Selected Grants
Gene therapy for glycogen storage disease type III
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2022 - 2027Stable therapy in Pompe disease through genome editing
ResearchCo Investigator · Awarded by National Institute of Arthritis and Musculoskeletal and Skin Diseases · 2021 - 2026CHARACTERIZATION OF GSD IX ¿2 and GSD VI MOUSE MODELS
ResearchInvestigator · Awarded by Kriya Therapeutics · 2022 - 2024AAV gene therapy for the treatment of Glycogen Storage Disease IX y2
ResearchInvestigator · Awarded by Kriya Therapeutics · 2022 - 2024Gene therapy by Lipid Nanoparticle-Mediated Delivery of Agl Messenger RNA in GSD III dogs
ResearchCo Investigator · Awarded by Ultragenyx Pharmaceutical · 2017 - 2023Gene therapy for glycogen storage disease type III
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2022 - 2027Stable therapy in Pompe disease through genome editing
ResearchCo Investigator · Awarded by National Institute of Arthritis and Musculoskeletal and Skin Diseases · 2021 - 2026CHARACTERIZATION OF GSD IX ¿2 and GSD VI MOUSE MODELS
ResearchInvestigator · Awarded by Kriya Therapeutics · 2022 - 2024AAV gene therapy for the treatment of Glycogen Storage Disease IX y2
ResearchInvestigator · Awarded by Kriya Therapeutics · 2022 - 2024Gene therapy by Lipid Nanoparticle-Mediated Delivery of Agl Messenger RNA in GSD III dogs
ResearchCo Investigator · Awarded by Ultragenyx Pharmaceutical · 2017 - 2023Optimized GAA (GAA) as potentially improved therapeutic for the treatment of Pompe disease
ResearchPrincipal Investigator · Awarded by Codexis, Inc. · 2020 - 2022Gene therapy for Adult polyglucosan body disease (APBD)
ResearchPrincipal Investigator · Awarded by University of Pennsylvania · 2020 - 2021Feasibility of using Bortezomib-based immunosuppressive approach to deplete anti-AAV antibodies in mice
ResearchPrincipal Investigator · Awarded by Asklepios BioPharmaceutical, Inc. · 2018 - 2020Activity and Biodistribution of the AAV2/8-LSPhGAA in GAA-knockout mice
ResearchPrincipal Investigator · Awarded by Actus Therapeutics · 2018 - 2018Preclinical study of use of SVP-Rapamycin to induce immune tolerance to ERT in Pompe disease mice
ResearchPrincipal Investigator · Awarded by Selecta Biosciences, Inc. · 2017 - 2018Evaluation of the use of Myozyme for treatment of glycogen storage disease type III and IV in murine disease models
ResearchPrincipal Investigator · Awarded by Roivant Sciences, Inc. · 2015 - 2018Liver-specific knockdown of M6PR with RNAi therapeutics to increase enzyme delivery to muscle tissues of Pompe disease mice during ERT
ResearchPrincipal Investigator · Awarded by Alnylam Pharmaceuticals · 2018 - 2018In vivo efficacy studies of Fab-GAA treatment for GSDII, III, and IV in murine disease models
ResearchPrincipal Investigator · Awarded by Valerion Therapeutics, Inc · 2015 - 2017To test the ability of SVP to prevent immune response in Pompe disease mouse model.
ResearchCo Investigator · Awarded by Selecta Biosciences, Inc. · 2015 - 2016External Relationships
- Asklepios BioPharmaceutical, Inc
- Bayer
- Kriya Therapeutics, Inc.
This faculty member (or a member of their immediate family) has reported outside activities with the companies, institutions, or organizations listed above. This information is available to institutional leadership and, when appropriate, management plans are in place to address potential conflicts of interest.