neurodevelopmental-disorders
(3)
Axonis Therapeutics
Axonis Therapeutics is a Boston-based, neuro-focused biotechnology company developing first- and best-in-class oral small-molecule medicines that target KCC2, a chloride transporter that acts as the brain's master regulator of inhibitory neurotransmission. Spun out of research from Boston Children's Hospital, Harvard Medical School and Universite Laval, Axonis is advancing its lead candidate AXN-027 - a first-in-class KCC2 potentiator - toward clinical proof of concept in epilepsy, chronic pain and other CNS disorders, backed by an oversubscribed $115 million Series A closed in 2024.
Unravel Biosciences
Unravel Biosciences is a clinical-stage therapeutics company spun out of Harvard's Wyss Institute that starts with a patient's RNA rather than a drug target. Its BioNAV probabilistic network model of human health screens 40,000-plus compounds in silico - reporting roughly 200x the hit rate of conventional screens - to find existing molecules that can push a disease back toward health. The company validates predictions in engineered whole-organism models (including CRISPR-edited tadpoles) and then in patients, aiming to bring effective, lower-cost therapies to rare and overlooked diseases. Its lead asset, RVL-001 (a proprietary formulation of vorinostat), holds FDA Orphan Drug Designation for Rett syndrome and has entered clinical studies.
Newleos Therapeutics
Newleos Therapeutics is a Boston-based, clinical-stage biotechnology company building a new generation of treatments for neuropsychiatric disorders. Launched in February 2025 with an oversubscribed $93.5 million Series A, the company in-licensed four clinical-ready oral small molecules from Roche - targeting GABAA-y1, V1a, TAAR1, and GABAA-a5 - aimed at generalized anxiety, social anxiety, substance use disorders, and cognitive impairment. Its name nods to 'eos,' the Greek word for dawn: a new dawn for mental health treatment.