Transcripta Bio Raises $24M for AI-Driven Neurological and Neuromuscular Therapies

Transcripta Bio, a Palo Alto, Calif.-based AI-driven drug discovery company, has raised $24 million in new financing backed by Mayo Clinic and Omnimed to accelerate development of experimental therapies for neurological and neuromuscular diseases. The company said the capital will fund an investigational new drug and prepare lead programs in autism spectrum disorder (ASD) and facioscapulohumeral muscular dystrophy (FSHD) for human clinical trials. Existing investors Jazz Venture Partners, BlueYard Capital, and a group of life sciences family offices also participated.

Unlike many companies that primarily use AI to identify biological targets, Transcripta combines computational analysis with laboratory validation through a closed-loop drug discovery platform. The system compares gene expression patterns from patients with the molecular signatures produced by thousands of compounds, searching for drugs capable of reversing disease-associated genetic activity.

The company's proprietary platform uses patient-derived single cell RNA-seq (scRNA-seq) data to identify disease signatures. An in-house generated "drug atlas" measures the effects of small molecule perturbations across 80% of the transcriptome, capturing full dose-response profiles in diverse cellular contexts, including glutamatergic and motor neurons, fibroblasts, and keratinocytes. These data power AI models that identify promising compounds that can therapeutically modulate gene expression.

In 19q12 syndrome, a form of ASD, the company demonstrated that entrectinib, an FDA-approved oncology drug, could reverse disease when given at low concentrations. One patient case demonstrated clinical benefit within nine months after taking the drug. In Huntington's disease, the platform identified novel molecules that could downregulate DNA mismatch repair protein and validated therapeutic target MSH3, and the company plans to file an IND next year. Transcripta is also pursuing pre-IND research in FSHD and myotonic dystrophy.

Last year, Transcripta entered a collaboration with SOLVE FSHD to screen approved and late-stage compounds for potential use in treating FSHD. Founded in 2023, the company currently houses fifteen employees. Its pipeline is structured as a tiered portfolio of novel molecules and repurposed clinical-stage assets, with the latter benefiting from existing human safety data that can shorten development timelines and lower costs.

The company's founder and CEO, who has spent more than 25 years in pharmaceutical research and drug discovery, said the platform has already demonstrated results and that this capital accelerates work for diseases that have waited long enough for real treatments. He emphasized that de-risking therapies early is key to improving drug discovery success rates, which often fall below 10%. Transcripta plans to introduce another cohort of therapeutic programs by Q2 of 2027.