“Additionally, the data demonstrate our ability to preferentially target the toxic, mutant allele and preserve DMPK RNA generated from the wild-type allele, which is one of several distinguishing features of our CORRECTx platform. These data demonstrate a robust reduction of toxic RNA foci, a hallmark of this repeat expansion disorder, in the nucleus as well as the correction of splicing and a significant reduction of myotonia with Cas13d and PUF constructs via different mechanisms of action,” said John Leonard, Ph.D., chief scientific officer at Locanabio. “We are pleased with the continued progress of our DM1 program.
Using its CORRECTxTM platform, Locanabio scientists demonstrated a dose-dependent reduction in toxic CUG foci in both DM1 patient muscle cells and in a preclinical mouse model of DM1 that led to a correction in alternative RNA splicing and a statistically significant reduction in myotonia, or muscle weakness. SAN DIEGO, Novem– Locanabio, Inc., a genetic medicines company developing therapeutics for patients with severe neuromuscular, neurodegenerative and retinal diseases, today announced the presentation of new preclinical data from its myotonic dystrophy type 1 (DM1) program. – Company is advancing multiple DM1 leads into non-human primate studies in 2022 – New data show allele-specific reduction of CUG repeats and corrected splicing and myotonia in HSALR mice across multiple leads Locanabio Presents New Preclinical Data from Myotonic Dystrophy Type 1 Program at Society for Muscle Biology Frontiers in Myogenesis Conference 2021
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