Transcripta Bio and Mayo Clinic have teamed up to uncover new therapeutic targets for amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease with few effective treatments.
The research collaboration and licensing agreement will combine Mayo Clinic’s clinical and neuropathology expertise with Transcripta Bio’s AI-powered drug discovery platform to build a detailed molecular map of ALS and identify potential new treatments.
The initiative will use single-cell RNA sequencing to analyse donated patient tissue, generating molecular signatures of ALS that Transcripta Bio will compare against its drug-response data. The goal is to pinpoint disease-driving mechanisms, identify promising therapeutic targets and nominate candidate treatments.
The research could also shed new light on how ALS progresses and why its underlying biology varies from patient to patient, potentially opening new avenues for targeted treatment.
“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, institutional review board-approved protocol. The study will include both familial ALS, associated with genetic backgrounds such as SOD1, C9orf72, TARDBP and FUS, and sporadic ALS, which occurs without a clear inherited cause.
Researchers will pair tissue-derived molecular data with de-identified clinical information, including genetic status, medications, age at onset, disease duration and rate of progression.
By linking these clinical details with molecular patterns, the collaboration aims to identify biological differences across patients and disease stages. Such insights could help researchers pinpoint new drug targets and guide the development of treatments tailored to the biology of individual patients.
The work remains at the research and discovery stage; the announcement does not identify a specific drug candidate or establish that any resulting treatment will be effective in patients.
