Transcripta Bio has raised $24 million since its Series A financing to advance an AI-powered drug discovery platform focused on neurological and neuromuscular diseases. New investors Mayo Clinic and Omnimed joined existing backers JAZZ Venture Partners, BlueYard Capital and several life sciences-focused family offices.
The Palo Alto, California-based company plans to use the capital for investigational new drug-enabling studies across its portfolio. It will also prepare its autism spectrum disorder and facioscapulohumeral muscular dystrophy programs for clinical development.
IND-enabling studies are the preclinical safety, manufacturing and pharmacology studies typically required before a company can request authorization to begin testing a drug candidate in humans.
Transcripta Bio’s pipeline includes four programs targeting myotonic dystrophy, facioscapulohumeral muscular dystrophy, autism spectrum disorder and Huntington’s disease.
The company’s closed-loop discovery engine uses transcriptomic signature matching to identify compounds that could reverse molecular patterns associated with a disease. Transcriptomics involves studying how genes are expressed within cells and tissues.
Transcripta compares patient-derived disease signatures with the molecular effects produced by existing and experimental compounds. The goal is to identify drugs whose gene-expression patterns move in the opposite direction from those associated with a disease.
The platform consists of three primary components. Its Disease Signature Atlas is built from single-cell RNA sequencing data collected from patients and is intended to capture disease-related biological changes across specific cell types.
The Drug-Gene Atlas maps how FDA-approved and novel compounds affect gene expression. This information allows Transcripta to compare a drug’s molecular effects with the biological signatures observed in patients.
Conductor AI is a machine learning system trained on more than one billion gene responses. Transcripta said the system begins at lead optimization rather than the earlier hit-identification stage, potentially allowing the company to prioritize candidates that already show promising biological characteristics.
Lead optimization involves refining a potential drug to improve properties such as potency, selectivity, safety and suitability for clinical development.
Transcripta believes combining patient biology, compound-response data and machine learning can identify development risks before candidates enter clinical trials. However, the company’s programs remain subject to preclinical testing, regulatory authorization and clinical studies establishing safety and effectiveness.
Mayo Clinic has a financial interest in technology referenced by Transcripta Bio. The organization said any related revenue it receives will support its nonprofit work in patient care, education and research.
KEY QUOTES:
“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.”
“Transcripta Bio is positioned to demonstrate what AI-native drug discovery can deliver for patients with neurological and neuromuscular diseases.”
Chris Moxham, Founder and CEO of Transcripta Bio
“Transcripta Bio has developed a discovery platform grounded in patient biology and applied it across four programs with significant unmet need.”
“We look forward to seeing what they can accomplish moving forward.”
Greg Wood, Managing Director of Omnimed
