This Cooperative Agreement award from the National Institutes of Health (NIH) under the Trans-NIH Research Support program (CFDA 93.310) provides $856,513 to optimize and integrate two computational and experimental approaches, CRISPRME and ABSOLVE-SEQ, into a comprehensive pipeline for assessing variant-associated off-target risks in genome editing therapeutics. The award aims to support Investigational New Drug (IND) applications by delivering standardized software modules, standard...
This Project Grant award from the National Institute of General Medical Sciences (NIGMS) Biomedical Research and Research Training Program (CFDA 93.859) provides $445,000.00 to The Children's Hospital of Philadelphia (CHOP) to develop technologies for delineating haplotype-resolved, full-length transcriptomes in bulk tissues and single cells. The project aims to characterize transcript isoforms of haploinsufficient genes to discover novel therapeutic targets, as well as establish a program for...
This Project Grant award from the National Institutes of Health (NIH) under the Trans-NIH Research Support program (CFDA 93.310) provides $1,602,000.00 to The Children's Hospital of Philadelphia Research Institute to develop and optimize a new class of Epigenetic Reprogramming Factors (ERFs) that can target hundreds, thousands, or tens of thousands of DNA regions in a pathway-specific manner. The goal is to leverage this ERF technology to prevent or reverse epigenetic signatures associated...
This federal Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 - Cardiovascular Diseases Research) provides $667,704 to The Trustees of the University of Pennsylvania, doing business as the Clinical Practices of the University of Pennsylvania, to develop chemically inducible split base editors for precise and controllable in vivo genome editing. The goal is to address the root cause of hypertrophic cardiomyopathy (HCM), a genetic heart condition affecting 1 in...
This $356,000 Project Grant award from the Defense Health Agency under the Military Medical Research and Development program (CFDA 12.420) supports the development of a novel gene therapy for mitochondrial complex I deficiency at the Children's Hospital of Philadelphia (CHOP). The award period runs from March 1, 2024 to February 28, 2026. CHOP, a leading pediatric hospital and research institution, will leverage its expertise in areas like rare genetic disorders to advance this innovative...
This Project Grant award from the National Institutes of Health (NIH) Office of Research Infrastructure Programs (CFDA 93.351) provides $222,500.00 to The Children's Hospital Corporation (doing business as Boston Children's Hospital) from May 15, 2025 to March 31, 2027. The funding supports the development of a modular system for mouse CRISPR genome and epigenome editing, including the creation of mouse lines that express CRISPR-interference/activation (CRISPR-I/A) and base/prime editing...
This Cooperative Agreement award, provided by the National Institutes of Health (NIH) under the Trans-NIH Research Support program (CFDA 93.310), is focused on evaluating a high-throughput extracellular vesicle (EV) loading platform for therapeutic genome editing. The $452,378 award to the University of Florida aims to optimize and assess this EV loading platform for GMP manufacturing, establish critical quality attributes (CQAs) for EV-based genome editing therapeutics, and conduct...
This Project Grant award, provided by the National Institutes of Health (NIH) under the Research Infrastructure Programs (CFDA 93.351), aims to rapidly develop advanced mouse resource strains through the application of Prime Editing technology. The $265,500 award, granted on September 1, 2024, will fund research at the Jackson Laboratory to create a novel combinatorial system that can efficiently modify thousands of mouse genes by applying Prime Editing techniques. The key innovation is the...
This Project Grant award from the National Human Genome Research Institute (CFDA 93.172 - Human Genome Research) aims to gain a multi-perspective understanding of the ethical challenges arising in patient-focused drug development (PFDD) for rare diseases and to develop tools to manage these challenges. The $1,276,313 award, effective February 1, 2025 through January 31, 2029, will fund a study with three key objectives: 1) explore stakeholder perspectives on ethical issues in PFDD, 2)...
This 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), provides $178,000.00 to The Children's Hospital of Philadelphia (CHOP) to design and validate a Pelizaeus Merzbacher Disease (PMD) severity scale. The goal is to develop a clinician-reported outcome measure that can be used to stratify PMD patients by disease severity, particularly early in the disease...