DM1 is a rare, autosomal dominant repeat expansion disorder characterized by progressive muscle wasting and weakness. It also affects the central nervous system (CNS) and heart. DM1 is caused by CTG nucleic acid repeats in the DMPK gene that produce a hairpin structure in the transcribed DMPK mRNA. The hairpin structure sequesters critical splice regulators and results in the mis-splicing of multiple gene transcripts. Furthermore, the binding of splice regulators traps the mutant DMPK mRNA in the nucleus, resulting in DMPK protein haploinsufficiency, or half the level of protein that is needed for normal function, which is thought to exacerbate the CNS and cardiac symptoms that are characteristic of DM1 (as knock-out mice for Dmpk show both severe cardiac conduction defects as well as issues with neuronal cytoskeletal remodeling manifesting in aberrant long-term potentiation). The prevalence of DM1 is >5/100,000 in the general population. There are currently no approved treatments for DM1.

NeuBase Therapeutics, Inc. will discuss these data during a webcasted conference call with slides today, December 16, 2020, at 8:00 a.m. EST. To access the webcast, please click here. An archived recording of this presentation will be available following the call through the IR Calendar page on the Investors section of the Company's website, www.neubasetherapeutics.com.

About NeuBase Therapeutics, Inc.
NeuBase is accelerating the genetic revolution using a new class of synthetic medicines. NeuBase's designer PATrOL™ therapies are centered around its proprietary drug scaffold to address genetic diseases at the source by combining the highly targeted approach of traditional genetic therapies with the broad organ distribution capabilities of small molecules. With an initial focus on silencing disease-causing mutations in debilitating neurological, neuromuscular and oncologic disorders, NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions. To learn more, visit www.neubasetherapeutics.com.

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