Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation

Phase 1/2 Active not recruiting 1 enrolled n-Lorem Foundation Massachusetts General Hospital · collab

NaSingle-groupOpen-labelTreatment

Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Childhood-Onset Neurodegeneration with Brain Atrophy (CONDBA) due to a heterozygous missense gain-of-function mutation in UBTF

Timeline

Start: 2025-02-05
Primary completion: 2027-02
Completion: 2027-02

Drugs

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

Indications

No indication recorded.