Project Grant R21HD122115
- The National Institute of Child Health and Human Development awarded Duke University $593,848 on September 1, 2026, under the Child Health and Human Development Extramural Research program (CFDA 93.865) to develop therapeutic approaches for glutaric aciduria type I (GA-1), an autosomal recessive metabolic disorder caused by deficiency of the enzyme glutaryl-CoA dehydrogenase (GCDH). The funded research builds on prior demonstration that murine GA-1 models can be rescued through liver-directed...
- The National Institute of Neurological Disorders and Stroke awarded a cooperative agreement of $649,600 to The Regents of the University of California, San Francisco on April 1, 2026, to develop prenatal AAV9 gene replacement therapy for patients with severe infantile GM1 gangliosidosis. The award funds research into prenatal somatic cell gene therapy (PSCGT) targeting GM1 gangliosidosis, a severe, fatal neurodegenerative lysosomal storage disorder resulting from biallelic mutations in the...
- The National Institute on Aging awarded the University of California Irvine $431,750 on May 15, 2026, under the Aging Research program (CFDA 93.866) to develop antisense oligonucleotide therapies targeting valosin-containing protein (VCP) disease pathology. The project, titled "Correction of VCP Disease Pathology with Antisense Oligonucleotides," addresses the neuromuscular component of VCP multisystem proteinopathy 1, an autosomal dominant disorder characterized by inclusion body...
- The National Institute of Child Health and Human Development awarded University of Massachusetts Medical School $460,625 on June 1, 2026, under the Child Health and Human Development Extramural Research program (CFDA 93.865) to develop humanized muscle xenograft models for optimizing and assessing genome editing therapeutics targeting limb-girdle muscular dystrophy R9 (LGMDR9). The project delivers two specific research aims. Aim 1 optimizes prime editing correction of the FKRP L276I mutation in...
- The National Institute of Child Health and Human Development awarded the University of Florida $419,375 on June 2, 2026, under the Child Health and Human Development Extramural Research program (CFDA 93.865) to develop a combined pharmacological and gene therapy approach for pyruvate dehydrogenase complex deficiency in children. The research combines dichloroacetate (DCA), an oral medication that stimulates residual pyruvate dehydrogenase complex activity, with next-generation adeno-associated...
- The National Institute of Child Health and Human Development awarded Icahn School of Medicine at Mount Sinai $726,448 on August 13, 2026, under the Child Health and Human Development Extramural Research program (CFDA 93.865) to discover novel inhibitors of the AASS enzyme as a treatment for glutaric aciduria type 1 through substrate reduction. Glutaric aciduria type 1 (GA1) is an autosomal recessive inborn error of metabolism caused by deficiency of glutaryl-CoA dehydrogenase, leading to...
- 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...
- Federal Project Grant Award Summary The National Institute of Neurological Disorders and Stroke (NINDS) awarded $395,009 to the University of California, Davis on August 1, 2025, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The two-year project, extending through July 31, 2027, supports foundational research to develop antisense oligonucleotide (ASO) therapy for ADNP syndrome (Helsmoortel-van der Aa syndrome), a severe...
- 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,...
- Federal Grant Award Summary The National Institute of Child Health and Human Development (NICHD) awarded $422,916 in project grant funding under the Child Health and Human Development Extramural Research program (CFDA 93.865) to The Research Foundation For The State University Of New York (RF-SUNY) at the University at Albany, effective August 1, 2025, through July 31, 2027. This project grant supports investigator-initiated biomedical research designed to advance understanding of human...
The National Institute of Child Health and Human Development awarded University of California Irvine $431,750 on September 1, 2026, under the Child Health and Human Development Extramural Research program (CFDA 93.865) to develop an oligonucleotide-based therapeutic targeting muscle glycogen synthase 1 (GYS1) as a substrate inhibitor for Pompe disease. The award funds preclinical research to suppress GYS1 expression using antisense oligonucleotides (ASOs), designed to reduce glycogen accumulation in lysosomes, correct autophagic dysfunction, and improve disease pathology in patient-derived myotubes. Current enzyme replacement therapy for Pompe disease, caused by acid alpha-glucosidase (GAA) deficiency, shows limited efficacy particularly in skeletal muscle, where patients continue to accumulate glycogen despite treatment. The proposed substrate reduction strategy addresses an unmet clinical need by targeting persistent glycogen synthesis. Preliminary work by Ionis Pharmaceuticals identified three potent ASO candidates that demonstrated safety and robust GYS1 suppression in mouse models when administered subcutaneously. The performance period runs from September 1, 2026, through August 31, 2028, with work performed in California. This is a Project Grant, the standard assistance type for NIH research funding.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $431.8k | 8/21/26 |