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Scientific presentation

Considerations, Strategies, and Advances in Early Drug Development to Mitigate Clinical Holds and Achieve Successful CGT Outcomes

Azadeh Golipour, PhD, AVROBIO
J. Kelly Ganjei, AmplifyBio
Scott R. Burger, MD, Advanced Cell and Gene Therapy
Matthew Cato


New and rapid innovations in cell and gene therapy (CGT) development are making the promise of personalized medicine closer to reality. However, processes for developing cell and gene therapies are inherently complex, posing new challenges that may impact therapeutic effectiveness and safety. Of note, there have been several recent delays to product approvals and clinical holds on various late-stage CGTs due to lack of product characterization and their correlation with de novo pathologies arising during clinical trial, respectively.

This engaging panel discussion covers:

  • Innovations in analytical characterization to improve outcomes
  • Regulatory guidance, best practices, and forward-looking considerations in early drug development that can lead to process improvements
  • Mitigating clinical holds
  • Latest technologies and innovative approaches in CGT development and manufacturing, including how early-stage deep characterization at the single-cell, multiomic level can provide the critical molecular insights that drives a successful path to the clinic.


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