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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.

Dyne Therapeutics, Inc., a clinical-stage muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States. It is developing a portfolio of muscle disease therapeutics, including programs in myotonic dystrophy type 1; duchenne muscular dystrophy; and facioscapulohumeral dystrophy, as well as rare skeletal muscle, and cardiac and metabolic muscle diseases using its FORCE platform that delivers disease-modifying therapeutics. Dyne Therapeutics, Inc. was incorporated in 2017 and is headquartered in Waltham, Massachusetts.

Indivior PLC, together with its subsidiaries, engages in the development, manufacture, and sale of buprenorphine-based prescription drugs for the treatment of opioid dependence and co-occurring disorders in the United States, the United Kingdom, and internationally. The company develops medicines to treat substance use disorders, serious mental illnesses, and opioid overdose. Its core marketed products include SUBLOCADE and SUBUTEX PRO buprenorphine extended-release monthly injections; SUBOXONE, a buprenorphine and naloxone sublingual film; SUBOXONE, a buprenorphine and naloxone sublingual tablet; and SUBUTEX, a buprenorphine sublingual tablet for the treatment of opioid use disorder. The company also offers OPVEE nasal spray for opioid overdose reversal; and PERSERIS extended-release injectable suspension for the treatment of schizophrenia in adults. In addition, it is developing INDV-2000, a selective orexin-1 receptor antagonist that completed phase 1 clinical trial for the treatment of opioid use disorder (OUD); INDV-1000, a selective GABAb positive allosteric modulator, which is in pre-clinical development phase for the treatment of alcohol use disorder in collaboration with ADDEX therapeutics; INDV-6001, a buprenorphine-based long-acting injectable for the treatment of OUD in collaboration with Alar Pharmaceuticals Inc.; and CT-102, a digital therapeutic for the treatment of OUD in collaboration with Click Therapeutics. Further, the company is developing INDV-5004, a drinabant injection to treat acute cannabinoid overdose. It has a strategic partnership with Aelis Farma to develop AEF0117, a synthetic CB1 specific signaling inhibitor that is in phase 2B clinical trial for the treatment of cannabis use disorder. The company was incorporated in 2014 and is headquartered in North Chesterfield, Virginia.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and ferroptosis and inflammation platforms, including vatiquinone to treat Friedreich ataxia and utreloxastat for the treatment of amyotrophic lateral sclerosis. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd, Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.