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 $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) 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 Neurological Disorders and Stroke (NINDS) awarded a $226,500 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to the University of Georgia Research Foundation, Inc. to characterize and treat peripheral nervous system dysfunction in Sanfilippo syndrome (mucopolysaccharidosis type IIIA). The project aims to develop novel cellular disease models using patient-derived induced pluripotent stem cells to...
This federal Project Grant award for $142,452.00 was provided 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. The funding supports research to develop novel mRNA-based gene therapies for the treatment of diffuse midline glioma (DMG), a lethal pediatric brain tumor. The principal investigator will optimize the delivery of mRNA using lipid nanoparticles to target the...
The National Institute of Child Health and Human Development (NICHD) awarded a $295,923 Project Grant to Biostrategies LC under the Child Health and Human Development Extramural Research program (CFDA 93.865). The purpose of the award is to develop a "delivery-enhanced" gene therapy drug comprising an RTB:GALNS fusion protein for treating the rare metabolic disorder Mucopolysaccharidosis Type IVA (Morquio A Syndrome). The key objectives are to: 1) optimize the RTB:GALNS construct for...
This federal Project Grant award of $694,134 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 research to develop an improved treatment for globoid cell leukodystrophy (GLD), also known as Krabbe disease. The research aims to enhance the engraftment of autologous, genetically modified hematopoietic stem cells (HSCs) that produce the functional GALC...
The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $423,500 Project Grant (CFDA 93.853 - Extramural Research Programs in the Neurosciences and Neurological Disorders) to the Augusta University Research Institute, Inc. The objective is to develop and evaluate engineered exosomes carrying neuron-targeting peptides and a therapeutic protein (neuroglobin) for treating ischemic stroke. The research will: 1) characterize the engineered exosomes and assess their...
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...
The National Institute of Neurological Disorders and Stroke (NINDS) awarded a 24-month, $430,375 Project Grant (CFDA 93.853) to Emory University to develop human organoid models for pediatric high-grade gliomas (PHGGs). The research aims to 1) create PHGG organoid models using human patient-derived cancer stem cells and 2) model how interactions between PHGG cells and the human neural microenvironment affect responses to tyrosine kinase inhibitors and other anti-tumor drugs. The goal is to...
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. Specifically, the research aims to optimize the formulation, validate stability, and characterize pharmacokinetic properties of the SAPC-DOPS-FGCASE nanodrug in the R61 phase, and then evaluate its in vivo efficacy in a mouse model of neuronopathic Gaucher disease in the R33 phase. This work has the potential to radically improve enzyme replacement therapy delivery to the central nervous system, providing a promising treatment option for patients with this rare and debilitating neurological disorder.