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Agios Pharmaceuticals, Inc., a biopharmaceutical company, discovers and develops medicines in the field of cellular metabolism in the United States. Its lead product includes PYRUKYND (mitapivat), an activator of wild-type and mutant pyruvate kinase (PK), enzymes for the treatment of hemolytic anemias. The company develops AG-946, a PK activator for treating lower-risk myelodysplastic syndrome and hemolytic anemias; and AG-181, a phenylalanine hydroxylase stabilizer for the treatment of phenylketonuria. Its preclinical product is siRNA for the treatment of polycythemia vera, a rare blood disorder. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

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. It offers EMPAVELI for the treatment of paroxysmal nocturnal hemoglobinuria, C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis, and hematopoietic stem cell transplantation-associated thrombotic microangiopathy; and SYFOVRE for treating geographic atrophy secondary to age-related macular degeneration and geographic atrophy (GA). The company also develops APL-3007, a small interfering RNA, or siRNA, which is in a Phase 1 clinical trial, as well as an oral complement inhibitor that is in preclinical development. It has a collaboration and license agreement with Swedish Orphan Biovitrum AB (publ) for development and commercialization of pegcetacoplan; and a collaboration with Beam Therapeutics Inc. focused on the use of Beam's base editing technology to discover new treatments for complement-driven diseases. The company was incorporated in 2009 and is based in Waltham, Massachusetts.

Arvinas, Inc., a clinical-stage biotechnology company, engages in the discovery, development, and commercialization of therapies to degrade disease-causing proteins. The company engineers proteolysis targeting chimeras (PROTAC) targeted protein degraders that are designed to harness the body's own natural protein disposal system to degrade and remove disease-causing proteins. Its product pipeline includes Bavdegalutamide and ARV-766, investigational orally bioavailable PROTAC protein degraders for the treatment of men with metastatic castration-resistant prostate cancer, which are in Phase 1/2 clinical trials; and ARV-471, an orally bioavailable estrogen receptor degrading PROTAC targeted protein degrader for the treatment of patients with locally advanced or metastatic estrogen receptor+/human epidermal growth factor receptor 2-breast cancer, which is Phase 3 clinical trial. Arvinas, Inc. has collaborations with Pfizer Inc., Genentech, Inc., F. Hoffman-La Roche Ltd., Carrick Therapeutics Limited, and Bayer AG. The company was founded in 2013 and is based in New Haven, Connecticut.

CytoDyn Inc., a clinical-stage biotechnology company, engages in the development of treatments for multiple therapeutic indications. The company is involved in the clinical development of leronlimab, a novel humanized monoclonal antibody targeting the C-C chemokine receptor type 5 receptor in the areas of COVID-19, human immunodeficiency virus (HIV), Metabolic dysfunction-associated steatohepatitis (MASH), and solid tumors in oncology, such as metastatic triple-negative breast cancer. Its leronlimab is currently under phase 2 development for the treatment of NASH, HIV- NASH, and solid tumors, as well as under pre-clinical development for the treatment of HIV-PrEP and HIV-Cure. The company was formerly known as RexRay Corporation. CytoDyn Inc. was incorporated in 2002 and is based in Vancouver, Washington.

Global Blood Therapeutics, Inc., a biopharmaceutical company, engages in the discovery, development, and delivery of treatments for underserved patient communities with sickle cell disease (SCD). The company offers Oxbryta tablets, an oral, once-daily therapy for SCD. It also evaluates the safety and pharmacokinetics of single and multiple doses of Oxbryta in a Phase II a clinical trial of adolescent and pediatric patients with SCD. In addition, the company is developing its lead product candidate inclacumab, novel human monoclonal antibody, which is in Phase III a clinical trial to reduce the incidence of painful vaso-occlusive crises, and resulting hospital admissions; and GBT601, a hemoglobin polymerization inhibitor that is in Phase I development. It has a license and collaboration agreement with Syros Pharmaceuticals, Inc. to discover, develop, and commercialize therapies for SCD and beta thalassemia; and Sanofi S.A. to two early-stage research programs in SCD. Global Blood Therapeutics, Inc. was incorporated in 2011 and is headquartered in South San Francisco, California.