eClinical Technology and Industy News

NeuBase Presents New Preclinical Data at ASGCT 2022 for Its DM1 Program Demonstrating Wide Tissue Distribution and Supporting a Differentiated Whole-Body Treatment Solution

Excerpt from the Press Release:

PITTSBURGH and CAMBRIDGE, Mass., May 17, 2022 (GLOBE NEWSWIRE) — NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology platform company Drugging the Genome™ to address disease at the base level using a new class of precision genetic medicines, today announced the presentation of preclinical pharmacokinetics (PK) and biodistribution data for its lead development candidate, NT-0231.F, supporting a differentiated whole body treatment solution for myotonic dystrophy type 1 (DM1). These new data are being presented today in a poster session at the American Society of Gene and Cell Therapy (“ASGCT”) 25th Annual Meeting, taking place virtually and in person in Washington, D.C., May 16-19, 2022.

Following a single intravenous (IV) injection of 30 mg/kg in wild-type BALB/c mice, NT-0231.F was cleared rapidly from the systemic compartment and demonstrated rapid and wide distribution into tibialis anterior muscle, heart muscle, and brain tissues. NT-0231.F rapidly cleared the plasma, and each tissue evaluated displayed an extended elimination phase with tissue concentrations measurable for at least four weeks following a single IV dose administration.

Sandra Rojas-Caro, M.D., Chief Medical Officer of NeuBase, said, “We continue to build a robust and compelling data set potentially supporting a whole body treatment solution for DM1. These PK and biodistribution data presented today show IV administration of our lead candidate results in exposure in the major tissues that are affected by DM1.

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