Clinical Trial: Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation

U.S., Oct. 29 -- ClinicalTrials.gov registry received information related to the study (NCT07221760) titled 'Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation' on Oct. 24.

Brief Summary: This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with dentatorubral-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide expansion in ATN1

Study Start Date: Nov., 2025

Study Type: INTERVENTIONAL

Condition: Dentatorubral-Pallidoluysian Atrophy

Intervention: DRUG: nL-ATN1-001

Personalized Antisense Oligonucleotide

Recruitment Status: NOT_YET_RECRUITING

Sponsor: n-Lorem Foundation

Disclaimer: Cur...