Insmed Incorporated, a biopharmaceutical company, develops and commercializes therapies for patients with serious and rare diseases. The company offers ARIKAYCE for the treatment of Mycobacterium avium complex lung disease as part of a combination antibacterial drug regimen for adult patients.

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.

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.

Alnylam Pharmaceuticals, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics based on ribonucleic acid interference. The company's pipeline of investigational RNAi therapeutics focuses on genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS)/ocular diseases.

Exelixis, Inc., an oncology-focused biotechnology company, focuses on the discovery, development, and commercialization of new medicines to treat cancers in the United States.

Alector, Inc., a clinical stage biopharmaceutical company, develops therapies for the treatment of neurodegeneration diseases. Its products include AL001, a humanized recombinant monoclonal antibody, which is in Phase III clinical trial for the treatment of frontotemporal dementia, Alzheimer's, Parkinson's, and amyotrophic lateral sclerosis diseases; and AL101 that is in Phase I clinical trial for the treatment of neurodegenerative diseases, including Alzheimer's and Parkinson's diseases.

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.

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).

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

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.

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.

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

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.

Protagonist Therapeutics, Inc., a biopharmaceutical company, discovers and develops peptide-based therapeutic drugs to address hematology and blood disorders, and inflammatory and immunomodulatory diseases.

Larimar Therapeutics, Inc., a clinical-stage biotechnology company, focuses on developing treatments for rare diseases using its novel cell penetrating peptide technology platform.

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.