Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
antisense oligonucleotide targeting mutant NEFL
- Development stage
- Preclinical
- Lead developer
- University of Miami
- Modality
- 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
01
Overview
Antisense oligonucleotide targeting mutant NEFL is a preclinical genetic therapeutic being developed by researchers at the University of Miami for the treatment of Charcot-Marie-Tooth disease type 2E (CMT2E). CMT2E is an axonal form of Charcot-Marie-Tooth disease caused by dominant missense mutations (such as p.N98S) in the NEFL gene, which encodes the neurofilament light chain (NF-L) protein. The antisense oligonucleotide (ASO) is designed with 2'-O-methyl or 2'-O-methoxyethyl and phosphorothioate modifications to selectively target and knockdown mutant NEFL transcripts, thereby reducing the formation of toxic NF-L aggregates and mitigating axonal degeneration.
- Other names
- NEFL-targeting antisense oligonucleotideantisense oligonucleotide targeting NEFL
Beyond the preview
Go deeper on antisense oligonucleotide targeting mutant NEFL.
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 antisense oligonucleotide targeting mutant NEFL.