RNA-based Gene Therapy Reverses Molecular and Functional Features of Myotonic Dystrophy Type 1 in Mice

By StartUp City | Tuesday, September 15, 2020

Preclinical data published today in Nature Biomedical Engineering shows a reversal of disease phenotypes, indicating the potential of RNA-targeting systems as treatments for DM1 and other RNA mediated diseases

FREMONT, CA: Locanabio, a pioneer in RNA-targeted gene therapy, announced that the results from a preclinical study of the company's therapeutic systems for the potential treatment of myotonic dystrophy type 1 (DM1) were published in Nature Biomedical Engineering

Scientists at Locanabio, collaborating with academics at UC San Diego School of Medicine and the University of Florida, skilfully assessed whether an RNA-targeting CRISPR Cas9 system (RCas9) would provide the molecular and functional rescue of dysfunctional RNA processing in a DM1 mouse model. The RCas9 system was tested after administered with one dose of an AAV gene therapy vector. Results observed in both adult and neonatal mice and the use of intramuscular and systemic delivery displayed continued as the RCas9 expression even at three months’ post-injection with efficient reversal of molecular and physiological (myotonia) features of DM1. Notably, It is of no significant adverse responses to the treatment.

“These results are consistent with earlier findings from several in vitro studies in muscle cells derived from DM1 patients published by Locanabio’s scientific co-founder Dr. Gene Yeo of UC San Diego and further indicate the significant potential of our RNA-targeting gene therapy as a DM1 treatment,” said Jim Burns, Ph.D., Chief Executive Officer at Locanabio. “Data show that our RNA-targeting system is able to destroy the toxic RNA at the core of this devastating genetic disease and thereby correct the downstream molecular and biochemical changes that result in myotonia, which is a hallmark symptom of DM1. We are pleased that Nature Biomedical Engineering recognizes the value of these preclinical data and we look forward to further advancing this developmental program to the benefit of DM1 patients.”

“Currently available treatments for DM1 can improve specific symptoms but do not target the underlying biology and cause of the disease. These data demonstrate that RNA-targeting systems may efficiently and specifically eliminate toxic RNA repeats that cause DM1 and potentially lead to a more effective treatment option for patients,” said Dr. Yeo. “The results also indicate that RNA-targeting gene therapy has potential applications in the treatment of other diseases, such as Huntington’s disease and certain genetic forms of ALS, which are also caused by a buildup of toxic RNA repeats.”

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