California-based Transcripta Bio, an AI-powered drug discovery company focused on neurological and neuromuscular diseases, has raised USD 24 million in follow-on funding to its Series A round, capital it will use to complete IND-enabling studies and prepare for clinical trials in autism spectrum disorder (ASD) and facioscapulohumeral muscular dystrophy (FSHD).
Mayo Clinic and Omnimed joined the round as new investors alongside existing backers JAZZ Venture Partners, BlueYard Capital, and a group of life sciences family offices; no lead investor was specified. Mayo Clinic holds a financial interest in the technology referenced in the announcement and said proceeds would support its research mission. The raise follows a USD 10 million Series A closed in April 2024, bringing Transcripta's total disclosed funding to roughly USD 34 million. The company has also struck non-equity collaborations relevant to its pipeline, including a March 2025 partnership with the patient advocacy group SOLVE FSHD to fund drug screening for FSHD, and a research collaboration announced in May 2025 with Microsoft Research to expand its AI-driven gene discovery work.
Transcripta's platform, built around what it calls a closed-loop discovery engine, uses transcriptomic signature matching to identify compounds whose molecular effects are the inverse of disease-specific gene expression patterns derived from patient single-cell RNA sequencing data. The approach draws on a Disease Signature Atlas, a Drug-Gene Atlas mapping the transcriptomic response of FDA-approved and experimental compounds, and Conductor AI, a machine learning system trained on more than one billion gene responses. The company's furthest-advanced program involves entrectinib, an FDA-approved oncology drug being repositioned for a genetically defined ASD subtype linked to 19q12 deletions, an application the company has described in a peer-reviewed publication. Its FSHD program targets fibro-adipogenic progenitor cells implicated in disease pathology, with SOLVE FSHD backing the screening work. Two earlier-stage programs address myotonic dystrophy and Huntington's disease. Founder and CEO Chris Moxham, who previously worked on transcriptomics-driven discovery at Eli Lilly and Fulcrum Therapeutics, said the funding would accelerate efforts to bring candidates toward the clinic across the four-program pipeline.
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