Transcripta Bio Announces $24 Million in Funding to Advance AI-Powered Drug Discovery Platform for Neurological and Neuromuscular Diseases

PALO ALTO, Calif.--(BUSINESS WIRE)--Transcripta Bio, an AI-powered drug discovery company focused on neurological and neuromuscular diseases, today announced it has raised $24 million since its Series A to advance IND-enabling studies across its portfolio and support clinical preparation in its Autism Spectrum Disorder (ASD) and Facioscapulohumeral Muscular Dystrophy (FSHD) programs. Mayo Clinic and Omnimed join existing investors JAZZ Venture Partners, BlueYard Capital, and a group of life sciences family offices as the company advances its pipeline toward human clinical trials. The company's pipeline spans four programs across Myotonic Dystrophy, FSHD, ASD, and Huntington's Disease (HD).

Transcripta Bio's closed-loop discovery engine is designed to use transcriptomic signature matching to identify drugs whose molecular signatures are the inverse of patient-derived disease signatures. The platform combines a Disease Signature Atlas built from actual patient single-cell RNA sequencing data, a Drug-Gene Atlas mapping the full transcriptomic response of FDA-approved and novel compounds, and Conductor AI, a machine learning system trained on more than one billion gene responses that begins at lead optimization rather than hit identification. Together, these three components systematically assess compounds, with the goal of de-risking drug development by addressing the root causes of clinical failure before candidates ever reach the clinic.

"Transcripta was founded to address the failure points that keep drugs from reaching the clinic. Our platform has already demonstrated results and this capital accelerates that work for diseases that have waited long enough for real treatments," said Chris Moxham, founder and CEO, Transcripta Bio. "Transcripta Bio is positioned to demonstrate what AI-native drug discovery can deliver for patients with neurological and neuromuscular diseases."

"Transcripta Bio has developed a discovery platform grounded in patient biology and applied it across four programs with significant unmet need," said Greg Wood, Managing Director, Omnimed. "We look forward to seeing what they can accomplish moving forward."

Mayo Clinic has a financial interest in the technology referenced in this press release. Mayo Clinic will use any revenue it receives to support its not-for-profit mission in patient care, education and research.

About Transcripta Bio
Transcripta Bio is an AI-powered drug discovery company developing treatments for neurological and neuromuscular diseases. The company's closed-loop discovery engine uses transcriptomic signature matching to identify drug candidates whose molecular signatures are the inverse of patient-derived disease signatures, enabling a faster, more capital-efficient path to the clinic. Transcripta Bio's pipeline spans four programs including Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy (FSHD), Autism Spectrum Disorder (ASD), and Huntington's Disease (HD). The company is based in Palo Alto, California. For more information, visit transcriptabio.com.