Project Grant R41NS145933
- This $803,958 federal Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program supports the development of an innovative approach to deliver a more biologically stable form of the enzyme acid β-glucosidase (GCase) through the blood-brain barrier into the central nervous system. The goal is to enable treatment for neuronopathic Gaucher disease, a...
- This $699,818 Project Grant awarded on July 8, 2025 by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) aims to develop an effective adeno-associated virus (AAV) gene therapy product for treating Mucopolysaccharidosis Type IIIC (MPS IIIC), a rare and devastating lysosomal storage disease. The project seeks to leverage extracellular vesicle-mediated mRNA trafficking to...
- This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) will lay the foundation for developing a molecular therapy for ADNP syndrome. The $395,009 award to the University of California, Davis will fund research to: 1) test the mechanism of action of antisense oligonucleotides (ASOs) that can increase ADNP protein expression in human neurons, and 2)...
- This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, will support preclinical testing of a novel gene therapy for a rare pediatric neurodegenerative disease. The $419,482 award to the University of Texas Southwestern Medical Center will fund studies to determine the minimal effective dose and post-symptomatic treatment efficacy of the...
- This $147,486 Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) supports research to understand the disease mechanism and develop gene therapy for a common and understudied STXBP1 missense variant that causes severe developmental and epileptic encephalopathy. The applicant has established a mouse model that recapitulates key neurological symptoms of this...
- This Project Grant award from the National Institutes of Health (NIH) Office of Research Infrastructure Programs (CFDA 93.351) provides $722,587 to Neurogt Inc., a biotechnology company in North Carolina, to further develop an effective gene replacement therapy product for treating Mucopolysaccharidosis (MPS) I. The goal of this Phase II Small Business Innovation Research (SBIR) project is to advance a novel self-complementary adeno-associated virus (scAAV) gene therapy vector,...
- This Project Grant award of $1,489,811.00 from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, is focused on validating an ex vivo gene correction strategy and ensuring the safety, therapeutic efficacy, and delivery of an autologous cell replacement therapy for adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). The award aims to...
- This $147,486 Project Grant awarded by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program will support research to gain deeper insight into the pathophysiology of SCN3A-related neurodevelopmental disorder (SCN3A-NDD) and advance the development of effective therapeutic interventions. The research will utilize a novel mouse model of SCN3A-NDD to: 1) evaluate the...
- The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $391,903 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to the Allen Institute to develop a new antisense oligonucleotide (ASO) therapeutic for Dravet syndrome, a severe epileptic encephalopathy. The grant will fund a two-phase project to optimize the ASO, establish its pharmacokinetics and pharmacodynamics, and validate a large-scale batch of the lead...
- This federal Project Grant award, funded by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, aims to develop a non-viral gene-editing therapy using lipid nanoparticle (LNP)/mRNA complexes to correct UBE3A gene overexpression in neural stem progenitor cells during fetal brain development. The $654,339 award to the University of California, Davis will support research to...
The Project Grant award of $500,000 from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) is supporting the development of a novel blood-brain barrier (BBB) penetrable adeno-associated virus (AAV) gene therapy for Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD), a rare genetic neurological disorder. The funding will allow Galibra Neuroscience, Inc. to further optimize the dosing and delivery of their AAV-FLNP-hALDH5A1 therapy (named GAL005) using an engineered AAV-BI-hTfR1 capsid that can cross the BBB, building on their previous proof-of-concept studies demonstrating phenotype reversal in SSADHD mouse models. This project aims to advance a potential disease-modifying treatment for the intellectual disability, autism-like features, and epilepsy associated with SSADHD, which currently lacks effective therapeutic options.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $500.0k | 8/12/25 |