Transcripta Bio Announces Research Collaboration to Advance Discovery of New ALS Treatments

PALO ALTO, Calif.--(BUSINESS WIRE)--Transcripta Bio, an AI-powered drug discovery company focused on neurological and neuromuscular diseases, today announced a research collaboration and licensing agreement with Mayo Clinic centered on amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease with few effective treatments.

The collaboration plans to combine Mayo Clinic’s clinical and neuropathology expertise with Transcripta Bio’s transcriptomic drug-discovery platform to build a high-resolution, patient-derived molecular map of ALS and identify novel therapeutic targets. Using single-cell RNA sequencing on donated tissue provided by Mayo Clinic, Transcripta Bio will apply its platform to build patient-derived ALS disease signatures, matching them against the company's drug-response data to nominate novel therapeutic targets and candidate treatments. The work is designed to deepen understanding of ALS disease progression, generate insights that could inform clinical care, and accelerate the discovery of targeted treatments.

“Mayo Clinic is one of the country’s leading centers for ALS care and research, with exceptional clinical expertise and a deep understanding of how the disease progresses in patients,” said Chris Moxham, founder and CEO, Transcripta Bio. “We now have the opportunity to connect that clinical depth with molecular data derived directly from patient tissue, giving us a new way to understand the biology of ALS and identify therapeutic targets and potential treatments grounded in patients themselves.”

Mayo Clinic is prospectively enrolling living ALS patients under a newly established, IRB-approved protocol. The cohort is designed to span both familial ALS – including genetic backgrounds such as SOD1, C9orf72, TARDBP, and FUS – and sporadic ALS, paired with de-identified clinical metadata such as genetic status, medications, rate of progression, age at onset, and disease duration. Bringing together patient-derived tissue with genetic and clinical information has strong potential to enable a rich analysis of how ALS biology varies across patients and over the course of disease, helping identify molecular patterns that can point to new therapeutic targets and potential treatments.

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.