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. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.

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.

Rapport Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery and development of small molecule medicines for patients suffering from central nervous system (CNS) disorders. Its lead product candidate is receptor associated protein (RAP)-219, an investigational small molecule that is designed to inhibit TARPy8-containing AMPARs with picomolar affinity for the treatment of focal epilepsy and other CNS disorders, including peripheral neuropathic pain and bipolar disorder. The company also develops RAP-199, a TARPy8 targeted molecule with differentiated chemical and pharmacokinetic properties; and nicotinic acetylcholine receptor (nAChR) programs, such as a6 nAChR to treat chronic pain and a9a10 nAChR for the treatment of hearing disorders. Rapport Therapeutics, Inc. was incorporated in 2022 and is headquartered in Boston, Massachusetts.

PureTech Health plc, engages in the development and commercialization of biotechnology and pharmaceutical solutions in the United States. The company is developing LYT-100, which is under Phase 2 stage, to treat idiopathic pulmonary fibrosis (IPF); and LYT-200, a IgG4 monoclonal antibody, currently under Phase 1/2 stage, targeting galectin-9 for treating solid tumors and hematological malignancies. It also develops SPT-300, an oral drug of allopregnanolone for the treatment of anxious depression; SPT-320, a novel prodrug of agomelatine for the treatment of generalized anxiety disorder; and SPT-348, a prodrug of a non-hallucinogenic neuroplastogen for the treatment of mood and other neuropsychiatric disorders. In addition, the company is developing hydrogels to enable the oral administration of peptide therapeutics in preclinical development. The company was incorporated in 2015 and is headquartered in Boston, 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.

NeuroSense Therapeutics Ltd., a clinical-stage biotechnology company, focuses on discovering and developing treatments for patients suffering from debilitating neurodegenerative diseases in the United States and internationally. The company's lead product is PrimeC, an extended-release oral formulation of a fixed-dose combination of ciprofloxacin and celecoxib, currently under Phase 2b/3 clinical trials for the treatment of amyotrophic lateral sclerosis, and completed Phase 2 clinical trials for the treatment of Alzheimer's, as well as under preclinical studies for the treatment of Parkinson's disease. Its preclinical pipeline includes StabiliC for the treatment of Parkinson's disease; and CogniC for the treatment of Alzheimer's disease. The company was incorporated in 2017 and is headquartered in Herzliya, Israel.

Cerevel Therapeutics Holdings, Inc., a clinical-stage biopharmaceutical company, develops various therapies for neuroscience diseases in the United States. It is developing Emraclidine, a positive allosteric modulator (PAM) that is in phase 1b clinical trials for the treatment of schizophrenia; and Darigabat, a PAM, which is in Phase 2 proof-of-concept trial in patients with drug-resistant focal onset seizures or focal epilepsy, as well as in phase 1 trial to treat panic symptoms model. The company's products also comprise Tavapadon, a selective dopamine D1/D5 partial agonist that is in phase 3 clinical trial for the treatment of early- and late-stage Parkinson's disease; CVL-871, a selective dopamine D1/D5 partial agonist, which is in Phase 2a clinical trial to treat dementia-related apathy; and CVL-354, a selective kappa-opioid receptor antagonist to treat major depressive disorder and substance use disorder. It is also involved in the development of an M4 agonist program for the treatment of psychosis and related indications; and PDE4 inhibitor for the treatment of psychiatric, neuroinflammatory, and other disorders. Cerevel Therapeutics Holdings, Inc. was founded in 2018 and is headquartered in Cambridge, Massachusetts.

PaxMedica, Inc., a clinical stage biopharmaceutical company, focuses on the development of anti-purinergic drug therapies for the treatment of disorders with intractable neurologic symptoms. Its lead product candidate is PAX-101 that is in Phase 3 clinical trials, an intravenous formulation of suramin for the treatment of autism spectrum disorder, myalgic encephalomyelitis/chronic fatigue syndrome, long COVID-19 syndrome, fragile X syndrome, human African trypanosomiasis (HAT), and fragile X-associated tremor/ataxia syndrome. The company is developing PAX-102, an intranasal formulation of suramin for neurologic indications, as well as other new chemical entities that are targeted and selective antagonists of purine receptor subtypes; and PAX-HAT-301 for the treatment of HAT. It has a research collaboration agreement with PolarMar Health for Phase II clinical trial in austism spectrum disorder for emodin, which retains the rights for PolarMar to develop and commercialize any non-prescription supplement form of the product, and for PaxMedica, Inc. to retain exclusive rights to develop and commercialize a highly purified form of emodin. The company was formerly known as Purinix Pharmaceuticals LLC and changed its name to PaxMedica, Inc. in April 2020. PaxMedica, Inc. was incorporated in 2018 and is based in Tarrytown, New York.