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Dwight D. Koeberl

Professor of Pediatrics
Pediatrics, Medical Genetics
Box 103856 DUMC, Durham, NC 27710
595 LaSalle Street, Gsrbi, Room 4006, Durham, NC 27710

Selected Grants


Stable therapy in Pompe disease through genome editing

ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2021 - 2026

Cell and Molecular Biology Training Program

Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 2021 - 2026

Genetic and Genomics Training Grant

Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 2020 - 2025

A 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 - 2025

Stable therapy in Pompe disease through genome editing

ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2021 - 2026

Cell and Molecular Biology Training Program

Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 2021 - 2026

Genetic and Genomics Training Grant

Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 2020 - 2025

A 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 - 2025

Unified Program for Therapeutics in Children

Inst. Training Prgm or CMEPreceptor · Awarded by National Institutes of Health · 2019 - 2024

Viral Oncology Training Grant

Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 1980 - 2024

Reformulation of Pharmaceutical to Treat ALS

ResearchPrincipal Investigator · Awarded by North Carolina Biotechnology Center · 2020 - 2022

Genome editing for the correction of Pompe disease

ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2020 - 2022

Identifying Pathogenic Non-Coding Mutations in Rare Mendelian Disease

ResearchCo Investigator · Awarded by National Institutes of Health · 2019 - 2022

Observational Study of Males with Creatine Transporter Deficiency

Clinical TrialPrincipal Investigator · Awarded by Ultragenyx Pharmaceutical · 2017 - 2021

Investigating Autophagy in GSD-Ia

ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2020 - 2021

Pharmacodynamic 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 - 2021

Feasibility of using Bortezomib-based immunosuppressive approach to deplete anti-AAV antibodies in mice

ResearchCo Investigator · Awarded by Asklepios BioPharmaceutical, Inc. · 2018 - 2020

Genetics Training Grant

Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 1979 - 2020

Organization and Function of Cellular Structure

Inst. Training Prgm or CMEMentor · Awarded by National Institutes of Health · 1975 - 2020

A 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 - 2020

Potency Assays

ResearchPrincipal Investigator · Awarded by Actus Therapeutics · 2018 - 2019

Translational studies of GAA deficiency in bioengineered human muscle

ResearchCo-Principal Investigator · Awarded by National Institutes of Health · 2013 - 2019

LDN: CRIM responses in Pompe disease

ResearchCo Investigator · Awarded by University of Minnesota · 2009 - 2019

Rapid Neonatal Testing for Ammonia Disorder Biomarkers

ResearchPrincipal Investigator · Awarded by Baebies, Inc · 2018 - 2019

Inborn Errors of Long Chain Fat Metabolism

ResearchPrincipal Investigator · Awarded by University of Pittsburgh · 2016 - 2019

Activity and Biodistribution of the AAV2/8-LSPhGAA in GAA-knockout mice

ResearchInvestigator · Awarded by Actus Therapeutics · 2018 - 2018

New Indications for Beta2 Agnoists in Glycogen Storage Disease Type Ia, NAFLD/NASH, and Pompe Disease

ResearchPrincipal Investigator · Awarded by Roivant Sciences, Inc. · 2015 - 2018

TBR agonists for treatment in GSD Ia

ResearchPrincipal Investigator · Awarded by Viking Therapeutics, Inc. · 2016 - 2018

Supplemental Funding for Phase 1/2 study of Clenbuterol for the Treatment of Pompe Disease

Clinical TrialPrincipal Investigator · Awarded by Roivant Sciences, Inc. · 2015 - 2016

Phase 1/2 Study of Clenbuterol for the Treatment of Pompe Disease

Clinical TrialPrincipal Investigator · Awarded by Food and Drug Administration · 2013 - 2016

Lysosomal enzymes, Glycosaminoglycans and Outflow Pathway Physiology

ResearchCollaborator · Awarded by Glaucoma Research Foundation · 2015 - 2016

Dietary therapy in mitochondrial trifunctional protein deficiency

ResearchPrincipal Investigator · Awarded by Ultragenyx Pharmaceutical · 2014 - 2015

Training course for expanded (MS/MS) newborn screening laboratory follow-up coordinators

ConferenceTraining Faculty · Awarded by Association of Public Health Laboratories · 2014 - 2014

Clinical Trial Planning in Pompe Disease

ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2013 - 2014

Gene delivery to striated muscle by systemic AAV vectors

ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2006 - 2012

Mechanisms for immune tolerance in Pompe Disease

ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2009 - 2011

External Relationships


  • Acacia Therapeutics Inc.
  • AskBio
  • Bayer
  • Moderna Therapeutics, Inc.
  • Pfizer Inc.
  • 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.