Project Grant R43NS115425

Summary: Mechanisms of ALS/FTD Determined by Multimodal Single Cell Spatial Transcriptomics The University of California, San Diego received a $3.08M Project Grant from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853), awarded September 12, 2025, with a completion date of June 30, 2029. This research grant funds the development and application of a multimodal spatial...

Federal Project Grant Award Summary D4Z4 Medicine, Inc. received a $313,372 Project Grant from the National Center for Advancing Translational Sciences (NCATS, CFDA 93.350) awarded on August 7, 2025, with a completion date of July 31, 2026. The award funds development of a long-read sequencing-based molecular diagnostic assay for facioscapulohumeral muscular dystrophy (FSHD), a rare genetic disorder currently lacking an effective diagnostic tool. The company will deliver two primary products:...

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 $437,250 to the University of California, San Diego (UCSD) to conduct pooled in vivo screening of combination gene therapies for enhancing axonal regeneration after spinal cord injury (SCI). The key objectives are to 1) perform CRISPR screening to identify the most effective...

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, supports the development of a blood-based biomarker for characterizing disease progression and therapeutic response in amyotrophic lateral sclerosis (ALS). The awardee, Neurodex Inc., a small for-profit organization, is advancing the validation of an assay for measuring...

Myogene Bio LLC received a $499,300 Project Grant award from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846), effective September 1, 2025 through July 31, 2028. The award supports the advancement of manufacturing and analytical testing capabilities for a dual adeno-associated virus (AAV) vector gene editing therapy targeting Duchenne muscular dystrophy (DMD). Under this...

The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $450,313 Project Grant (CFDA 93.853 - Extramural Research Programs in the Neurosciences and Neurological Disorders) to The Johns Hopkins University to develop high-throughput screening (HTS) assays to identify activators of the nemo-like kinase (NLK) protein. The goal is to find small molecule compounds that can lower mutant huntingtin (mHTT) protein levels as a potential therapeutic approach for Huntington's...

Federal Grant Award Summary Quiver Bioscience Inc. received a $862,420 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, effective May 20, 2025, with completion targeted for April 30, 2028. The award supports the development of an integrated platform combining machine learning (ML)-based antisense oligonucleotide (ASO) design tools with in vitro...

Acrigen Biosciences, Inc. was awarded a $1,000,000 cooperative agreement from the National Science Foundation under the Engineering program (CFDA 47.041) to develop a curative gene therapy for spinal muscular atrophy. Over the two year period from March 2022 to February 2024, Acrigen will engineer an anti-CRISPR protein to increase the precision and safety of CRISPR-Cas gene editing. They will pair this with a novel CRISPR nuclease and validate the system for editing and disease correction in...

This SBIR Phase I project awarded by the National Science Foundation (NSF) under the Technology, Innovation, and Partnerships (CFDA 47.084) program seeks to develop a new class of gene therapies that can be delivered more safely, cost-effectively, and at scale for a wide range of serious genetic disorders. The project leverages short oligonucleotides conjugated with small molecules to induce proximity of endogenous epigenetic machinery to disease-relevant genes in a precise and reversible...

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...