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rna editing oligonucleotide (TARDBP)
- Development stage
- Preclinical
- Lead developer
- Korro Bio
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
The RNA editing oligonucleotide targeting TARDBP is a preclinical therapeutic candidate being developed by Korro Bio for the treatment of Amyotrophic Lateral Sclerosis (ALS). Utilizing Korro's proprietary OPERA (Oligonucleotide-based RNA Editing) platform, the drug consists of a synthetic oligonucleotide designed to bind specifically to TARDBP mRNA transcripts. Once bound, it recruits endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to catalyze a site-specific adenosine-to-inosine (A-to-I) conversion. This precise RNA editing is intended to produce variants of the TDP-43 protein that are resistant to the pathological cytoplasmic aggregation and mislocalization characteristic of ALS, while simultaneously restoring its essential nuclear splicing functions, such as the regulation of STMN2 and POLDIP3.
- Other names
- TARDBP RNA editing oligonucleotideOPERA TARDBP program
02
Targets
TARDBP (TAR DNA-binding protein 43)
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