The Pathway from Idea to Regulatory Approval: Considerations for Clinical Trial Applications and Regulatory Clearances

March 21, 2023 4:00pm

Jason Bablak
Vice President, Regulatory Science, U.S.
Orchard Therapeutics

Deborah Cho
Hogan Lovells
(Former Associate Chief Counsel, FDA)

Charles G. Raver
Hyman, Phelps & McNamara PC

Deborah Hursh
Hursh Cell Therapy Consulting, LLC
(Former Senior Investigator, CMC Reviewer, CBER of FDA)

FDA has issued draft guidances addressing the development of the new modalities driving the biopharma revolution. These guidances offer recommendations on the information that should be included in an Investigational New Drug (IND) application. During this regulatory session, points of discussion will include:

  • Analyzing the key considerations put forth in the three available guidances on gene editing, CAR T products, and regenerative medicine therapies
  • Detailing FDA’s recommendations for preclinical and clinical testing, chemistry, manufacturing, and controls (CMC)
  • Outlining what FDA recommends be included in Investigational New Drug applications (IND)
    • Ensuring proper identity, potency/strength, quality and purity
  • Reviewing the already approved biopharma revolution therapies, ongoing clinical trials, and their impact on clinical practice