NeuroNOS
Biopharmaceutical company developing small-molecule, brain-penetrant therapies that modulate neuronal nitric oxide (NO) signaling for neurodevelopmental and neurodegenerative disorders (notably autism spectrum disorder, Alzheimer’s disease, and glioblastoma). The company conducts preclinical validation in rodent and human stem-cell models, is advancing formulation and manufacturing partnerships, is preparing IND-enabling studies and first-in-human trials, and collaborates with academic technology transfer and research partners.
Industries
Nr. of Employees
small (1-50)
NeuroNOS
Boston, Massachusetts, United States, North America
Products
Lead small-molecule CNS-penetrant therapeutic candidates (injectable and oral formulations)
Investigational small molecules designed to modulate neuronal nitric oxide production in the brain; being advanced through preclinical validation, formulation development (subcutaneous and prospective oral), and IND-enabling work.
BA-101
Lead investigational therapy by NeuroNOS granted FDA Orphan Drug Designation for the treatment of Glioblastoma, an aggressive primary brain tumor with limited treatment options and poor prognosis.
NeuroNOS small-molecule therapy
A novel drug for Autism Spectrum Disorder (ASD) and other neurological conditions designed for subcutaneous injection or oral administration to regulate nitric oxide (NO) levels in the brain, showing significant promise in preclinical studies.
BA-102
Lead investigational therapy granted FDA Orphan Drug Designation for the treatment of Phelan-McDermid Syndrome, a neurodevelopmental disorder linked to Autism Spectrum Disorder. Planned first-in-human clinical trials in the U.S. in 2026.
BA102
Lead compound developed by NeuroNOS targeting neuronal nitric oxide synthase (nNOS) to reduce nitrosative stress and treat core symptoms of Autism Spectrum Disorder (ASD).
Lead small-molecule CNS-penetrant therapeutic candidates (injectable and oral formulations)
Investigational small molecules designed to modulate neuronal nitric oxide production in the brain; being advanced through preclinical validation, formulation development (subcutaneous and prospective oral), and IND-enabling work.
BA-101
Lead investigational therapy by NeuroNOS granted FDA Orphan Drug Designation for the treatment of Glioblastoma, an aggressive primary brain tumor with limited treatment options and poor prognosis.
NeuroNOS small-molecule therapy
A novel drug for Autism Spectrum Disorder (ASD) and other neurological conditions designed for subcutaneous injection or oral administration to regulate nitric oxide (NO) levels in the brain, showing significant promise in preclinical studies.
BA-102
Lead investigational therapy granted FDA Orphan Drug Designation for the treatment of Phelan-McDermid Syndrome, a neurodevelopmental disorder linked to Autism Spectrum Disorder. Planned first-in-human clinical trials in the U.S. in 2026.
BA102
Lead compound developed by NeuroNOS targeting neuronal nitric oxide synthase (nNOS) to reduce nitrosative stress and treat core symptoms of Autism Spectrum Disorder (ASD).
Expertise Areas
- NO/nNOS-targeted neurotherapeutics
- Preclinical behavioral pharmacology
- Translational stem cell modeling
- Proteomics and nitrosylation biology
Key Technologies
- nNOS inhibition (nitric oxide modulation)
- Small-molecule CNS-penetrant drug design
- Rodent behavioral assays (elevated plus maze, novel object recognition, sociability assays)
- Patient-derived stem cell neuronal models
News & Updates
U.S. FDA granted Orphan Drug Designation to the company's lead investigational therapy for Phelan-McDermid Syndrome; company planning first-in-human ASD trials in 2026.
Peer-reviewed research by the company's CSO reporting shared NO-related molecular mechanisms in Alzheimer’s disease mouse models and suggesting NO modulation as a therapeutic strategy.
Raised initial $2.0 million in private equity to accelerate preclinical and formulation development; engaged a U.S.-based contract manufacturer.
Appointments of Prof. Roger D. Kornberg and Prof. Dan Shechtman to the Scientific Advisory Board to provide molecular and structural biology expertise.
U.S. FDA granted Orphan Drug Designation to a lead investigational therapy for glioblastoma, expanding the company’s oncology-focused development.
Publications include studies on nitric oxide’s role in ASD, proteomics revealing mTOR alterations, and preclinical evaluations of nNOS inhibitors and formulations.
U.S. FDA granted Orphan Drug Designation to the company's lead investigational therapy for Phelan-McDermid Syndrome; company planning first-in-human ASD trials in 2026.
Peer-reviewed research by the company's CSO reporting shared NO-related molecular mechanisms in Alzheimer’s disease mouse models and suggesting NO modulation as a therapeutic strategy.
Raised initial $2.0 million in private equity to accelerate preclinical and formulation development; engaged a U.S.-based contract manufacturer.
Appointments of Prof. Roger D. Kornberg and Prof. Dan Shechtman to the Scientific Advisory Board to provide molecular and structural biology expertise.
U.S. FDA granted Orphan Drug Designation to a lead investigational therapy for glioblastoma, expanding the company’s oncology-focused development.
Publications include studies on nitric oxide’s role in ASD, proteomics revealing mTOR alterations, and preclinical evaluations of nNOS inhibitors and formulations.