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nL-FLVC-001
- Development stage
- Phase 1
- Lead developer
- University of Colorado Denver
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Injection, Intravitreal
01
Overview
nL-FLVC-001 is an antisense oligonucleotide (ASO) designed as a FLVCR1 inhibitor. It works by preventing the inclusion of a pseudo-exon in the FLVCR1 gene, specifically targeting an ectopic 3' splice site. The drug was developed for the treatment of Posterior Column Ataxia with Retinitis Pigmentosa (PCARP), a rare genetic disorder characterized by progressive sensory ataxia and retinitis pigmentosa. nL-FLVC-001 is administered via injection, with intravitreal (IVT) dosing used in clinical trials.
02
Targets
FLVCR1 (FLVCR choline and heme transporter 1)
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