Transcripta Bio has entered into a research collaboration and licensing agreement with Mayo Clinic to identify potential treatments and therapeutic targets for amyotrophic lateral sclerosis (ALS).
The initiative centres on creating a high-resolution, patient-derived molecular map of the fatal neurodegenerative disorder, which currently has few effective therapies.
Under the terms of the agreement, Mayo Clinic is prospectively recruiting living ALS patients through an Institutional Review Board-approved protocol.
The study group includes individuals with sporadic ALS as well as familial forms of the disease, spanning genetic profiles such as chromosome 9 open reading frame 72 (C9orf72), fused in sarcoma (FUS), superoxide dismutase 1 (SOD1), and TAR DNA-binding protein (TARDBP).
Donated patient tissue will undergo single-cell RNA sequencing and be evaluated alongside anonymised clinical information, including age at onset, disease duration, progression rates, genetic status, and prescribed medications.
Transcripta Bio plans to apply its transcriptomic drug-discovery platform to the tissue samples to generate patient-derived disease profiles.
By comparing these signatures against its existing drug-response data, the company seeks to nominate new intervention targets and prospective drug candidates.
The work aims to elucidate how ALS biology varies across individual patients and throughout disease progression, while generating findings that could inform patient care and accelerate the development of targeted therapies.
Transcripta Bio founder and CEO Chris Moxham said: “Mayo Clinic is one of the country’s leading centres for ALS care and research, with exceptional clinical expertise and a deep understanding of how the disease progresses in patients.
“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.”
Headquartered in Palo Alto, California, US, Transcripta Bio is an AI-driven biotechnology company focused on treatments for neurological and neuromuscular illnesses.
Its discovery platform matches disease profiles with opposing compound signatures to identify prospective drugs.
In addition to its ALS research, the company maintains discovery programmes in Autism Spectrum Disorder (ASD), Facioscapulohumeral Muscular Dystrophy (FSHD), Huntington's Disease (HD), and Myotonic Dystrophy.
Earlier this year, Merck & Co (MSD) and Mayo Clinic entered a research and development collaboration to utilise AI, advanced analytics, and multimodal clinical data in support of drug discovery and precision medicine.
