Michigan Receives $3.9 Million NIH Award to Advance RNA Therapy for CLN3 Batten Disease
Published in Brain/Neurology, Drugs development & discovery.
Published October 6, 2026. Source date: October 5, 2026.
The University of Michigan announced a $3.9 million award from the NIH Ultra-rare Gene-based Therapy program to develop an antisense oligonucleotide medicine for CLN3 Batten disease. The project targets the most common genetic variant of this rare disorder, which causes progressive neurological decline.
Led by Michelle Hastings, the effort aims to advance the candidate toward clinical development. The planned initial approach is to treat a small group of children while assessing safety, biomarkers and early effectiveness. The new project builds on a collaboration that previously developed a personalized therapy for a different genetic cause of the disease.
The university’s Nucleic Acid Therapeutics Core supports molecular design, oligonucleotide synthesis and preclinical testing. This announcement reports research funding and development plans, not approval of a new treatment or new efficacy results for the commonly affected patient group.
Source: University of Michigan Health official announcement.