Selected Grants
A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
Clinical TrialPrincipal Investigator · Awarded by Moderna Therapeutics, Inc. · 2021 - 2029Triacetin Treatment for Propionic Acidemia by Rebalancing the Acetyl-CoA/Propionyl-CoA Metabolism
ResearchCollaborator · Awarded by National Institutes of Health · 2024 - 2026Stable therapy in Pompe disease through genome editing
ResearchPrincipal Investigator · Awarded by National Institute of Arthritis and Musculoskeletal and Skin Diseases · 2021 - 2026Cell and Molecular Biology Training Program
Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 2021 - 2026Genetic and Genomics Training Grant
Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 2020 - 2025A Phase 1 Study of the Safety of AAV8-LSPhGAA (ACTUS-101) in Late-onset Pompe Disease (LOPD)- Cohort II
Clinical TrialProgram Leader · Awarded by Asklepios BioPharmaceutical, Inc. · 2019 - 2025A Global, Phase 1/2, Open-label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia
Clinical TrialPrincipal Investigator · Awarded by Moderna Therapeutics, Inc. · 2021 - 2025Viral Oncology Training Grant
Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 1980 - 2025Unified Program for Therapeutics in Children
Inst. Training Prgm or CMEPreceptor · Awarded by National Institutes of Health · 2019 - 2024Reformulation of Pharmaceutical to Treat ALS
ResearchPrincipal Investigator · Awarded by North Carolina Biotechnology Center · 2020 - 2022Genome editing for the correction of Pompe disease
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2020 - 2022Identifying Pathogenic Non-Coding Mutations in Rare Mendelian Disease
ResearchCo Investigator · Awarded by National Institutes of Health · 2019 - 2022Observational Study of Males with Creatine Transporter Deficiency
Clinical TrialPrincipal Investigator · Awarded by Ultragenyx Pharmaceutical · 2017 - 2021Investigating Autophagy in GSD-Ia
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2020 - 2021MaP: Mapping the Patient Journey in Methylmalonic and Propionic Acidemia A longitudinal, exploratory, natural history study to further characterize and describe the signs and symptoms of patients with organic acidemias
ResearchPrincipal Investigator · Awarded by Moderna Therapeutics, Inc. · 2018 - 2021Pharmacodynamic study using rhaGLU in a Pompe mouse model. Nonclinical GAA-KO mouse study design proposal rhaGLU
ResearchPrincipal Investigator · Awarded by Pharming Group N.V. · 2019 - 2021A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency
Clinical TrialPrincipal Investigator · Awarded by Moderna Therapeutics, Inc. · 2019 - 2021Feasibility of using Bortezomib-based immunosuppressive approach to deplete anti-AAV antibodies in mice
ResearchCo Investigator · Awarded by Asklepios BioPharmaceutical, Inc. · 2018 - 2020A retrospective chart review study to assess the clinical outcome of triheptanoin treatment in patients with long-chain fatty acid oxidation disorders (LC-FAOD) treated under expanded access program
Clinical TrialPrincipal Investigator · Awarded by Ultragenyx Pharmaceutical · 2019 - 2020Genetics Training Grant
Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 1979 - 2020Organization and Function of Cellular Structure
Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 1975 - 2020A Phase I Study of the Safety of AAV2/8 LSPhGAA in Late-onset Pompe Disease
Clinical TrialPrincipal Investigator · Awarded by National Institutes of Health · 2017 - 2020Potency Assays
ResearchPrincipal Investigator · Awarded by Actus Therapeutics · 2018 - 2019Translational studies of GAA deficiency in bioengineered human muscle
ResearchCo-Principal Investigator · Awarded by National Institutes of Health · 2013 - 2019LDN: CRIM responses in Pompe disease
ResearchCo Investigator · Awarded by University of Minnesota · 2009 - 2019Rapid Neonatal Testing for Ammonia Disorder Biomarkers
ResearchPrincipal Investigator · Awarded by Baebies, Inc · 2018 - 2019Inborn Errors of Long Chain Fat Metabolism
ResearchPrincipal Investigator · Awarded by University of Pittsburgh · 2016 - 2019Activity and Biodistribution of the AAV2/8-LSPhGAA in GAA-knockout mice
ResearchInvestigator · Awarded by Actus Therapeutics · 2018 - 2018New Indications for Beta2 Agnoists in Glycogen Storage Disease Type Ia, NAFLD/NASH, and Pompe Disease
ResearchPrincipal Investigator · Awarded by Roivant Sciences, Inc. · 2015 - 2018TBR agonists for treatment in GSD Ia
ResearchPrincipal Investigator · Awarded by Viking Therapeutics, Inc. · 2016 - 2018Supplemental Funding for Phase 1/2 study of Clenbuterol for the Treatment of Pompe Disease
Clinical TrialPrincipal Investigator · Awarded by Roivant Sciences, Inc. · 2015 - 2016Phase 1/2 Study of Clenbuterol for the Treatment of Pompe Disease
Clinical TrialPrincipal Investigator · Awarded by Food and Drug Administration · 2013 - 2016Lysosomal enzymes, Glycosaminoglycans and Outflow Pathway Physiology
ResearchCollaborator · Awarded by Glaucoma Research Foundation · 2015 - 2016Dietary therapy in mitochondrial trifunctional protein deficiency
ResearchPrincipal Investigator · Awarded by Ultragenyx Pharmaceutical · 2014 - 2015Training course for expanded (MS/MS) newborn screening laboratory follow-up coordinators
ConferenceTraining Faculty · Awarded by Association of Public Health Laboratories · 2014 - 2014Clinical Trial Planning in Pompe Disease
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2013 - 2014Gene delivery to striated muscle by systemic AAV vectors
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2006 - 2012Mechanisms for immune tolerance in Pompe Disease
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2009 - 2011External Relationships
- Acacia Therapeutics Inc.
- AskBio
- Bayer
- Moderna Therapeutics, Inc.
- Pfizer Inc.
- Takeda Pharmaceutical Co. Ltd.
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.