Project Grant R21TR005433
Award Date
7/1/25
Completion Date
6/30/26
Dollars Obligated
$402K
Federal Agency
National Center for Advancing Translational Sciences
Federal Grant Program
93.350
Assistance Type
Project Grant
Place of Performance
Pennsylvania, USA
Similar Awards
This Project Grant award from the Food and Drug Administration (FDA), under the FDA Research program (CFDA 93.103), provides $826,251 to The Trustees of the University of Pennsylvania to conduct a clinical trial of gene augmentation therapy for patients aged 13-18 with Leber Congenital Amaurosis type 5 (LCA5), a rare inherited retinal disease causing severe vision loss. The award will also support the development of novel outcome measures for evaluating treatments in younger pediatric LCA5...
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 Human Genome Research Institute (CFDA 93.172 - Human Genome Research) totaling $1,276,313 aims to explore ethical challenges and develop management strategies for patient-focused drug development (PFDD) in rare disease research. The primary objectives are to: 1) conduct stakeholder interviews to identify ethical challenges in PFDD, 2) characterize stakeholder values, interests, and relationships to pinpoint areas of conflict and alignment, and...
This $506,984 Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), aims to investigate the role of the LRP1 gene in the development of congenital heart disease (CHD). The primary objectives are to: Examine the deployment and migration of cardiac neural crest cells (CNCCs) and their dependence on LRP1 using in vitro and in vivo cell lineage fate mapping analysis. Investigate the cellular and molecular...
This Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) will support research to investigate the genetic and environmental factors contributing to the development of complex lymphatic anomalies (CLAs), a group of rare and debilitating vascular diseases. The $508,436 award to The University of Texas Southwestern Medical Center will test the hypothesis that CLAs require both a genetic mutation and a permissive lymphangiogenic...
This Project Grant award from the National Center for Advancing Translational Sciences (NCATS), under CFDA program 93.350, provides $489,500.00 to The Children's Hospital of Philadelphia Research Institute to conduct research on familial platelet disorder with associated myeloid malignancy (FPDMM). The key objectives are to study the effects of the compound Repsox on the RUNX1 transcription factor to correct both the platelet defects and leukemic risk associated with FPDMM, a rare genetic...
This $299,504 Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), supports the development of a novel therapeutic approach to clear small dense low-density lipoprotein (SDLDL) from circulation. The research aims to reengineer a proprietary APOC-III antagonist to include bioactive APOE mimetic peptides, creating a dual-domain therapeutic candidate that can bind APOC-III on SDLDL particles and...
This Project Grant awarded by the Food and Drug Administration (FDA) Office of Orphan Products Development under the FDA Research program (CFDA 93.103) will provide $479,560 to Palvella Therapeutics, Inc. to conduct a Phase 3 clinical trial evaluating the safety and efficacy of PTX-022, a 3.9% topical sirolimus gel, for the treatment of microcystic lymphatic malformations (microcystic LM). Microcystic LM is a rare, serious, and chronic disease of the lymphatic system with no FDA-approved...
The National Center for Advancing Translational Sciences (NCATS), under the CFDA Program 93.350, awarded a $295,923 Project Grant to Kinetiq Therapeutics LLC to conduct preclinical efficacy studies of a subcutaneous enzyme therapy for Fabry disease. The proposed therapy, named BRYSQ, aims to transform the current standard of care by providing a convenient, self-administered treatment that could improve patient quality of life, reduce hospital use, and lower treatment costs compared to the...
This federal Project Grant award of $694,134 was 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) program. The award aims to develop an approach to efficiently engineer autologous hematopoietic stem cells (HSCs) with a CSF1R inhibitor resistant variant and conditional galactosylceramidase (GALC) overexpression. This therapeutic approach seeks to enhance the...