Project Grant R21TR006111
- This $288,000 Project Grant was awarded on August 4, 2025 by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) under the Child Health and Human Development Extramural Research federal grant program (CFDA 93.865). The grant supports research conducted by the Van Andel Research Institute in Grand Rapids, Michigan to investigate fatty acid metabolism and identify potential therapeutic targets for treating mitochondrial fatty acid synthesis (MTFAS)...
- 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...
- The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $543,351 project grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to the Translational Genomics Research Institute (TGen) in Phoenix, Arizona. The grant funds the development and characterization of a novel transgenic mouse model to investigate the mechanisms by which mutations in the MTFMT gene disrupt mitochondrial translation and cause Leigh syndrome, a...
- 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, aims to develop a high-throughput small molecule screening platform to reduce the endogenous levels of the MSH3 protein for a potential disease-modifying therapy for Huntington's disease (HD). The research initiative focuses on leveraging a multi-faceted approach,...
- This $490,501 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 Janusq LLC, a startup biotech company spun out of Case Western Reserve University, in developing a peptide-based therapy called HV-3 for the treatment of Huntington's disease (HD). The key objectives of this 16-month project are to: (1) perform preliminary safety and toxicity...
- 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,...
- The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $461,160 Project Grant to The Trustees of Columbia University in the City of New York under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program. The project, titled "From Metabolic Stress to Energy Enhancement: Unraveling Complex V Dimerization in Mitochondrial Disorders", aims to explore the role of ATP synthase dimerization in energy regulation and...
- 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...
- This federal Project Grant award of $489,500 from the National Center for Advancing Translational Sciences (NCATS) CFDA 93.350 program supports research to define patient-centric outcome measures for Multiple Sulfatase Deficiency (MSD), an ultra-rare neurodegenerative disorder. The project, led by The Children's Hospital of Philadelphia (CHOP), aims to (1) identify patient-centric tools for measuring the burden of MSD disease and defining clinical benefit, and (2) validate the use of...
- This $1,016,229 Project Grant awarded by the National Institute of General Medical Sciences (NIGMS) under CFDA 93.859 Biomedical Research and Research Training program supports Perlara PBC's "REMIT (RESCUE MITOCHONDRIAL THERAPEUTICS): UNLOCKING BROAD-SPECTRUM MITOCHONDRIAL RESCUERS" research project. The project aims to screen 8,400 repurposable drugs and nutraceuticals using the Yarrowia lipolytica yeast model to identify therapeutic candidates capable of restoring mitochondrial...
This $398,750.00 Project Grant awarded by the National Center for Advancing Translational Sciences (NCATS, CFDA 93.350) supports a research study on 4-hydroxymandelate (4-HMA) as a potential treatment for two forms of mitochondrial encephalopathy caused by biallelic mutations in the HPDL gene - neurodevelopmental disorder with progressive spasticity and brain white matter abnormalities (NEDSWMA) and autosomal recessive spastic paraplegia 83 (SPG83). The grant will fund a natural history study of 10 patients with the NEDSWMA phenotype, including testing 4-HMA and urine organic acids as disease biomarkers and collecting other outcome measures to support a future clinical trial of 4-HMA supplementation. The award was made to the University of California, San Diego, which has an established patient registry and expertise in rare disease research. This work aims to provide preclinical proof-of-concept data to advance a potential treatment for these mitochondrial disorders.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $398.8k | 8/18/25 |