Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
PATrOL-enabled PNA
- Development stage
- Discontinued
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intramuscular, Intravenous, Subcutaneous
01
Overview
PATrOL-enabled PNA (including the lead candidate NT-0231.F under the NT0200 program) is an investigational peptide nucleic acid (PNA) genetic therapy developed by NeuBase Therapeutics for the treatment of myotonic dystrophy type 1 (DM1). Utilizing NeuBase's proprietary Peptide-Nucleic Acid Antisense Oligonucleobase Platform (PATrOL™), the therapy is designed to target mutant DMPK pre-mRNA containing toxic CUG trinucleotide repeat expansions. By selectively binding to these toxic CUG hairpins, the PNA pharmacophore sterically blocks the expansion, preventing the sequestration of Muscleblind-like (MBNL) splicing proteins, thereby restoring normal RNA splicing and downstream protein production. In preclinical models, the therapy demonstrated broad tissue distribution, including penetration of the blood-brain barrier, and successfully reversed myotonia. However, development was halted in August 2023 when NeuBase Therapeutics announced the cessation of its research programs and subsequent plans for company dissolution.
- Other names
- DM1 investigational genetic therapyDM-1 investigational genetic therapyDM 1 investigational genetic therapyPATrOL-enabled peptide nucleic acid
02
Targets
DMPK (Dystrophia myotonica protein kinase)
Beyond the preview
Go deeper on PATrOL-enabled PNA.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on PATrOL-enabled PNA.