Project Grant R44NS139748
- This $694,134 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) supports research to enhance the engraftment of autologous, genetically modified hematopoietic stem cells (HSCs) for the treatment of globoid cell leukodystrophy (GLD), also known as Krabbe disease. The goal is to improve the replacement of dysfunctional macrophages with competent donor...
- The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $1,499,212 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to Aquinnah Pharmaceuticals, Inc. The grant supports the development of a novel oral, brain penetrant therapeutic to slow and potentially reverse the progression of Amyotrophic Lateral Sclerosis (ALS). The key product being delivered is an innovative new clinical drug candidate designed to...
- This Project Grant award of $532,381 from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) supports research into the pathogenic mechanisms of the KIF5A gene mutation associated with amyotrophic lateral sclerosis (ALS). The key objectives of this 5-year research project are to: 1) determine the gain-of-function effects of the ALS-associated KIF5A exon 27 skipping variant...
- This federal Project Grant award of $592,233.00 from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) will support research on the regulation of sphingolipid biosynthesis and its relationship to amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease. The research will utilize cellular models and mouse models to investigate how mutations in the serine...
- This Project Grant award, funded 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 advanced base and prime editing strategies to correct common ALS-causing mutations in the SOD1 gene. The $124,246 award, granted to the University of Massachusetts Medical School, will support in vitro and in vivo studies to create next-generation base and prime editors for...
- This federal Project Grant award of $488,507, 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, is focused on investigating synaptic dysfunction in lissencephaly, a severe brain malformation resulting from mutations in the LIS1 gene. The award supports a series of preclinical translational studies using sophisticated cellular, molecular,...
- 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 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,...
- 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 $645,391 to The Trustees of Columbia University in the City of New York to conduct research on the effects of re-expressing embryonic motor neuron selector transcription factors in mouse models of amyotrophic lateral sclerosis (ALS) and in ALS motor neurons derived from induced...
- This Project Grant award of $374,349 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 and optimization of a new animal model for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The goal is to create a mouse model that accurately recapitulates the human disease spectrum by incorporating an acetylation-mimicking...
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 provide the necessary preclinical data to support an investigational new drug (IND) application, with the potential to significantly improve treatment for ALSP and extend patient survival. Key efforts include validating a gene-corrected protocol, demonstrating the safety and tolerability of the induced microglial cell (iMGL) product, and completing the development of a custom-designed cannula device for intraparenchymal cell delivery to the central nervous system.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $1.5m | 7/31/25 |