On February 28, 2022 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, reported that new preclinical data from its myotonic dystrophy type 1 (DM1) program will be featured in presentations at the 2022 MDA Clinical & Scientific Conference (Press release, NeuBase Therapeutics, FEB 28, 2022, View Source [SID1234609138]). MDA 2022 will be taking place virtually and in-person in Nashville, Tennessee, from March 13-16, 2022, and the abstracts will be available on the meeting website.
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Details of the presentations are as follows:
Title: Pharmacology, Biodistribution and Tolerability of a PATrOL-Enabled Investigational Genetic Therapy for Myotonic Dystrophy, Type 1
Type: Poster Presentation
Date: Available for viewing Sunday, March 13 through Tuesday, March 15
Title: A PATrOL- Enabled Investigational Genetic Therapy for DM1: Mouse Pharmacokinetics, Biodistribution, and CNS Penetration after Systemic Administration
Type: Oral Presentation
Date: Wednesday, March 16, 9:30am-9:45am CST
Expanding upon initial data presented in June 2021, the presentations will include new data of a PATrOL-enabled investigational genetic therapy for DM1. NeuBase will present molecular and functional pharmacological activity of its DM1 investigational genetic therapy in the HSALR mouse model following single and repeated subcutaneous, intravenous, and intramuscular administration. The HSALR model carry the long repeat (LR) length of the DMPK repeat expansion and recapitulates many aspects of the clinical presentation of DM1. Additional pharmacokinetic data will be presented of NeuBase’s DM1 investigational genetic therapy in wild-type mice demonstrating distribution and pharmacologic activity throughout the body, including the brain and muscle, following systemic administration.
Patients with DM1 suffer from cognitive deficits and muscle pathology caused by a trinucleotide expansion in the DMPK gene. NeuBase’s DM1 investigational genetic therapy targets DMPK pre-mRNA with a novel peptide-nucleic acid (PNA) pharmacophore and is designed to selectively engage with the toxic RNA hairpin structure and release the splicing proteins to restore RNA splicing and downstream protein production. The PNA pharmacophore is conjugated to NeuBase’s novel delivery technology that is designed for broad distribution, including into the deep brain, with the potential for a whole body, disease-modifying solution for DM1. For more information, visit View Source