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. We believe the ability of our drug candidate to reach skeletal and heart muscles as well as brain tissue may potentially deliver a differentiated therapeutic approach to not only treat the myotonia, muscle weakness, and cardiac effects but also the cognitive impairments seen in DM1. We have now demonstrated in preclinical models that our lead candidate NT-0231.F achieves clinically relevant molecular and functional rescue as well as whole-body distribution.”
The data will be presented today from 5:30 pm to 6:30 pm ET in a poster entitled, “Pharmacokinetics, Biodistribution, and CNS Penetration of a PATrOL™-Enabled Investigational Genetic Therapy for Myotonic Dystrophy Type 1 Following Systemic Administration in Mice.”
Further details of the presentation and the poster can be found here.
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 https://www.neubasetherapeutics.com/pipeline/.
About NeuBase Therapeutics
NeuBase is accelerating the genetic revolution by developing a new class of precision genetic medicines that Drug the Genome™. The Company’s therapies are built on a proprietary platform called PATrOL™ that encompasses a novel peptide-nucleic acid antisense oligonucleobase technology combined with a novel delivery shuttle that overcome many of the hurdles to selective mutation engagement, repeat dosing, and systemic delivery of genetic medicines. 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, who currently have limited to no treatment options.
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