Companies in #Neurology

Develops and commercializes medicines for neurological disorders and rare diseases, including Parkinson's disease psychosis, Alzheimer's disease psychosis, and Rett syndrome.

ADARx Pharmaceuticals is a clinical-stage biotechnology company developing RNA-based therapeutics using its proprietary ADAR (Adenosine Deaminase Acting on RNA) editing platform. The company is allied with AbbVie and has three drugs in the clinic.

Medical device company developing clinically-proven precision light-filtering lenses for people living with migraine and light sensitivity. Received the first FDA De Novo authorization for a lens classification targeting migraine-associated light sensitivity.

Biohaven is a clinical-stage biopharmaceutical company developing therapies for neurological and neuropsychiatric diseases. Following the spin-off of its commercial migraine portfolio to Pfizer, Biohaven is advancing next-generation neuroscience programs including IGF-1 targeted therapies and rare disease programs.

Filana Therapeutics is a clinical-stage biopharmaceutical company developing simufilam, a small-molecule modulator of filamin A, for tuberous sclerosis complex (TSC)-related epilepsy. Formerly known as Cassava Sciences and, before that, Pain Therapeutics, the company rebranded and refocused on refractory TSC-associated seizures in March 2026 after an earlier Alzheimer's disease development programme. In September 2026 the FDA lifted a full clinical hold on the company's investigational new drug application for simufilam, clearing the way for a planned 16-week, multicenter, randomized, double-blind Phase 2a proof-of-concept study evaluating two doses in 40 patients aged 12 to 55 at 13 U.S. sites, with patient screening expected to begin in the first quarter of 2027.

Therapeutics company focused on Friedreich's Ataxia. Received IND clearance for GV101 from the FDA in August 2026.

Immunovant is a clinical-stage immunology company and a trailblazer in anti-FcRn technology, developing targeted therapies that reduce harmful immunoglobulin G autoantibodies to enable normal lives for people with autoimmune diseases. Its lead investigational product, imeroprubart (IMVT-1402), is a fully human monoclonal antibody advancing across multiple autoimmune indications.

Commercial-stage biopharmaceutical company developing small-molecule therapies for rare and neurodegenerative diseases; its lead drug AQNEURSA (levacetylleucine) is FDA-approved for Niemann-Pick disease type C and, as of September 2026, ataxia-telangiectasia — the first approved treatment for that disease anywhere.

Ionis Pharmaceuticals is a leader in RNA-targeted therapeutics, discovering and developing antisense oligonucleotide (ASO) drugs for serious diseases.

Ionis Pharmaceuticals (NASDAQ: IONS) is a Carlsbad, California-based antisense oligonucleotide (ASO) drug discovery and development company with a deep pipeline spanning neurology, cardiometabolic disease, renal disease, and rare disorders.

Karuna Therapeutics is a clinical-stage biopharmaceutical company developing novel therapies for neuropsychiatric disorders, with a focus on muscarinic receptor-targeted treatments.

Oncology-focused drug development company based in Sydney, Australia, developing brain-penetrant and targeted therapies for cancers with high unmet need; its lead asset paxalisib was licensed from Genentech in 2016 and has been studied in more than 15 clinical trials.

Kyverna Therapeutics is a clinical-stage biopharmaceutical company developing autologous and allogeneic CAR T-cell therapies for autoimmune diseases. Kyverna is poised to become the first company to file a regulatory application for a CAR T-cell therapy in autoimmune disease.

Latigo Biotherapeutics is a clinical-stage biotechnology company developing non-opioid pain drugs that target sodium ion channels. The company recently outlined plans to go public on NASDAQ under ticker LTGO.

New York City-based clinical-stage genetic medicine company advancing disease-modifying gene therapies for genetic cardiovascular conditions and genetic conditions of the central nervous system; in September 2026 it agreed to acquire Mantle Therapeutics to add Friedreich ataxia assets.

Mentari is a biotechnology company focused on developing therapies for migraine and related neurological disorders.

NeuShen Therapeutics is a US-China biotech company focused on developing brain-health drugs. Founded by former I-Mab chief Joan Shen, the company has raised over $80 million to advance three neuroscience programs.

Ovid Therapeutics is a clinical-stage neurology biotech focused on developing treatments for rare epilepsies and seizure-related disorders. The soticlestat program is being spun out into a Perceptive Advisors-backed NewCo with Takeda.

Precision Neuroscience is a medical technology company developing brain-computer interface (BCI) technology. The company has demonstrated that people can control electronic devices with thought alone, without electrodes implanted in the brain.

Quanterix is a life-sciences company commercializing single-molecule array (Simoa) technology for ultra-sensitive protein biomarker detection, with platforms including the HD-X and SR-X instruments and the SP-X planar-array system. It operates in neurology, oncology, immunology, and inflammation, selling instruments, assays, and contract research services to academic, government, and pharmaceutical laboratories. Founded by David Walt in 2007 and listed on Nasdaq in 2017, the company acquired Akoya Biosciences in 2025 to add multiplexed spatial biology. In Q2 2026 it reported revenue up 34% year over year to $32.9 million while cutting its full-year 2026 revenue outlook.

Clinical-stage biopharmaceutical company that builds and launches modular subsidiary companies (Vants) developing transformative therapies across immunology, neurology, oncology, and rare disease.

Sangamo Therapeutics (NASDAQ: SGMO) is a genomic medicine company headquartered in Richmond, California, focused on translating ground-breaking science into genomic medicines using zinc finger nucleases (ZFNs), zinc finger protein transcription factors (ZFP-TFs), and adeno-associated virus (AAV) delivery. Sangamo's clinical-stage pipeline includes giroctocogene fitelparvovec (hemophilia A gene therapy, partnered with Pfizer) and isavufermin or SAR445136 (alpha-1 antitrypsin deficiency, partnered with Sanofi). The company is also developing preclinical in vivo genome-editing programs and ex vivo gene-edited cell therapies.

Roswell, GA-based rare disease biotech developing therapies for pain, osteoarthritis, spasticity, and mitochondrial disorders, with a strategic focus on underserved CNS indications.

Specialty pharmaceutical company focused on rare disease therapies. Develops and commercializes treatments for rare and ultra-rare diseases, including CUTX-101 (copper histidinate) for Menkes disease.

Discovery-based pharmaceutical company (Shionogi & Co., TYO:4507, Osaka, Japan, founded 1878) with US subsidiary marketing Fetroja (MDR Gram-negative antibiotic), Radicava ORS (ALS), and Xocova (COVID-19 antiviral).

Skyhawk Therapeutics is a clinical-stage biotechnology company developing small-molecule RNA splicing modulators using its proprietary SkySTAR platform, with programs in neurology, oncology, autoimmune, and fibrotic diseases.

UniXell Biotechnology Co., Ltd. is a Shanghai-based cell therapy company developing allogeneic induced pluripotent stem cell (iPSC)-derived therapies for neurological disorders. Its lead program UX-DA003, an allogeneic iPSC-derived midbrain dopaminergic progenitor cell therapy, secured FDA IND clearance in June 2026 paired with a parallel NMPA clearance — the first Chinese iPSC Parkinson's program to clear both agencies.