Project Grant R21NS146929
- The National Institute of Neurological Disorders and Stroke (NINDS) awarded the University of Massachusetts Medical School $659,245 on March 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to develop a gene therapy for UBA5 deficiency, an ultra-rare autosomal recessive neurological disease. UBA5 deficiency presents in patients with infantile spasms, failure to thrive, hypotonia, developmental delay, microcephaly,...
- 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 awarded a cooperative agreement of $1,038,135 to the University of Massachusetts Medical School on September 2, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds research to develop and optimize a trivalent small interfering RNA (siRNA) therapeutic targeting Huntington's disease pathogenesis. The investigative approach employs multiple simultaneously...
- The National Institute of Neurological Disorders and Stroke, part of the Department of Health and Human Services National Institutes of Health, awarded University of California, Davis $442,750 on April 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853, Project Grant R21NS142879). UC Davis will conduct high-throughput screening of novel inverted terminal repeat (ITR) domains in the mouse brain to optimize adeno-associated viral...
- The National Institute of Neurological Disorders and Stroke awarded the University of Massachusetts Medical School $551,557 on August 15, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate nucleic acid sensing pathways in demyelinating disease. The funded research dissects the role of endosomal toll-like receptors (eTLRs) in neuroinflammation and demyelination, specifically examining how aberrant eTLR signaling in...
- The National Institutes of Health Office of the Director awarded Neurogt Inc. $722,587 on September 16, 2025, to develop a self-complementary AAV9 gene replacement therapy targeting central nervous system neuropathy in mucopolysaccharidosis (MPS) I, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). This Phase II SBIR project advances a novel self-complementary AAV vector, scAAV-MCMV-HIDUAOP, designed to deliver a miniaturized human IDUA...
- The National Institute of Neurological Disorders and Stroke awarded Massachusetts General Hospital a $2.541 million cooperative agreement on May 1, 2026, to develop targeted gene repair therapy for multisystem smooth muscle dysfunction syndrome caused by ACTA2 R179H mutation, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds IND-enabling studies for a novel CRISPR/Cas9-directed adenine base editor delivered via AAV-PR...
- The National Institute of Neurological Disorders and Stroke awarded Massachusetts General Hospital $161,880 on May 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The award funds investigation of TTYH3 protein and its relationship to CLN3 Batten disease, a rare lysosomal disorder affecting approximately 1 in 100,000 live births. The research will develop tools to study TTYH3 in the context of CLN3 disease, including...
- The National Institute of General Medical Sciences awarded the University of Massachusetts Medical School $1,675,000 on May 1, 2026, under the Biomedical Research and Research Training program (CFDA 93.859) to interrogate the roles of truncated and mutated inverted terminal repeat (ITR) sequences in adeno-associated virus (AAV) vectors and their effects on transgene stability through episome formation and host cell genome integration. The project, designated R01GM155648, addresses a critical gap...
- Federal Project Grant Award Summary The National Institute of Neurological Disorders and Stroke (NINDS) awarded $460,625 to the University of Massachusetts Medical School on June 7, 2025, under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program. This exploratory research project, scheduled for completion by May 31, 2027, investigates the roles of the deubiquitylation pathway in TDP-43 (TAR DNA-binding protein 43) pathology relevant to...
The National Institute of Neurological Disorders and Stroke awarded the University of Massachusetts Medical School $460,625 on September 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) for development of AAV-mediated gene silencing to treat hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC), a rare, progressive leukodystrophy caused by mutations in the TUBB4A gene. The research develops an AAV9 vector delivering artificial microRNAs (amir-TUBB4A) to suppress TUBB4A expression across multiple central nervous system cell types as a therapeutic approach for H-ABC, which currently has no FDA-approved disease-modifying treatments. Prior work with the AAV9-amir-TUBB4A vector showed promise in animal models through extended survival, increased body weight gain, and delayed seizure onset via oligodendrocyte delivery. The project will enhance therapeutic effects by incorporating a newly developed AAV capsid (BI-HTFR1) that binds to the human transferrin receptor (HTFR1) to improve biodistribution and efficacy before advancing to the next stage of development. Performance occurs at the University of Massachusetts Medical School in Worcester, Massachusetts. The period of performance runs through August 31, 2028. This is a Project Grant assistance type under the NINDS extramural research portfolio supporting basic and applied neuroscience research.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $460.6k | 8/31/26 |