This $1,369,242 project grant awarded by the National Institute of Neurological Disorders and Stroke (NINDS, CFDA 93.853) aims to develop an adeno-associated virus (AAV) gene therapy product for treating Mucopolysaccharidosis (MPS) IIIB, a rare lysosomal storage disease. The project focuses on optimizing and scaling up the manufacturing of the AAV9-based gene therapy product containing a codon-optimized human NAGLU cDNA. Phase I activities will involve engineering trials to develop and...
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) provides $349,909 to Neurogt Inc. to develop an adeno-associated virus (AAV) gene therapy product for treating Mucopolysaccharidosis Type IIIC (MPS IIIC), a rare lysosomal storage disease that currently lacks an effective treatment. The key objectives are to further advance a novel AAV9-based gene...
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...
The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $803,958 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to the Children's Hospital Medical Center in Cincinnati, Ohio. The grant funding supports the development of an innovative nanocarrier drug delivery system to enable stable and sustained delivery of a novel enzyme therapy to the brain for the treatment of neuronopathic Gaucher disease....
This federal Project Grant award of $189,056 from the National Institute of Child Health and Human Development (NICHD), under the Child Health and Human Development Extramural Research program (CFDA 93.865), supports research by Neurosant Therapeutics LLC to develop therapeutics that can correct the underlying genetic cause of Smith-Magenis Syndrome (SMS). The key goals of the project are to demonstrate that the expression of the RAI1 gene, which is reduced in SMS due to a genetic deletion,...
This Project Grant award from the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), under the Child Health and Human Development Extramural Research program (CFDA 93.865), aims to improve the reach and efficacy of adeno-associated virus (AAV)-based gene therapy by utilizing endogenously produced extracellular vesicles (EVs) to transport engineered transgene mRNA or protein products among cells and across tissues. The $860,750 award, effective June 1, 2025...
This Project Grant award of $230,250 from the National Institute of Child Health and Human Development (NICHD) under the Child Health and Human Development Extramural Research program (CFDA 93.865) supports research on reversing disrupted developmental sterol biosynthesis caused by prescription medications. The research aims to find a treatment for Smith-Lemli-Opitz Syndrome (SLOS), a rare genetic disorder that disrupts cholesterol biosynthesis, by reducing toxic 7-DHC levels and counteracting...
This federal Project Grant award of $249,000 from the National Institute of Child Health and Human Development (NICHD) under the Child Health and Human Development Extramural Research program (CFDA 93.865) aims to identify new therapeutics and understand molecular mechanisms for Congenital Disorders of Glycosylation (CDGs). The key objectives are to: 1) Determine the mechanisms of modifier genes that can rescue a DPAGT1-CDG cell model, 2) Conduct a drug repurposing screen in a DPAGT1-CDG...
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, provides $461,701 to Tega Therapeutics Inc. to develop and validate an intracerebroventricular (ICV) enzyme replacement therapy for the treatment of GM1 gangliosidosis, a rare lysosomal storage disease affecting the central nervous system. The key objectives are to engineer cell lines to...
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $295,294 Phase I SBIR project grant (CFDA 93.846) to Gigamune, Inc. to develop a novel lentiviral gene delivery platform for in vivo delivery of the full-length dystrophin gene to muscle cells. The goal is to create an efficient and targeted approach for gene therapy to treat Duchenne muscular dystrophy, a severe genetic disorder causing muscle wasting. The project aims to leverage Gigamune's...