Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

Phase 1/2 Active not recruiting 1 enrolled n-Lorem Foundation Mayo Clinic · collab

NaSingle-groupOpen-labelTreatment

Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Autosomal Dominant Leukodystrophy (ADLD) due to LMNB1 mutation

Timeline

Start: 2025-03-17
Primary completion: 2027-03
Completion: 2027-03

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	nL-LMNB1-001	Antisense oligonucleotide	—	—

Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

Summary

Timeline

Drugs

Indications