment of fibrosis and related diseases in the United States.

Syndax Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapies for the treatment of cancer. Its lead product candidates are SNDX-5613, which is in phase 1/2 clinical trial targeting the binding interaction of Menin with the mixed lineage leukemia 1 protein for the treatment of MLL-rearranged (MLLr) and nucleophosmin 1 mutant acute myeloid leukemia (NPM1c AML); and SNDX-6352 or axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1, or CSF-1 receptor for the treatment of patients with chronic graft versus host disease (cGVHD).

Revolution Medicines, Inc., a clinical-stage precision oncology company, focuses on developing therapies to inhibit frontier targets in RAS-addicted cancers. The company is developing RMC-4630, an inhibitor of SHP2, which is in Phase 1/2 clinical trial for the treatment of solid tumors, such as gynecologic and colorectal cancer tumors.

Viridian Therapeutics, Inc., a biotechnology company, develops treatments for patients suffering from serious diseases. It develops VRDN-001, a humanized monoclonal anti-IGF-1R antibody that is in Phase 1/2 clinical trial for the treatment of thyroid eye disease (TED); VRDN-002, an IGF-1R antibody, which is in Phase 1 clinical trial; and VRDN-003, a therapeutic antibody targeting IGF-1R for the treatment of TED.

Inozyme Pharma, Inc., a rare disease biopharmaceutical company, engages in developing therapeutics for treating abnormal mineralization impacting the vasculature, soft tissue, and skeleton diseases.