Transcripta Bio Announces Research Collaboration to Advance Discovery of New Treatments for Autism Spectrum Disorder

PALO ALTO, Calif.--(BUSINESS WIRE)--Transcripta Bio, an AI-powered drug discovery company focused on neurological and neuromuscular diseases, today announced a know-how agreement with Mayo Clinic focused on the molecular characterization of autism spectrum disorder (ASD). The collaboration seeks to combine Mayo Clinic's clinical genomics and neurodevelopmental expertise with Transcripta Bio's transcriptomic drug discovery platform to investigate the molecular mechanisms underlying ASD and identify biologically defined patient subgroups that could inform the development of targeted treatments.

Autism spectrum disorder is a complex neurodevelopmental condition with significant genetic and biological diversity. While researchers have identified numerous genetic variants associated with ASD, many cases have no identified genetic cause, making it challenging to develop treatments targeting specific underlying mechanisms. The collaboration focuses on attempting to characterize transcriptomic subtypes of autism, or subgroups of patients defined by shared biology at the level of altered gene expression rather than by an individual genetic variant.

"Major medical breakthroughs in therapeutic discovery have occurred as a result of targeting cell populations in a given disease that share similar gene expression patterns and downstream biological pathways despite having distinct or divergent underlying genetic mutations," said Chris Moxham, CEO of Transcripta Bio. "This collaboration aims to apply that same precision medicine approach to the diversity of phenotypes that constitute Autism spectrum disorder by combining deep molecular characterization with our transcriptomic drug discovery platform, to ultimately identify effective therapeutics defined by this concept of transcriptomic convergence.”

Mayo Clinic will support the design and execution of a prospective molecular characterization study integrating whole-genome sequencing, RNA sequencing and clinical phenotyping of patients with ASD. These complementary analyses examine genetic variation, patterns of gene expression and clinical characteristics together. The study focuses on characterizing transcriptomic subtypes of autism and examining biological differences that may not be apparent through genetic analysis alone.

Transcripta Bio previously published peer-reviewed research in Scientific Reports demonstrating the application of its transcriptomic drug discovery platform to identify a potential therapeutic candidate for a genetically defined form of ASD.

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.