Project Grant R41HD092110

The National Institute of Child Health and Human Development (NICHD) awarded a $295,923 Project Grant to Biostrategies LC under the Child Health and Human Development Extramural Research program (CFDA 93.865). The purpose of the award is to develop a "delivery-enhanced" gene therapy drug comprising an RTB:GALNS fusion protein for treating the rare metabolic disorder Mucopolysaccharidosis Type IVA (Morquio A Syndrome). The key objectives are to: 1) optimize the RTB:GALNS construct for...

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...

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 from the National Institute of Child Health and Human Development (NICHD), under the Child Health and Human Development Extramural Research program (CFDA 93.865), will fund the design and preparation of diagnostic biomarker reference standards for mucopolysaccharidosis type I (MPS-I) and type II (MPS-II). These lysosomal storage disorders are included in newborn screening programs, but current first-tier enzyme activity screening has a high rate of false...

This Project Grant award of $226,500.00, 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) federal grant program, aims to develop novel cellular models to improve understanding of peripheral nervous system (PNS) dysfunction in Sanfilippo syndrome (mucopolysaccharidosis type IIIA). The primary objectives are to utilize patient-derived induced pluripotent stem cells to...

This Cooperative Agreement award from the National Institute of Neurological Disorders and Stroke (NINDS), part of the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) federal grant program, provides $2,285,718.00 in funding to the University of Florida to advance the development of an AAV8 gene therapy vector for the treatment of Sanfilippo Syndrome Type B. The project aims to complete the necessary preclinical studies, assay development, toxicology...

This $6,925,376 Cooperative Agreement awarded by the National Institutes of Health (NIH) Office of the Director under the Trans-NIH Research Support program (CFDA 93.310) aims to develop a master protocol for rapid development of personalized prime-editing therapies for severe, rare hepatic inborn errors of metabolism (IEMs). The primary objectives are to establish a therapeutic platform using a single prime editor and AAV delivery system for numerous hepatic IEMs, and to streamline regulatory...

This Project Grant award of $306,872.00 from the National Institute of General Medical Sciences (NIGMS), under the Biomedical Research and Research Training Program (CFDA 93.859), aims to develop a new 3D shape matching methodology that accounts for water molecules when comparing ligands during drug discovery. The key products and services to be delivered include: Adapting an existing algorithm (WATGEN) to predict water positions in unbound proteins and protein-ligand complexes, and calculate...

This Project Grant award from the National Institute of General Medical Sciences (NIGMS), under the Biomedical Research and Research Training program (CFDA 93.859), provides $202,274 to develop an easy-to-use computer program for predicting the structure and degrading efficiency of proteolysis targeting chimeras (PROTACs). PROTACs are heterobifunctional molecules that induce the degradation of target proteins by recruiting them to E3 ubiquitin ligases. The awarded project aims to implement a...

The National Institute of General Medical Sciences (NIGMS) awarded a $379,131 Project Grant under the Biomedical Research and Research Training program (CFDA 93.859) to the University of North Carolina at Chapel Hill. The 5-year project aims to develop rapid characterization strategies and predictive algorithms to guide the design of macromolecule-based therapeutics, such as peptides and peptidomimetics, for disrupting protein-protein interactions. Key objectives include establishing...