Soleno Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of novel therapeutics for the treatment of rare diseases. Its lead candidate is Diazoxide Choline Extended-Release tablets, a once-daily oral tablet, which is in Phase III clinical trials for the treatment of Prader-Willi Syndrome. The company was formerly known as Capnia, Inc. and changed its name to Soleno Therapeutics, Inc. in May 2017. Soleno Therapeutics, Inc. was incorporated in 1999 and is based in Redwood City, California.

Mineralys Therapeutics, Inc., a clinical-stage biopharmaceutical company that develops therapies for the treatment of hypertension and chronic kidney diseases. It clinical-stage product candidate is lorundrostat, a proprietary, orally administered, highly selective aldosterone synthase inhibitor for the treatment of cardiorenal conditions affected by abnormally elevated aldosterone. The company was formerly known as Catalys SC1, Inc. and changed its name to Mineralys Therapeutics, Inc. in May 2020. The company was incorporated in 2019 and is headquartered in Radnor, Pennsylvania.

Dianthus Therapeutics, Inc., a clinical-stage biotechnology company, develops complement therapeutics for patients with severe autoimmune and inflammatory diseases. It is developing DNTH103, a monoclonal antibody, which is in Phase 2 clinical trial, for the treatment of generalized myasthenia gravis, multifocal motor neuropathy, and chronic inflammatory demyelinating polyneuropathy. Dianthus Therapeutics, Inc. was founded in 2019 and is headquartered in New York, New York.

CorMedix Inc., a biopharmaceutical company, focuses on developing and commercializing therapeutic products for the prevention and treatment of infectious and inflammatory diseases in the United States. Its lead product candidate is DefenCath, an antimicrobial catheter lock solution to reduce the incidence of catheter-related bloodstream infections in adult patients with kidney failure. The company was formerly known as Picton Holding Company, Inc. and changed its name to CorMedix, Inc. in January 2007. CorMedix Inc. was incorporated in 2006 and is based in Berkeley Heights, New Jersey.

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels. Its lead clinical candidate is STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. The company also develops STK-001, which is in phase I/II clinical trial to treat Dravet syndrome; and programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye. The company has a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.

Neumora Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in developing therapeutic treatments for brain diseases, neuropsychiatric disorders, and neurodegenerative diseases. The company develops navacaprant (NMRA-140), a novel once-daily oral kappa opioid receptor antagonist, which is in phase 3 clinical trials for the treatment of major depressive disorder. It also develops NMRA-511 that is in phase 1 clinical trials in patients with agitation associated with dementia due to Alzheimer's disease; and NMRA-266, which is in the phase 1 clinical trial for the treatment of schizophrenia and other neuropsychiatric disorders. In addition, its preclinical phase product includes NMRA-NMDA for the treatment of schizophrenia; NMRA-CK1d, a CK1d inhibitor program for the treatment of amyotrophic lateral sclerosis; NMRA-NLRP3 for the treatment of certain neurodegenerative conditions; and NMRA-GCase for the treatment of Parkinson's disease. The company was formerly known as RBNC Therapeutics, Inc. and changed its name to Neumora Therapeutics, Inc. in October 2021. Neumora Therapeutics, Inc. was incorporated in 2019 and is headquartered in Watertown, Massachusetts.

MediciNova, Inc., a biopharmaceutical company, focuses on developing novel and small molecule therapeutics for the treatment of serious diseases with unmet medical needs in the United States. It is developing MN-166 (ibudilast), an oral anti-inflammatory and neuroprotective agent for treating neurological and other disorders, such as primary and secondary progressive multiple sclerosis, amyotrophic lateral sclerosis, chemotherapy-induced peripheral neuropathy, degenerative cervical myelopathy, glioblastoma, and substance dependence and addiction, as well as prevention of acute respiratory distress syndrome, and long COVID. The company's product pipeline also includes MN-221 (bedoradrine), a selective beta-2-adrenergic receptor agonist for the treatment of acute exacerbations of asthma; MN-001 (tipelukast), an orally bioavailable small molecule compound to treat fibrotic and other diseases, including nonalcoholic fatty liver disease and idiopathic pulmonary fibrosis; and MN-029 (denibulin), a tubulin binding agent for treating solid tumor cancers. It has license agreements with Kyorin Pharmaceutical Co., Ltd; Angiogene Pharmaceuticals, Ltd.; and Meiji Seika Kaisha, Ltd. The company was incorporated in 2000 and is headquartered in La Jolla, California.

Biodexa Pharmaceuticals Plc, a clinical stage biopharmaceutical company, focuses on developing a pipeline of products for the treatment of Type 1 diabetes and rare/orphan cancers of the brain. Its lead product candidate Tolimidone, a selective activator of the lyn kinase enzyme, currently under Phase II studies for the treatment of Type 1 diabetes. The company is also developing MTX110, which is currently in Phase I studies for the treatment of diffuse intrinsic recurrent glioblastoma, diffuse midline glioma, and medulloblastoma; and MTD217, a program centered around a water-soluble drug formulation that can be easily infused or injected simultaneously, or sequentially, directly into the cancer microenvironment, currently under preclinical studies for the treatment of leptomeningeal disease. In addition, the company offers drug delivery platforms, such as Q-Sphera, a polymer microsphere microtechnology used for sustained release drug delivery; MidaSolve, an oligosaccharide nanotechnology used to solubilize drugs so that they can be administered in liquid form directly and locally into tumors; and MidaCore, a gold nanoparticle used for targeting sites of disease by using chemotherapeutic agents or immunotherapeutic agents. It has collaboration agreements with Janssen and Melior. The company was formerly known as Midatech Pharma plc and changed its name to Biodexa Pharmaceuticals Plc in March 2023. Biodexa Pharmaceuticals Plc was founded in 2000 and is headquartered in Cardiff, the United Kingdom.