Project Grant K99NS151274
- The National Institute of Neurological Disorders and Stroke awarded The Rockefeller University $593,250 on September 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to model ataxia-telangiectasia (A-T) using human stem cell-derived Purkinje cells in a novel laminar microfluidic system. The funded research develops and employs a laminar microfluidic model to differentiate human induced pluripotent stem cells into Purkinje...
- The National Institute of Neurological Disorders and Stroke awarded the University of Massachusetts Medical School $137,828 on November 1, 2025, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to conduct single-cell analysis of somatic mutation rates, mechanisms, and impacts in human ataxia telangiectasia cerebellum. The research characterizes how loss-of-function mutations in the ATM locus impact Purkinje cell genome stability and...
- The Department of Health and Human Services Food and Drug Administration awarded Boston Children's Hospital $3,577,616 on September 25, 2025, under the Food and Drug Administration Research program (CFDA 93.103) to conduct a clinical trial testing the safety and effectiveness of atipeksen, a 22-nucleotide antisense oligonucleotide designed to treat ataxia-telangiectasia (A-T) in individuals bearing the ATM C.7865C>T mutation. Atipeksen is administered via intrathecal injection and functions...
- The National Institute of Neurological Disorders and Stroke awarded The Broad Institute, Inc. $404,282 on May 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to engineer transferrin receptor 1 (TFR1) binding adeno-associated virus (AAV) capsids that evade pre-existing neutralizing antibodies while maintaining central nervous system delivery capability. The funded research addresses a critical barrier to AAV gene therapy for...
- The National Institute of Neurological Disorders and Stroke (NINDS), part of the Department of Health and Human Services National Institutes of Health, awarded $153,560 to the Broad Institute, Inc. (doing business as Broad Institute of MIT and Harvard) on December 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds research examining how excitatory neurons regulate interneuron fate, position, and survival during...
- The National Institute of Neurological Disorders and Stroke awarded The Trustees of Columbia University in the City of New York $126,900 on March 7, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds research to exploit RNA biogenesis, specifically alternative splicing programs, to accelerate maturation of human neurons in vitro and to develop models of age-related tauopathies. The research addresses a fundamental...
- The National Institute of Neurological Disorders and Stroke awarded $551,888 to Massachusetts General Hospital on August 19, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate the causes and consequences of tRNA ligation defects in neurodegeneration. The research examines how genomic GGC repeat expansions drive neurodegenerative diseases through polyglycine protein aggregation. The study biochemically characterized...
- The National Institute of Neurological Disorders and Stroke awarded President and Fellows of Harvard College $595,961 on September 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate mitochondrial DNA mutations and gene editing approaches in neural dysfunction. The research addresses mechanisms of central nervous system dysfunction triggered by mitochondrial DNA (mtDNA) mutations, particularly mutations in...
- The National Institute of Neurological Disorders and Stroke, part of the Department of Health and Human Services National Institutes of Health, awarded $555,000 to Augusta University Research Institute, Inc. on May 7, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds development of a novel optogenetic assay to study intracellular transport defects in neurodevelopmental diseases. The research addresses how motor...
- The National Institute of Neurological Disorders and Stroke awarded the University of California, Los Angeles $196,875 on June 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to evaluate and discover blood-based biomarkers for genetic ataxia. The project combines biomarker discovery using proteomic and transcriptomic approaches with testing of candidate biomarkers in blood samples from patients with common dominant and...
The National Institute of Neurological Disorders and Stroke, part of the Department of Health and Human Services National Institutes of Health, awarded The Broad Institute, Inc. $123,890 on August 6, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds research into allele-agnostic approaches for treating ataxia-telangiectasia, a childhood-onset neurodegenerative disorder caused by loss-of-function mutations in the ATM gene. The research will develop prime editing-mediated readthrough of premature termination codons (PERT), a genome editing strategy that installs optimized suppressor transfer RNAs directly into the genome to restore translation across multiple nonsense alleles simultaneously. During the K99 phase, the investigator will use directed evolution to identify synergistic combinations of mutations that maximize engineered suppressor transfer RNA efficiency and stability, then validate their ability to restore ATM expression and DNA damage response in patient-derived ataxia-telangiectasia cell lines. Twin prime editing will be used to install full-length engineered suppressor transfer RNAs at safe harbor loci for stable expression. The R00 phase will reprogram transfer RNA introns into circularized small RNAs, including splice-skipping antisense oligonucleotides, to enable permanent correction of ATM splice-site mutations. The period of performance extends through July 31, 2028. Work is performed in Cambridge, Massachusetts.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $123.9k | 8/5/26 |