On May 2, 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 two abstracts have been accepted for presentation 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 (Press release, NeuBase Therapeutics, MAY 2, 2022, View Source [SID1234613291]).
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NeuBase will present new preclinical data on the biodistribution in key tissues of the company’s lead candidate, NT-0231.F, a precision genetic medicine in development to treat myotonic dystrophy, type 1 ("DM1"). NeuBase recently presented data at the 2022 MDA Clinical & Scientific Conference demonstrating that systemic administration of NT-0231.F in the HSALR model achieves clinically relevant molecular and functional rescue, including rescue of the spliceopathy, resolution of nuclear aggregates, and reversal of myotonia (delayed muscle relaxation after contraction). Pharmacokinetic studies of NT-0231.F in wild-type BALB/C mice showed a single IV or SC dose achieved high volume of distribution.
The presentations are listed below, and the full abstracts are available on the ASGCT (Free ASGCT Whitepaper) meeting website. All times are listed in Eastern Time (ET).
Title: Pharmacokinetics, Biodistribution, and CNS Penetration of a PATrOL-Enabled Investigational Genetic Therapy for Myotonic Dystrophy-Type 1 Following Systemic Administration Systemic Administration in Mice
Presenter: Noel Monks, Ph.D.
Session Date/Time: Tuesday, May 17, 5:30 PM – 6:30 PM
Poster Board Number: Tu-90
Session Title: Oligonucleotide Therapeutics II
Room: Hall D
Abstract Number: 585
Title: Pharmacology, Biodistribution and Tolerability of a PATrOL-Enabled Investigational Genetic Therapy for Myotonic Dystrophy, Type 1
Presenter: Sandra Rojas-Caro, M.D.
Session Date/Time: Wednesday, May 18, 5:30 PM – 6:30 PM
Poster Board Number: W-175
Session Title: Musculo-skeletal Diseases
Room: Hall D
Abstract Number: 1049
About NeuBase’s DM1 Program
Patients with DM1 suffer from cognitive deficits and muscle pathology caused by a trinucleotide expansion in the DMPK gene which, when transcribed, results in an RNA hairpin structure that sequesters RNA splice proteins. NeuBase’s DM1 investigational genetic therapy, NT-0231.F, targets mutant DMPK pre-mRNA with a novel peptide-nucleic acid ("PNA") pharmacophore and is designed to selectively engage with the toxic RNA hairpin structure, release the splicing proteins, and 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 www.neubasetherapeutics.com/pipeline/.