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.

Replimune Group, Inc., a biotechnology company, develops oncolytic immuno-gene therapies to treat cancer. It uses its proprietary Immunotherapy platform to design and develop product candidates that are intended to activate the immune system against cancer.

Mirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases.

Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a multi-platform biotechnology company that focuses on developing gene therapies for rare and devastating diseases.

Avidity Biosciences, Inc., a biopharmaceutical company, engages in the development of oligonucleotide-based therapies. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat a range of serious diseases.

uniQure N.V., a gene therapy company, engages in the development of treatments for patients suffering from genetic and other devastating diseases.

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

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.

Immunocore Holdings plc, a commercial-stage biotechnology company, engages in the development of immunotherapies for the treatment of cancer, infectious, and autoimmune diseases.

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.

Monte Rosa Therapeutics, Inc., a biopharmaceutical company, engages in the development of novel small molecule precision medicines that employ the body's natural mechanisms to selectively degrade therapeutically relevant proteins.

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops novel antisense oligonucleotide (ASO) medicines to treat the underlying causes of severe genetic diseases in the United States.

AN2 Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing treatments for rare, chronic, and serious infectious diseases. It is developing epetraborole, a once-daily oral treatment for patients with chronic non-tuberculous mycobacterial lung disease.

Passage Bio, Inc., a genetic medicines company, develops transformative therapies for central nervous system diseases. It develops PBGM01, which utilizes a proprietary, AAVhu68 capsid to deliver to the brain and peripheral tissues a functional GLB1 gene encoding lysosomal acid beta-galactosidase for infantile GM1; PBFT02, which utilizes an AAV1 capsid to deliver to the brain a functional granulin (GRN) and gene encoding progranulin (PGRN) for the treatment of FTD-GRN; and PBKR03, which utilizes a proprietary, AAVhu68 capsid to deliver to the brain and peripheral tissues a functional GALC gene encoding the hydrolytic enzyme galactosylceramidase for infantile Krabbe disease.

4D Molecular Therapeutics, Inc., a clinical-stage gene therapy company, develops product candidates using its adeno-associated viruses vectors. It develops a portfolio of gene therapy product candidates focuses in three therapeutic areas: ophthalmology, cardiology, and pulmonology.

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.

Kezar Life Sciences, Inc., a clinical-stage biotechnology company, engages in the discovery and development of novel small molecule therapeutics to treat unmet needs in immune-mediated diseases and cancer in the United States.