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.

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.

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.

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.