This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under CFDA 93.853 Extramural Research Programs in the Neurosciences and Neurological Disorders, is focused on developing an adeno-associated virus (AAV) gene therapy product for treating Mucopolysaccharidosis (MPS) IIIB, a rare lysosomal storage disease. The $1,369,242 grant awarded on Sep 1, 2024 with an ultimate completion date of Aug 31, 2025 aims to advance the gene therapy product towards...
The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $1,541,019 Cooperative Agreement under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) federal grant program to The Trustees of the University of Pennsylvania. The award supports a comprehensive translational and clinical research program to develop a safe and effective gene therapy treatment for the ultra-rare subset of Duchenne muscular dystrophy (DMD) patients...
This 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) provides $2,013,258 to the University of Massachusetts Medical School (UMass Medical) to develop an AAV gene therapy for the rare lysosomal storage disorder sialidosis. The project aims to: 1) determine the minimum effective dose and maximum tolerated dose of the AAV9-NEU1-BICI-CTSA construct...
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...
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, is supporting the development of a first-in-class disease-modifying gene therapy for idiopathic Parkinson's disease (IDPD). The award provides $494,040 to Mark Therapeutics, Inc., a woman-owned small business, to conduct feasibility studies and rigorous preclinical...
This federal Project Grant award of $694,134 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 award aims to develop an approach to efficiently engineer autologous hematopoietic stem cells (HSCs) with a CSF1R inhibitor resistant variant and conditional galactosylceramidase (GALC) overexpression. This therapeutic approach seeks to enhance the...
This federal Project Grant award of $345,114, 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, supports research focused on developing novel therapeutic strategies for Huntington's disease (HD) by targeting the MSH3 protein. The key products and services to be delivered under this award include: 1) Demonstrating the scalability, precision, and...
This $195,570 federal Project Grant award was made 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 award supports research by The Regents of the University of California, San Francisco (UCSF) to investigate the role of the molecular co-chaperone DNAJC7 in preventing the aggregation of misfolded proteins associated with neurodegenerative diseases such as...
This 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, aims to develop a new therapy for neurodevelopmental mitochondrial disorders. The $105,625 award to the Icahn School of Medicine at Mount Sinai, from August 1, 2024 to July 31, 2026, will support two main objectives: 1) analyzing how a specific succinyl-CoA precursor is...
This $428,724 federal Project Grant was 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. The goal of this 2-year project is to refine a gene therapy strategy for the rare genetic disorder Xeroderma Pigmentosum-Cockayne Syndrome (XP-CS), with the aim of improving neurological outcomes and life expectancy for individuals with this condition. The research...