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ACADIA Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization innovative medicines that address unmet medical needs in central nervous system (CNS) disorders and rare diseases in the United States. The company offers NUPLAZID (pimavanserin) for the treatment of hallucinations and delusions associated with Parkinson's disease psychosis; and DAYBUE, a novel synthetic analog of the amino-terminal tripeptide of insulin-like growth factor 1 for treatment of Rett Syndrome. It also develops Pimavanserin that is in Phase III ADVANCE-2 study to treat the negative symptoms of schizophrenia; ACP-101 whixh is in Phase III for the treatment of hyperphagia in Prader-Willi syndrome; ACP-204 which is in Phase II for the treatment of Alzheimer's disease psychosis; ACP-2591 that is in Phase I for Rett syndrome and Fragile X syndrome; preclinical antisense oligonucleotide programs; and other programs for neuropsychiatric symptoms. It has a license agreement with Neuren Pharmaceuticals Limited to develop and commercialize trofinetide for Rett syndrome and other indications; and a license and collaboration agreement with Stoke Therapeutics, Inc. to discover, develop and commercialize novel RNA-based medicines for the potential treatment of severe and rare genetic neurodevelopmental diseases of the CNS. The company was formerly known as Receptor Technologies, Inc. and changed its name ACADIA Pharmaceuticals Inc. in 1997. ACADIA Pharmaceuticals Inc. was incorporated in 1993 and is headquartered in San Diego, California.

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.

Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.

Reata Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, identifies, develops, and commercializes novel therapeutics for patients with serious or life-threatening diseases. The company is developing Phase 3 clinical trial programs, including bardoxolone methyl (bardoxolone) for the treatment of patients with chronic kidney disease (CKD) caused by Alport syndrome, as well as for a form of pulmonary arterial hypertension associated with connective tissue disease; omaveloxolone to treat Friedreich's ataxia; and conduct various form of CKD, such as, type 1 and type 2 diabetic CKD, type 1 and type 2 diabetic CKD, hypertensive CKD, focal segmental glomerulosclerosis, and others. It is also developing RTA 901 for neurological diseases; and bardoxolone for the treatment of autosomal dominant polycystic kidney disease. In addition, the company has a strategic collaboration agreement with Kyowa Kirin Co., Ltd. to develop and commercialize bardoxolone for renal, cardiovascular, diabetes, and various other related metabolic indications in Japan, China, Hong Kong, Macao, South Korea, Taiwan, Thailand, Singapore, the Philippines, Malaysia, Indonesia, Brunei, Vietnam, Laos, Myanmar, and Cambodia; AbbVie Inc. to jointly research, develop, and commercialize all second- and later-generation Nrf2 activators for all indications other than renal, cardiovascular, and metabolic indications. Reata Pharmaceuticals, Inc. was incorporated in 2002 and is headquartered in Plano, Texas.