Project Grant R43NS119090

This federal Project Grant award of $350,000.00 from the National Center for Advancing Translational Sciences (NCATS) under the CFDA 93.350 program is supporting the development of a fibroblast-derived engineered extracellular vesicle (FIEEV) platform for the targeted delivery of RNA therapeutics to treat Charcot-Marie-Tooth disease type 1A (CMT1A). The project aims to refine the FIEEV technology as a novel non-viral delivery method to overcome the limitations of current RNA therapeutic...

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 $339,999 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to Couragene, Inc., a for-profit organization located in Edison, New Jersey. The grant supports the development of a novel, chemically modified ribonucleoprotein (CRNP) delivery system for CRISPR-based gene editing to treat Angelman syndrome, a neurogenetic disorder. The project aims to optimize...

This federal Project Grant award from the National Institute of Neurological Disorders and Stroke (CFDA 93.853 - Extramural Research Programs in the Neurosciences and Neurological Disorders) to Merlin Therapeutics Inc. (UEI: MVKSHRPYUJ22) provides $1,256,291.00 in funding to support the development of an adeno-associated virus (AAV)-mediated gene therapy for the treatment of schwannomas. The gene therapy, termed MRL-102, utilizes a pro-apoptotic transgene to target and eliminate schwannoma...

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, is focused on developing a novel drug candidate to treat demyelinating neuropathies. The $352,073 award will support rigorous in vivo testing of the experimental ABCA1 agonist drug, CS6253, in validated mouse models of hereditary peripheral nerve disorders characterized by impaired...

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 $699,559 to Merlin Therapeutics Inc. to develop a novel adeno-associated viral vector 9 (AAV9) gene therapy for the treatment of neurofibromatosis type 2 (NF2). The therapy aims to express a healthy copy of the NF2 gene and rescue tumor characteristics in NF2...

This $226,500 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 a 2-year research project to characterize and treat peripheral nervous system dysfunction in Sanfilippo syndrome (mucopolysaccharidosis type IIIA). The award will enable researchers at the University of Georgia Research Foundation to develop novel disease...

This federal Project Grant award of $238,500 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 conducted by the University of California, San Diego (UCSD) to develop a new phosphorodiamidate morpholino oligonucleotide (PMO) therapeutic for treating Alzheimer's disease (AD). The research aims to create a highly effective PMO that can...

This 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), is focused on developing a novel nanovesicle-based therapy for myotonic dystrophy type 1 (DM1), a common adult-onset muscular dystrophy. The $306,859 award supports a two-phase effort to validate interfering RNA-loaded nanovesicles that can selectively modulate the toxic DMPK mRNA responsible for...

Renerva LLC, a for-profit manufacturer, is developing peripheral nerve matrix (PNM) technologies to facilitate functional recovery in animal models of direct muscle neurotization (DMN) through a $494,955 Project Grant from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853). The two-year project aims to 1) optimize the formulation of a clinical-grade PNM product to provide...