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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.

Cryoport, Inc. provides temperature-controlled supply chain solutions in biopharma/pharma, animal health, and human reproductive medicine markets in the Americas, Europe, the Middle East, Africa, and the Asia Pacific. The company offers Cryoport Express Shippers; Cryoport ELITE -80°C Gene Therapy Shipper; Cryoport ELITE Cryosphere Shipper; Cryoport consulting services, including physical, thermal, and shipping qualifications of shipping systems and/or packaging to developing custom packaging solutions; and Cryoport bioservices, such as controlled temperature storage, fulfilment, kit production, secondary packaging, labelling of therapeutic products, and GMP raw materials storage services. It also provides CRYOGENE, an unparalleled solution that offers pre-clinical temperature-controlled biological materials management services comprising specimen storage, processing, collection, and retrieval; CRYOPD, a temperature-controlled logistics solution, which include temperature-controlled packaging and transport solutions from cryogenic temperature to controlled ambient; and IntegriCell services that comprise apheresis/leukapheresis collection, Cryoshuttle transportation, cryo-process optimization and processing services. In addition, the company offers MVE Biological Solutions' Fusion cryogenic system, a self-sustaining cryogenic freezer; MVE Biological Solutions' Vario cryogenic system, a cryogenic freezer system that supports temperatures between -20°C and -150°C; Cryoport Cryoshuttle provides clients with dedicated local transportation support; and Tec4Med. Further, it provides biological specimen cryopreservation storage and maintenance; archiving, monitoring, tracking, receipt, and delivery of samples; frozen biological specimens transportation; and incoming and outgoing biological specimens management; and short-term logistics and engineering consulting services. Cryoport, Inc. was founded in 1999 and is headquartered in Brentwood, Tennessee.

Dicerna Pharmaceuticals, Inc. is a biopharmaceutical company, which engages in discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to selectively silence genes that cause or contribute to disease. The firm uses its proprietary GalXC RNAi technology platform to build a pipeline in these therapeutic areas. The company was founded by Douglas M. Fambrough III, Mark A. Behlke, Roberto Guerciolini, James Craig Jenson, and John J. Rossi in October 2006 and is headquartered in Lexington, MA.

Intra-Cellular Therapies, Inc., a biopharmaceutical company, focuses on the discovery, clinical development, and commercialization of small molecule drugs that address medical needs primarily in neuropsychiatric and neurological disorders by targeting intracellular signaling mechanisms in the central nervous system (CNS) in the United States. The company offers CAPLYTA for the treatment of schizophrenia and bipolar depression in adults. It is also involved in developing Lumateperone, which is in Phase 3 clinical trial for the treatment of various depressive disorders, as well as additional neuropsychiatric indications. In addition, the company is developing Lenrispodun (ITI-214) for the treatment of Parkinson's disease, CNS, and other disorders; ITI-1284 for the treatment of neuropsychiatric disorders and behavioral disturbances in dementia; and ITI-333 for substance use disorders, pain, and psychiatric comorbidities, including depression, anxiety, and sleep disorders. Intra-Cellular Therapies, Inc. was founded in 2002 and is headquartered in New York, New York.

Verve Therapeutics, Inc., a clinical stage genetic medicines company, engages in developing gene editing medicines for patients to treat cardiovascular diseases in the United States. The company's lead product candidate is VERVE-101, a single-course gene editing treatment that permanently turns off the PCSK9 gene in the liver; and VERVE-102, a product candidate that targets the PCSK9 gene for the treatment of HeFH. It engages in the development of ANGPTL3 program to permanently turn off the ANGPTL3 gene in the liver. It has a collaboration and license agreement with Beam Therapeutics Inc.; a development and option agreement with Acuitas Therapeutics, Inc.; and a Cas9 license agreement with The Broad Institute and the President and Fellows of Harvard College. The company was formerly known as Endcadia, Inc. and changed its name to Verve Therapeutics, Inc. in January 2019. Verve Therapeutics, Inc. was incorporated in 2018 and is headquartered in Cambridge, Massachusetts.