Project Team

The PaVe-GT pilot project is a collaborative effort that applies NCATS’ translational science model by bringing experts from multiple Institutes and Centers together to work as a single team. These collaborators will work together to test a translational science hypothesis: Can researchers significantly increase the efficiency of gene therapy trial startup by using the same capsid and manufacturing methods for different AAV gene therapies, thereby minimizing redundancies in the preclinical development of gene therapies for extremely rare diseases?

NCATS Approach to Translation: Develop, Demonstrate, Disseminate. Develop: NCATS designs a translational science experiment. Demonstrate: NHGRI and NINDS initiate and conduct four gene therapy programs. NCATS coordinates and harmonizes the trials. Disseminate: NCATS will publish all communications with the FDA, in partnership with NHGRI and NINDS.

National Center for Advancing Translational Sciences

NCATS is applying its collaborative translational science model to transform understanding, diagnosis and treatment of rare diseases. NCATS’ fundamental approach is to shift from considering each rare disease in isolation to identifying and developing treatments for rare diseases based on their commonalities. NCATS invests resources and expertise at the points where research is most difficult and therefore often abandoned. Translation is a team sport. The NCATS Division of Preclinical Innovation (DPI), Division of Rare Diseases Research Innovation (DRDRI), and Office of Strategic Alliances (OSA) work together to achieve PaVe-GT objectives.

Krishna Krishna "Balki" Balakrishnan, Ph.D., M.B.A.
Acting Director, Office of Strategic Alliances
P.J. Brooks, Ph.D. P.J. Brooks, Ph.D.
Acting Director, Division of Rare Diseases Research Innovation
Catherine Chen, Ph.D. Catherine Chen, Ph.D.
Research Scientist, Biology
Richa Lomash, Ph.D. Richa Lomash, Ph.D.
Scientific Project Manager
Venkata Mangalampalli, PhD Venkata Mangalampalli, PhD
Staff Scientist, Chemistry, Manufacturing and Controls (CMC)
Elizabeth Ottinger, Ph.D. Elizabeth Ottinger, Ph.D.
Acting Director, Therapeutic Development Branch
Rodica Stan, Ph.D. Rodica Stan, Ph.D.
Senior Project Manager
Mitali Tambe, Ph.D. Mitali Tambe, Ph.D.
Pramod Terse, Ph.D. Pramod Terse, Ph.D.
Team Leader, Safety Evaluation/Toxicology
London Toney, M.S. London Toney, M.S.
Scientific Project Analyst
Sury Vepa, Ph.D., J.D. Sury Vepa, Ph.D., J.D.
Senior Licensing and Patenting Manager
Erik Wagner Erik Wagner
Research Scientist, Bioanalytical
Amy Wang, Ph.D Amy Wang, Ph.D
Staff Scientist, Bioanalytical
Xin Xu, Ph.D. Xin Xu, Ph.D.
Director, Pharmacokinetics
National Human Genome Research Institute (NHGRI)

The NHGRI Division of Intramural Research develops and implements technology to understand, diagnose and treat genomic and genetic diseases. The NHGRI Organic Acid Research Section, led by Dr. Charles Venditti, focuses on the genetics, pathophysiology and treatment of the hereditary methylmalonic acidemias and disorders of intracellular cobalamin metabolism.

Eggerton Campbell, Ph.D. Eggerton Campbell, Ph.D.
Senior Licensing and Patenting Manager
Randy Chandler, Ph.D. Randy Chandler, Ph.D.
Staff Scientist
Eun-Young Choi, Ph.D. Eun-Young Choi, Ph.D.
Research Scientist
Susan Ferry, RN, B.S.N. Susan Ferry, RN, B.S.N.
Research Nurse Specialist
Irini Manoli, M.D., Ph.D. Irini Manoli, M.D., Ph.D.
Senior Clinician
Lina Li, M.D., Ph.D. Lina Li, M.D., Ph.D.
Research Scientist
Oleg Shchelochkov, M.D. Oleg Shchelochkov, M.D.
Senior Clinician
Jennifer Sloan, Ph.D., M.S., CGC Jennifer Sloan, Ph.D., M.S., CGC
Protocol Coordinator and Genetic Counselor
Carol Van Ryzin, RN, CPNP Carol Van Ryzin, RN, CPNP
Pediatric Nurse Practitioner
Charles Venditti, M.D., Ph.D. Charles Venditti, M.D., Ph.D.
Chief, Metabolic Medicine Branch
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Dr. Forbes D. Porter, M.D., Ph.D. is the Clinical Director for NICHD and serves as the Clinical Director for NCATS.

Forbes D. Porter, M.D., Ph.D. Forbes D. Porter, M.D., Ph.D.
Senior Investigator
National Institute of Neurological Disorders and Stroke (NINDS)

Investigators in the NINDS intramural program conduct research in the basic, translational and clinical neurosciences. The NINDS Neuromuscular and Neurogenetic Disorders of Childhood Section, led by Dr. Carsten Bonneman, focuses on early-onset neuromuscular disorders of childhood, on their genetic and molecular pathogenesis and the pathways involved, and on developing preclinical translational models to lead to actual clinical trials.

Gilberto V. Averion, RN, B.S.N. Gilberto V. Averion, RN, B.S.N.
Clinical Research Nurse
Carsten Bonnemann, M.D. Carsten Bonnemann, M.D.
Senior Investigator
Steven J. Burden, Ph.D. Steven J. Burden, Ph.D.
Scientific Advisor, Skirball Institute, NYU Medical School
Oksana Dukhanina, Ph.D. Oksana Dukhanina, Ph.D.
Senior Technology Portfolio Manager
A. Reghan Foley, M.D. A. Reghan Foley, M.D.
Staff Clinician
Adam Lin Adam Lin
Postbac IRTA
Christopher Mendoza Christopher Mendoza
Patient Care Coordinator
Rotem Orbach, M.D. Rotem Orbach, M.D.
Clinical Research Fellow
Julien Oury, Ph.D. Julien Oury, Ph.D.
Postdoctoral Fellow, Skirball Institute, NYU Medical School
Pankaj Pathak, Ph.D. Pankaj Pathak, Ph.D.
Research Fellow
Joshua J. Todd, Ph.D. Joshua J. Todd, Ph.D.
Staff Scientist, Clinical Trials Unit

The PaVe-GT team gratefully acknowledges the contributions of  former team members including: Asaf Alimardanov, Ph.D.; Jean Dehdashti, RAC; Claire Driscoll, M.S.; Eric Esposito; Janelle Hauserman, Ph.D.; Donald Lo, PhD; Anne Pariser, M.D; Deanna Portero; Lili Portilla, MPA; Dimah Saade, M.D.. 


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    Pave-GT Resources
    Rare Pediatric Disease (RPD) Designation Request for AAV9-hPCCA

    This pdf file contains the RPD designation request for AAV9-hPCCA (NCATSBL-0746) and associated communications between NCATS and FDA OOPD.

    Orphan Drug Designation (ODD) Request for AAV9-hPCCA

    This pdf file contains the ODD request for AAV9-hPCCA (NCATSBL-0746) and associated communications between NCATS and FDA OOPD.

    National Institutes of Health