This Project Grant award from the National Center for Advancing Translational Sciences (NCATS), CFDA Program 93.350, is focused on evaluating gene replacement therapy as a potential treatment for SCYL1 deficiency syndrome, a rare pediatric disorder. The $158,500 award will support the development of two mouse models to study the effects of restoring SCYL1 gene expression. One model will use a Cre-loxP system to conditionally activate SCYL1 expression, while the other will employ an inverted...
This federal Project Grant award, totaling $249,000.00 and spanning from September 1, 2024 to August 31, 2027, was provided by the National Institute of Child Health and Human Development (NICHD) under the Child Health and Human Development Extramural Research program (CFDA 93.865). The primary objective of this grant is to identify new therapies and elucidate molecular mechanisms for Congenital Disorders of Glycosylation (CDGs), a group of rare developmental disorders caused by inborn errors in...
This $110,000 Project Grant award from the National Center for Advancing Translational Sciences (NCATS) under CFDA 93.350 supports research by Greenstone Biosciences, Inc. to develop advanced drug discovery and development technologies for rare genetic disorders. The project aims to leverage induced pluripotent stem cell (iPSC) technology and artificial intelligence/machine learning (AI/ML) to accelerate and streamline the drug discovery process for myotonic dystrophy type 1 (DM1), a rare and...
This federal Project Grant award of $498,000.00 from the National Institute of Child Health and Human Development (NICHD), under the Child Health and Human Development Extramural Research program (CFDA 93.865), aims to develop the first murine animal model and adeno-associated virus (AAV)-based gene therapy for mNATP6 mitochondrial diseases. The primary objectives are to: 1) Generate a conditional truncated mNATP6 mouse model using a novel mitochondria-targeting base editing technology, and 2)...
This Project Grant award from the National Institutes of Health (NIH) under the Research Infrastructure Programs (CFDA 93.351) provides $844,954 to New York University School of Medicine to develop "Genomically Rewritten and Tailored Humanized Mouse Models for Various Organ Disorders". The project leverages advanced DNA synthesis and genome editing technologies to create mouse models that accurately represent human genetic variations and disease susceptibilities. These...
This Project Grant awarded by the National Institute of Child Health and Human Development (NICHD), under the Child Health and Human Development Extramural Research program (CFDA 93.865), aims to reverse the disrupted developmental sterol biosynthesis caused by prescription medications. The $230,250 award supports research to explore treatments for Smith-Lemli-Opitz Syndrome (SLOS), a developmental disorder resulting from compound heterozygous mutations in the DHCR7 gene that disrupt cholesterol...
This federal Project Grant award from the National Center for Advancing Translational Sciences (NCATS), CFDA 93.350, provides $110,000 to Myogene Bio LLC to conduct preclinical research on the long-term durability of a gene editing therapy for Duchenne muscular dystrophy (DMD). The gene editing therapy is designed to remove DMD exons 45-55 to restore the reading frame for 50% of DMD patients while retaining 87% of the protein coding sequence. The funded studies will assess molecular and...
This National Institutes of Health (NIH) Research Infrastructure Programs (CFDA 93.351) Project Grant award of $743,926 to the University of Oregon will develop a resource of rigorously validated, translatable zebrafish models of previously undiagnosed human genetic diseases. The project will leverage collaboration with clinical sites and research consortia to analyze and prioritize genetic variants from patients with rare undiagnosed diseases. CRISPR/Cas9 technology will then be used to...
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 from the National Institutes of Health (NIH) under the Research Infrastructure Programs (CFDA 93.351) provides $235,500 to Cornell University to develop novel mouse models and genetic tools for studying complex human diseases. The key products and services to be delivered through this 24-month award, beginning September 1, 2024, include: Generating a "unilateral mosaic" mouse model that can restrict disease-causing gene expression to one side of an organ (e.g.,...