American Society of Hematology

NCATS Seeking Information on Opportunities and Challenges for Platform Vector Human Gene Therapy Trials in Rare Diseases

Published on: September 01, 2017

The National Center for Advancing Translational Sciences (NCATS) released a request for information soliciting input on the use of viral vectors as platforms for therapeutic gene delivery to specific organs or cell types, and how such platforms might be developed most efficiently in clinical trials for the treatment of multiple diseases. Specifically, comments are requested in the following areas:

  • The most important factors limiting PVGT trials. Such factors may include, but are not limited to: vector design, biodistribution/toxicology studies, immune responses, clinical trial design, funding mechanisms, business strategy, intellectual property, and regulatory science.
  • Implications of PVGT trials for rare disease patients, policy-makers, regulatory agencies, biotech companies, and non-profit and philanthropic organizations.
  • Negative or unintended consequences to PVGT trials.
  • The existence of many rare diseases with small numbers of patients and how this affects the opportunities and challenges for PVGT trials.
  • Types of partnerships or collaborations that could facilitate PVGT trials.
  • Considerations and trade-offs between the development of new vectors and the use of existing vectors for PVGT trials.
  • Other strategies for accelerating human gene therapy clinical trials in rare diseases, aside from PVGT trials.
  • Impediments to extrapolating clinical pharmacology, safety, or effectiveness assessments from one gene therapy trial to another when the same vector is used.

ASH members are encouraged to submit comments by Tuesday, September 19, 2017, in response to this request. To submit comments, email NCATSGTPlatformRFI@mail.nih.gov. For additional questions regarding this solicitation, please contact Dr. Philip John Brooks, Program Director in the Division of Clinical Innovation at NCATS.

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