Federal Project Grant Award Summary

The Food and Drug Administration (FDA) awarded Boston Children's Hospital a $3.58 million Project Grant on September 25, 2025, under CFDA 93.103 (Food and Drug Administration Research) to conduct a clinical trial evaluating the safety and effectiveness of atipeksen, an antisense oligonucleotide (ASO) therapeutic for ataxia-telangiectasia (A-T). The trial, designated IND#144349, targets individuals with A-T who carry the specific ATM c.7865C>T mutation, a recurrent genetic variant affecting approximately 15% of the A-T patient population. The research focuses on testing whether atipeksen can slow neurodegeneration, delay symptom progression, and improve quality of life in pediatric patients with this rare, currently untreatable neurodegenerative condition that typically results in early mortality.

The study employs atipeksen—a 22-nucleotide ASO designed to correct aberrant gene splicing in the ATM exon 53—administered via intrathecal injection to distribute broadly across the brain and spinal cord. Outcomes will be measured using neurologic disability rating scales, brain imaging, and exploratory biomarkers to assess therapeutic efficacy against comparator groups. The award extends through August 31, 2029, with performance conducted at Boston Children's Hospital in Massachusetts. This project directly supports the FDA's mission under the Food and Drug Administration Research program to advance regulatory science and therapeutic development in rare pediatric diseases.