Wave Life Sciences Ltd., a clinical stage genetic medicine company, designs, optimizes, and produces novel stereopure oligonucleotides through PRISM, a discovery and drug developing platform.

CytomX Therapeutics, Inc. operates as an oncology-focused biopharmaceutical company in the United States. The company develops antibody therapeutics based on its Probody technology platform for the treatment of cancer.

Assembly Biosciences, Inc., a clinical-stage biotechnology company, discovers and develops therapeutic candidates for the treatment of hepatitis B virus (HBV) infection in the United States.

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.

Incyte Corporation, a biopharmaceutical company, focuses on the discovery, development, and commercialization of proprietary therapeutics in the United States and internationally.

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy.

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.

ProQR Therapeutics N.V., a biopharmaceutical company, engages in the discovery and development of RNA-based therapeutics for the treatment of genetic disorders.

Annexon, Inc., a clinical-stage biopharmaceutical company, discovers and develops therapeutics for autoimmune, neurodegenerative, and ophthalmic disorders. The company's C1q is an initiating molecule of the classical complement pathway that targets distinct disease processes, such as antibody-mediated autoimmune disease and complement-mediated neurodegeneration.

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.

Terns Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops small-molecule single-agent and combination therapy candidates for the treatment of non-alcoholic steatohepatitis (NASH) and obesity.

Amylyx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, engages in developing various therapeutics for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases.

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