Project Grant R44NS143501
- 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 federal Project Grant award from the National Institutes of Health's Research Infrastructure Programs (CFDA 93.351) provides $156,136 to Recombinetics Inc. to advance gene therapy for Mucopolysaccharidosis Type IVA (MPS IVA), a rare lysosomal storage disease. The key products and services to be delivered under this award include: 1) Generating a porcine model of MPS IVA to serve as a valuable tool for understanding the disease biology and developing genetic therapies; 2) Conducting a...
- This $226,500 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, will support a 2-year research project to characterize and treat peripheral nervous system dysfunction in Sanfilippo syndrome (mucopolysaccharidosis type IIIA). The award will enable researchers at the University of Georgia Research Foundation to develop novel disease...
- This federal Project Grant award from the National Eye Institute (NEI) at the Department of Health and Human Services provides $1,850,000 in funding to Biostrategies LC to advance the development of BIOS-GM1, a novel gene therapy and enzyme replacement therapy for the treatment of GM1 gangliosidosis. The grant supports Biostrategies' efforts to complete IND-enabling studies for BIOS-GM1, which combines an adeno-associated virus (AAV) gene therapy approach with a lectin-enhanced enzyme delivery...
- 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 SBIR Phase I Project Grant award from the National Science Foundation's (NSF) Technology, Innovation, and Partnerships (CFDA 47.084) program provides $305,000 to Junipero Therapeutics, Inc. to develop an innovative gene therapy platform using oligonucleotide conjugates to enable safer, more cost-effective, and scalable treatments for genetic disorders. The research focuses initially on neurodegenerative conditions like Huntington's disease and ALS, with the goal of establishing a...
- 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) federal grant program, provides $699,559 to Merlin Therapeutics Inc. to develop a novel adeno-associated viral vector 9 (AAV9) gene therapy for the treatment of neurofibromatosis type 2 (NF2). The therapy aims to express a healthy copy of the NF2 gene and rescue tumor characteristics in NF2...
- 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 federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), part of the Cardiovascular Diseases Research program (CFDA 93.837), provides $300,000 to Gigamune, Inc. to develop an in vivo gene therapy for sickle cell disease. The project aims to test a novel lentiviral vector technology for efficiently editing hematopoietic stem cells to knockout the BCL11A enhancer, a key genetic target for sickle cell treatment. Through in vitro assays, the Phase I effort will...
- 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 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, scAAV9-MCMV-hIDUAOP, towards clinical application and commercialization. This gene therapy targets the neurological impairments associated with the severe form of MPS I, known as Hurler syndrome, for which no effective treatment is currently available. The funding will support preclinical studies to evaluate the efficacy and safety of this scAAV9-mediated gene replacement therapy in treating the central nervous system manifestations of MPS I.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $722.6k | 9/16/25 |