Benzinga | Jun 8, 2021 12:30PM EDT

NeuBase Therapeutics Says Demonstrates Functional Rescue In Myotonic Dystrophy Type 1 Model And Allele-Selective Inhibition Of KRAS Mutations To Inhibit Tumor Growth

NeuBase Therapeutics, Inc. (NASDAQ:NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of precision genetic medicines, today announced positive new data and program updates for its development pipeline of PATrOL-enabled genetic medicines.

Dietrich A. Stephan, Ph.D., Chairman and CEO of NeuBase Therapeutics, said, "The data presented today from several programs in our pipeline highlight the unique potential of our PATrOL technology platform to generate new precision genetic medicines for a wide range of diseases with an incomparable flexibility to target distinctive gene dysfunctions. We successfully demonstrated that our platform can therapeutically modulate in vivo gene function at the DNA or RNA level with exquisite precision of target engagement in three different disease indications with different mechanisms of disease. These data show how we can utilize the PATrOL platform to design drug candidates to inhibit DNA transcription, RNA translation, and mutant protein production, as well as displace bound proteins to rescue mis-splicing."

Dr. Stephan continued, "We have made significant progress since our last data release, showing functional rescue in myotonic dystrophy type 1 after subcutaneous dosing, in vivo proof of concept in Huntington's disease with allele-selective mutant protein knock-down after getting compounds across the blood-brain barrier following systemic dosing, and initial data for a new oncology program against historically 'undruggable' KRAS driver mutations. With this tremendous momentum building across our expanding development pipeline, we look forward to entering the clinic with our DM1 program in 2022 and continuing to evaluate diseases where our PATrOL platform can deliver significant therapeutic value."