Denali Therapeutics Inc., a biopharmaceutical company, discovers and develops therapeutic candidates for neurodegenerative diseases in the United States. It offers leucine-rich repeat kinase 2 (LRRK2) inhibitor product candidate, including BIIB122/DNL151, a small molecule inhibitor, which is in phase I and phase Ib clinical trials for the treatment of Parkinson's disease.

argenx SE, a biotechnology company, engages in the developing of various therapies for the treatment of autoimmune diseases in the United States, Japan, Europe, Middle East, Africa, and China.

Harmony Biosciences Holdings, Inc., a commercial-stage pharmaceutical company, develops and commercializes therapies for patients with rare neurological disorders in the United States.

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Europe, and internationally.

Crinetics Pharmaceuticals, Inc., a clinical stage pharmaceutical company, focuses on the discovery, development, and commercialization of therapeutics for rare endocrine diseases and endocrine-related tumors.

Arvinas, Inc., a clinical-stage biopharmaceutical company, engages in the discovery, development, and commercialization of therapies to degrade disease-causing proteins. Its lead product candidates include Bavdegalutamide, a proteolysis targeting chimera (PROTAC) protein degrader that is in phase I clinical trial targeting the androgen receptor (AR) protein for the treatment of men with metastatic castration-resistant prostate cancer (mCRPC); ARV-471, a PROTAC protein degrader targeting the estrogen receptor protein for the treatment of patients with metastatic ER positive/HER2 negative breast cancer; and ARV-766 an investigational orally bioavailable PROTAC protein degrader for the treatment of men with mCRPC.

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.

Kura Oncology, Inc., a clinical-stage biopharmaceutical company, develops medicines for the treatment of cancer in the United States. The company's pipeline consists of small molecule product candidates that target cancer.

Mineralys Therapeutics, Inc., a clinical-stage biopharmaceutical company that develops therapies for the treatment of hypertension and associated cardiovascular diseases. It clinical-stage product candidate is lorundrostat, a proprietary, orally administered, highly selective aldosterone synthase inhibitor for the treatment of patients with uncontrolled or resistant hypertension.

Legend Biotech Corporation, a clinical-stage biopharmaceutical company, through its subsidiaries, engages in the discovery and development of novel cell therapies for oncology and other indications in the United States, China, and internationally.

Cytokinetics, Incorporated, a late-stage biopharmaceutical company, focuses on discovering, developing, and commercializing muscle activators and inhibitors as potential treatments for debilitating diseases.

Inhibrx Biosciences, Inc., a clinical-stage biopharmaceutical company, engages in the development of biologic therapeutics for people with life-threatening conditions. Its therapeutic candidates includes INBRX-109, a tetravalent therapeutic candidate targeting death-receptor 5 that is in phase 2 clinical trial for the treatment of unresectable or metastatic conventional chondrosarcoma; and INBRX-106, a hexavalent sdAb-based therapeutic candidate targeting OX4 that is in phase 2 clinical trial for the treatment of metastatic solid tumor, non-small cell lung cancer, melanoma, head and neck cancer, gastric (GIST) and gastroesophageal adenocarcinoma (GEA) cancer, renal cell carcinoma, and urothelial (transitional) cell carcinoma.

Edgewise Therapeutics, Inc., a biopharmaceutical company, develops small molecule therapies for musculoskeletal diseases. The Company's lead product candidate, EDG-5506, is an orally administered small molecule designed to address the root cause of dystrophinopathies including Duchenne muscular dystrophy and Becker muscular dystrophy which has completed Phase 1 clinical trial.

Dyne Therapeutics, Inc., a muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States.

Mereo BioPharma Group plc, a biopharmaceutical company, develops and commercializes therapeutics for the treatment of oncology and rare diseases in the United Kingdom and internationally.

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

Agios Pharmaceuticals, Inc., a biopharmaceutical company, engages in the discovery and development of medicines in the field of cellular metabolism and adjacent areas of biology.

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.

BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease.