This federal Project Grant award, valued at $680,591.00 and funded by the Defense Health Agency under the Military Medical Research and Development program (CFDA 12.420), supports a novel small molecule screening approach to directly and selectively target repeat expansion RNA in C9ORF72 amyotrophic lateral sclerosis (ALS). The research is being conducted by Wayfinder Biosciences Inc., a for-profit organization located in Seattle, WA. The award period runs from June 15, 2025 to June 14, 2026....
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 focused on developing peptide nucleic acid (PNA) oligomers to disrupt biomolecular condensates associated with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The award of $74,305 to Carnegie Mellon University aims to (1) fluorescently label RNAs...
The National Institute of Neurological Disorders and Stroke (NINDS), through the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853), has awarded a $869,959 Cooperative Agreement to The Regents of the University of California, San Francisco to develop a safe and effective genome editing therapy to treat frontotemporal dementia caused by the C9ORF72 gene mutation. The project aims to optimize a CRISPR/Cas9 genome editing strategy to excise the C9ORF72 repeat...
The Defense Health Agency awarded a $458,516 Project Grant to Mayo Clinic Jacksonville to support research titled "NOVEL THERAPEUTICS TARGETING STRESS GRANULES FOR C9ORF72-MEDIATED ALS." The grant period runs from February 1, 2021 through June 30, 2022. The grant supports research into novel therapeutics targeting stress granules for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72 gene. This work falls under the Military Medical Research 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 $697,907 to Thomas Jefferson University to investigate the role of the C-JUN/transposable element (TE)/cGAS/STING pathway in C9ORF72-associated amyotrophic lateral sclerosis and frontotemporal dementia (C9-ALS/FTD). The key objectives are to: Identify cell-type specific patterns of...
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, provides $374,349 to the University of North Carolina at Chapel Hill to develop and optimize a new animal model for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The research aims to create a CRISPR-integrated mutant mouse line with an...
The National Institute of Neurological Disorders and Stroke (NINDS) awarded Aquinnah Pharmaceuticals, Inc. a $1,499,212 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program. 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 proposed therapeutic targets the key pathological hallmark of ALS, the...
This $236,668 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 will support research to elucidate the effects of the C9ORF72 gene mutation associated with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The grant recipient, Cleveland Clinic Lerner College of Medicine, will investigate how the interactions between...
The National Institute on Aging (NIA), under the Aging Research Federal Grant Program (CFDA 93.866), awarded a $228,019 Project Grant to Acrobat Genomics, Inc. The grant supports the development of a high-throughput protein engineering platform (ACROBATX) to enhance the company's proprietary ARGN gene editing system. The goal is to create a new gene therapy for amyotrophic lateral sclerosis (ALS), a fatal neurological disease, by optimizing the ARGN system to deplete expression of the mutant...
The National Institute on Aging (NIA), under the Federal Grant Program for Aging Research (CFDA 93.866), awarded a $404,058 Project Grant to the Regents of the University of Minnesota to develop a novel approach to restore functional TDP43 for the treatment of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The grant aims to identify small molecule compounds that can stabilize functional, multimerized TDP43 in order to rescue TDP43-associated pathologies. This will be...