Project Grant R43HD097801

This 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 supports research on developing an enhanced gene therapy approach to treat Globoid Cell Leukodystrophy (GLD), also known as Krabbe disease. The $694,134 award to the University of Pennsylvania aims to investigate the therapeutic potential of using genetically-modified autologous hematopoietic...

This Project Grant award of $803,958 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 beta-glucosidase (GCase) through the blood-brain barrier into the central nervous system. The goal is to enable treatment for the neuronopathic forms of Gaucher...

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) program, supports the development of a blood-brain barrier penetrable adeno-associated virus (AAV) gene therapy for Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD), a rare neurological disorder. The funded project aims to optimize the dosage of the AAV-FLNP-HALDH5A1 gene...

The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) awarded a $1,204,698 Project Grant to The Trustees of the University of Pennsylvania to conduct research on Lysosomal Cholesterol Acyltransferase (LCAT) deficiency, a rare genetic disorder. The key objectives are to: Identify biomarkers to guide enrollment criteria and distinguish the two main LCAT deficiency syndromes (Familial LCAT Deficiency and Fish-Eye Disease), as well as determine...

The National Institute of Neurological Disorders and Stroke (NINDS), through the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853), awarded a $1,489,811 Project Grant to Savanna Biotherapeutics, Inc. to develop an engineered microglia cell therapy for the treatment of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), a rare and fatal neurodegenerative disease. The project aims to validate an ex vivo gene correction strategy,...

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 provide $1,000,000 to Tega Therapeutics Inc. to develop new clinical biomarkers for GM1 gangliosidosis. The goal is to confirm whether levels of metabolites in urine and/or cerebrospinal fluid (CSF) reflect changes in tissue levels following enzyme replacement therapy in a mouse model....

This Project Grant award from the National Center for Advancing Translational Sciences (NCATS) under CFDA 93.350 will provide $463,150 to The Regents of the University of California, San Francisco (UCSF) to establish proof-of-concept for enzyme replacement therapy (ERT) as a lifesaving treatment for sphingosine phosphate lyase insufficiency syndrome (SPLIS), an ultra-rare and often lethal metabolic disorder. The project aims to demonstrate the efficacy of ERT in preserving kidney function and...

The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), through the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847), has awarded a $299,648 Project Grant to Cornela Therapeutics Inc. to conduct enabling studies for CMT-101, a novel gene therapy for Glycogen Storage Disease 1B (GSD-1B). The two-year award, beginning on June 1, 2025, will support dose-finding and biodistribution studies, as well as an pre-Investigational New Drug (IND)...

The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $391,903 Project Grant to the Allen Institute under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to develop an oligonucleotide therapeutic for Dravet syndrome. The project aims to characterize the pharmacokinetics and pharmacodynamics of a new antisense oligonucleotide (ASO) that increases expression of the voltage-gated sodium channel Nav1.1, which is deficient in...

This Project Grant award of $613,558 from the National Institute of Diabetes and Digestive and Kidney Diseases (CFDA 93.847 Diabetes, Digestive, and Kidney Diseases Extramural Research) supports Genetobe Inc., a Michigan-based biotechnology company, in developing novel, selective sodium-dependent glucose cotransporter 1 (SGLT1) inhibitor compounds as an adjunctive therapy for cystic fibrosis (CF) associated metabolic disorders, including CF-associated liver disease and CF-related diabetes. The...