Companies in #Neurology

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.

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

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.

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.

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

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.

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.

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.