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Anderson Laboratory Awarded Research Funding for Trans-Splicing Technology

Thursday, January 6, 2022

The Anderson Lab has been awarded over $850,000 in research funding through Scriptr Global Inc. Douglas Anderson, Ph.D., assistant professor of Medicine, Aab Cardiovascular Research Institute, is the principal investigator. The funding will be provided through October 2023.

The funding will go towards developing a novel RNA trans-splicing technology, called StitchR, into a therapeutic approach for treating Duchenne Muscular Dystrophy (DMD) and other dystrophinopathies. StitchR, which stands for ‘Stitch RNA’, arose from the team’s serendipitous discovery that when two independent RNAs are cleaved by ribozymes (small self-cleaving RNAs), the resulting RNAs become trans-spliced, or ‘stitched’ together. Remarkably, if two halves of a protein-coding mRNA are used, the subsequent ‘stitched’ mRNA will be translated and express the full-length protein in cells. For this sponsored research agreement, Anderson’s group is using StitchR to develop a dual AAV-based gene therapy to deliver and express a large functional copy of the Dystrophin gene, which is normally too large to be encoded within the packaging limits of a single AAV vector. This project brings together their background in RNA and muscle biology, with the ultimate goal of making a positive impact for patients suffering with dystrophinopathies.

This work will also help validate the potential for StitchR-enabled AAV gene therapies to be utilized for other human diseases which occur from mutations in very large genes, such as hemophilia A, cystic fibrosis, dyferlinopathies, macular degeneration, and others.

Disclosures: The StitchR technology has been licensed from the University of Rochester to Scriptr Global, Inc., for which Douglas M. Anderson, Ph.D. is Chief Scientific Officer and Co-Founder.