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Locanabio Announces Upcoming Preclinical Data Presentations at American Society of Gene and Cell Therapy Annual Meeting


Locanabio Announces Upcoming Preclinical Data Presentations at American Society of Gene and Cell Therapy Annual Meeting
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SAN DIEGO, May 5, 2021 /PRNewswire/ -- Locanabio, Inc., an RNA-targeting gene therapy company focused on developing life-changing therapies for patients with severe neurodegenerative, neuromuscular and retinal diseases, today announced that new data from its preclinical research will be highlighted in oral and poster presentations at the 24th Annual American Society of Gene and Cell Therapy (ASGCT) Meeting, taking place virtually from May 11-14, 2021. The presentations include results from a preclinical
in vivo study showing a novel PUF RNA-binding protein system delivered via an adeno-associated virus serotype 9 (AAV9) vector is safe and effective in eliminating toxic CUG repeats in a mouse model of myotonic dystrophy type 1 (DM1). Data also provide insights on the potential of a unique CRISPR/Cas13d gene therapy system for skipping exon 13 mutations in the USH2A gene that are associated with usher syndrome type 2 and non-syndromic autosomal recessive retinitis pigmentosa.

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