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Locanabio Announces Upcoming Preclinical Data Presentations at American Society of Gene and Cell Therapy Annual Meeting News provided by Share this article Share this article 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 ....