Illimis Therapeutics is a Seoul-based biotechnology company developing protein-based medicines for central nervous system and immune diseases. Its proprietary GAIA (Gas6-mediated Anti-Inflammatory Adaptor) platform builds bispecific fusion proteins that engage the body's TAM receptors to clear misfolded proteins such as amyloid-beta through phagocytosis without triggering the inflammation and ARIA side effects associated with existing antibody drugs. Founded in 2021, the company raised a $42 million Series B in July 2025 and is advancing a lead Alzheimer's candidate, ILM01, toward an IND by the end of 2027.
NeuShen Therapeutics is a clinical-stage biotech developing novel small-molecule and gene-therapy treatments for central nervous system disorders such as schizophrenia, depression and epilepsy. Founded in 2021 by Joan Huaqiong Shen, MD, PhD, and headquartered in Shanghai with an R&D center in the Boston area, the company runs a pipeline led by NS-136, a selective M4 receptor positive allosteric modulator for schizophrenia, and NS-041, a highly selective KCNQ2/3 activator for epilepsy and depression.
Prosetta Biosciences is a San Francisco biotech using a proprietary cell-free protein synthesis and assembly (CFPSA) platform to discover small-molecule 'assembly modulators' - drugs that disrupt the multi-protein complexes behind viral infection, cancer, and neurodegeneration. Founded by UCSF physician-scientist Vishwanath Lingappa, the company is advancing candidates against ALS/FTD, Alzheimer's, pan-cancer targets, and antivirals.
Switch Therapeutics is a South San Francisco preclinical biotech building conditionally activated siRNA (CASi) therapies that only switch on inside the cell types where they're needed. The platform - born from research at Caltech, Harvard and City of Hope - aims to make RNA interference safe enough for the brain, with a lead program targeting APOE for Alzheimer's disease.
Autobahn Therapeutics is a San Diego clinical-stage biotech building small-molecule medicines that reach the brain on purpose. Using a brain-targeting chemistry and prodrug platform, the company tunes where a drug goes - central versus peripheral - to unlock validated CNS biology that has been hard to drug safely. Its lead program, elunetirom (ABX-002), is an oral, once-daily, brain-penetrant CNS thyroid hormone receptor agonist in Phase 2 trials for major depressive disorder and bipolar depression, backed by more than $200 million in venture funding and a 2026 FDA Fast Track designation.
CaaMTech is an Issaquah, Washington drug discovery company building patentable, psychedelic-inspired medicines. Founded in 2016 by chemist and patent attorney Andrew Chadeayne, it has synthesized and characterized a large library of novel tryptamines, solved crystal structures of compounds like 4-AcO-DMT and norpsilocin, and filed more than 100 patent applications. The company studies the 'entourage effect' in magic mushrooms to engineer compounds that keep the benefits while reducing side effects, and in 2021 raised a $22M Series A to push lead candidates toward human trials.
Noema Pharma is a Basel-based clinical-stage biotech building first-in-disease oral small-molecule therapeutics for central nervous system disorders. Founded in 2019 by Sofinnova Partners around four mid-stage assets in-licensed from Roche, the company targets overlooked neurological conditions - from seizures in tuberous sclerosis complex and pain in trigeminal neuralgia to Tourette syndrome and CNS-mediated symptoms of menopause. It has raised roughly CHF 130 million (about USD 147 million) in Series B financing from a syndicate including Forbion, Jeito Capital, Sofinnova, EQT Life Sciences and UPMC Enterprises.
Ilise Lombardo is the CEO of Noema Pharma, a Basel-based clinical-stage biotech advancing treatments for central nervous system disorders. A Yale-trained psychiatrist with a Cambridge M.Phil and a Brown degree, she spent two decades in clinical practice and academia before co-founding Arvelle Therapeutics, which sold to Angelini Pharma for roughly $1 billion in 2021. She now steers Noema's pipeline of Phase 2 programs targeting Tourette syndrome, trigeminal neuralgia, tuberous sclerosis seizures and menopause-related CNS symptoms.

Tom Large is the CEO and co-founder of Blue Oak Pharmaceuticals, a Waltham, Massachusetts startup hunting for the next generation of medicines for brain disorders. A neurobiologist by training, he spent two decades inside big pharma - leading neuroscience research at Eli Lilly and running preclinical research and translational medicine at Sunovion - before launching Blue Oak in 2016 to chase drugs with genuinely new mechanisms for bipolar depression, schizophrenia and treatment-resistant depression. His teams pioneered a target-agnostic approach to CNS drug discovery, and he later partnered with AI specialist Exscientia to design 'bispecific' small molecules that hit two targets at once.
Blue Oak Pharmaceuticals is a Waltham, Massachusetts biotech founded in 2016 to discover the next generation of drugs for brain disorders. Led by neurobiologist and former Eli Lilly and Sunovion executive Tom Large, the company designs novel, CNS-focused 'privileged chemotypes' and pairs them with systems-neurobiology behavioral assays and AI to hunt first-in-class small molecules for schizophrenia, bipolar disorder and treatment-resistant depression. Its work runs through partnerships with phenotypic-screening firm PsychoGenics and AI drug-design company Exscientia.
Leal Therapeutics is a Worcester, Massachusetts biotech founded in 2021 by repeat CNS entrepreneur Asa Abeliovich, the scientist behind Prevail Therapeutics (acquired by Eli Lilly). Leal builds first-in-class neuro-metabolic medicines on a single idea: many brain diseases share a broken metabolism, and correcting those imbalances can treat conditions from schizophrenia to ALS. Backed by roughly $114M in total funding, its pipeline includes LTX-001, a brain-penetrant oral glutaminase inhibitor in the clinic, and LTX-002, an antisense oligonucleotide for ALS.