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.

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.

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.

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.

Nuvalent, Inc., a clinical stage biopharmaceutical company, develops therapies for patients with cancer. Its lead product candidates are NVL-520, a brain-penetrant ROS1-selective inhibitor to inhibit ROS1 fusions that express the normal ROS1 kinase domain without any drug-resistant mutations and remain active in the presence of mutations conferring resistance to approved and investigational ROS1 inhibitors, which is under Phase I development; and NVL-655, a brain-penetrant ALK-selective inhibitor, to address the clinical challenges of emergent treatment resistance, central nervous system-related adverse events, and brain metastases that might limit the use of first-, second-, and third-generation ALK inhibitors that is under Phase I/II clinical trial.

Ventyx Biosciences, Inc., a clinical-stage biopharmaceutical company, develops small molecule product candidates for inflammatory diseases and autoimmune disorders. The company's lead product candidate is VTX958, a tyrosine kinase type 2 inhibitor that is in phase I clinical trials for the treatment of immune-mediated diseases, such as psoriasis, inflammatory bowel disease, psoriatic arthritis, Crohn's disease, and lupus.

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).

MannKind Corporation, a biopharmaceutical company, focuses on the development and commercialization of inhaled therapeutic products for endocrine and orphan lung diseases in the United States.

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

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.

Kiniksa Pharmaceuticals, Ltd., a biopharmaceutical company, focuses on discovering, acquiring, developing, and commercializing therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical needs worldwide.

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.

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

Vaxcyte, Inc., a clinical-stage biotechnology vaccine company, develops novel protein vaccines to prevent or treat bacterial infectious diseases. Its lead vaccine candidate is VAX-24, a 24-valent investigational pneumococcal conjugate vaccine that is in Phase 1/2 clinical trials to treat invasive pneumococcal disease and pneumonia.

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.