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.

IDEAYA Biosciences, Inc., a synthetic lethality-focused precision medicine oncology company, discovers and develops targeted therapeutics for patient populations selected using molecular diagnostics in the United States. The company's products in development include IDE196, a protein kinase C inhibitor that is in Phase 2/3 clinical trials for genetically defined cancers having GNAQ or GNA11 gene mutations; IDE397, a methionine adenosyltransferase 2a inhibitor that is in Phase 1/2 clinical trials for patients with solid tumors having methylthioadenosine phosphorylase gene deletions, such as non-small cell lung, bladder, gastric, and esophageal cancers; IDE161, a poly ADP-ribose glycohydrolase inhibitor that is in Phase 1 clinical trial to treat tumors with homologous recombination deficiency (HRD), and other genetic or molecular signatures; GSK101, a Pol Theta Helicase inhibitor that is in Phase 1 clinical trial for the treatment of tumors with BRCA or other homologous recombination, and HRD mutations; and Werner Helicase inhibitors for tumors with high microsatellite instability. It offers preclinical research programs focused on pharmacological inhibition; DECIPHER dual CRISPR library for synthetic lethality target and biomarker discovery; and INQUIRE chemical library and HARMONY machine-learning engines to enhance its drug discovery platform. The company has strategic alliances with GlaxoSmithKline, Pfizer Inc., Novartis International Pharmaceuticals Ltd., Amgen Inc., Gilead Sciences, Inc., Cancer Research UK, and the University of Manchester. IDEAYA Biosciences, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California.

Syndax Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapies for the treatment of cancer. Its lead product candidates are revumenib, a potent, selective, small molecule inhibitor of the menin-MLL binding interaction for the treatment of KMT2A rearranged, acute leukemias, and solid tumor; and SNDX-6352 or axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1, or CSF-1 receptor for the treatment of patients with chronic graft versus host disease (cGVHD) and idiopathic pulmonary fibrosis (IPF). The company is also developing Entinostat. It has an agreement with Eddingpharm International Company Limited for licensing, development, and commercialization of Entinostat. Syndax Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Waltham, Massachusetts.

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. It has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies; and Sanofi for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

MetaVia Inc., a clinical-stage biotechnology company focuses on developing and commercializing novel pharmaceuticals to treat cardiometabolic diseases. It develops DA-1241, a novel G-Protein-Coupled Receptor 119 agonist with development optionality as a standalone and/or combination therapy that is in Phase 2a clinical trial for metabolic dysfunction-associated steatohepatitis (MASH), and has completed Phase 1 clinical trial for the treatment of type 2 diabetes mellitus (T2DM); and DA-1726, a novel oxyntomodulin analogue functioning as a GLP-1 receptor and glucagon receptor dual agonist, which is in preclinical development for the treatment of obesity. The company's therapeutic programs include ANA001, a proprietary oral niclosamide formulation for the treatment of patients with moderate COVID-19; NB-01 for the treatment of painful diabetic neuropathy; NB-02 for the treatment of cognitive impairment; and Gemcabene for the treatment of dyslipidemia. It has a license agreement with Pfizer Inc. for the research, development, manufacture, and commercialization of Gemcabene; and joint research agreement with Dong-A ST and ImmunoForge for the development of DA-1726. The company was formerly known as NeuroBo Pharmaceuticals, Inc. and changed its name to MetaVia Inc. in November 2024. MetaVia Inc. is headquartered in Cambridge, Massachusetts.

Cyclerion Therapeutics, Inc., a biopharmaceutical company, develops treatments for serious diseases. It is developing Olinciguat, an orally administered vascular soluble guanylate cyclase (sGC) stimulator, which is in Phase 2 clinical trial to out-license for cardiovascular diseases; and Praliciguat, a systemic sGC stimulator that is licensed to Akebia Therapeutics, Inc. for the treatment of rare kidney disease. The company was incorporated in 2018 and is headquartered in Cambridge, Massachusetts.

Theriva Biologics, Inc., a clinical-stage company, develops therapeutics to treat cancer and related diseases in areas of high unmet need in the United States. The company's lead product candidate is VCN-01, a clinical stage oncolytic human adenovirus that is in a Phase 2 clinical study for the treatment of pancreatic cancer; a Phase 1 clinical study for the treatment of retinalblastoma; a Phase 1 clinical study for the treatment of head and neck squamous cell carcinoma; and a Phase 1 clinical study for the treatment of solid tumors. It also develops VCN-11 for treating solid tumors; SYN-004 designed to degrade various commonly used intravenous beta-lactam antibiotics in gastrointestinal (GI) tract for the prevention of microbiome damage to prevent overgrowth and infection by pathogenic organisms, such Clostridioides difficile infection and vancomycin resistant Enterococci, and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant recipients; and SYN-020, a recombinant oral formulation for the enzyme intestinal alkaline phosphatase to treat both local GI and systemic diseases. In addition, the company develops clinical stage products, such as SYN-006 to prevent aGVHD and infection by carbapenem resistant enterococci; and SYN-007 for preventing antibiotic associated diarrhea with oral ß-lactam antibiotics. It has collaboration agreements with The University of Texas at Austin, Catalan Institute of Oncology, Fundació Privada Institut d'Investigacio Biomedica de Bellvitge, Saint Joan De Déu, and Massachusetts General Hospital, as well as a clinical trial agreement with Washington University School of Medicine in St. Louis to conduct a Phase 1b/2a clinical trial of SYN-004. Theriva Biologics, Inc. is headquartered in Rockville, Maryland.

Finch Therapeutics Group, Inc., a clinical-stage microbiome therapeutics company, develops a novel class of orally administered biological drugs in the United States. The company develops FIN-211, a microbiome candidate designed to address the gastrointestinal and behavioral symptoms of autism spectrum disorder; and FIN-524 for the prevention, diagnosis, theragnosis or treatment of diseases in humans, including ulcerative colitis; FIN-525 for the treatment of Crohn's disease; and other microbiome product candidates. The company has collaboration and license agreements with Takeda Pharmaceutical Company Limited; and license agreements with Skysong Innovations LLC, OpenBiome, Arizona State University, and University of Minnesota. Finch Therapeutics Group, Inc. was incorporated in 2014 and is based in Boston, Massachusetts.