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Wednesday, June 19 • 1:15pm - 2:15pm
#345: Regulatory Considerations in Trial Design for Cell and Gene Therapies

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Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-648-L04-P; CME 1.00; RN 1.00

Cell and gene therapies have enormous potential to address unmet medical need. The application of validated clinical trials design tools along with biomarkers to assess safety and endpoints can help address regulatory uncertainty.

Learning Objectives

Discuss regulatory considerations for cell and gene therapies including the use of data modeling in clinical trial design and the application of biomarkers both for safety assessment and measures of outcome; Discuss how to plan for meeting regulatory requirements and be able to incorporate these plans into your company’s drug development plans.

Chair

Kanwaljit Singh, MD, MBA, MPH

Speaker

Regulatory Considerations for Development of Pediatric Cell and Gene Therapies: FDA Update
Najat Bouchkouj, MD

Regulatory Consideration for Pediatric Cell and Gene Therapies - Industry Perspective
Paulla Dennis

Path from Bench-to-Bedside for a Cell and Gene Therapy Drug Product
Stephanie Cherqui


Speakers
avatar for Najat Bouchkouj

Najat Bouchkouj

Associate Director for Pediatrics (Acting), Office of Clinical Evaluation, Offic, FDA, United States
Dr. Najat Bouchkouj is the Acting Associate Director for Pediatrics in the Office of Clinical Evaluation, OTP, CBER, FDA. Dr. Bouchkouj joined the FDA in 2016. She earned her medical degree from Damascus University, Syria and completed a residency in Pediatrics at the State University... Read More →
SC

Stephanie Cherqui

Director, UC San Diego Gene Therapy Initiative, University of California San Diego, United States
PD

Paulla Dennis

Senior Director, Rare Diseases, Advanced Therapies, and Pediatrics, Fortrea, United States
• Paulla Dennis, Senior Director, is a leading member of the Rare Disease, Advanced Therapies, and Paediatric Team (RAPT) with over 20 years in the clinical research industry. Paulla has expertise in global phase I through IV pediatric and rare disease operational strategy and planning... Read More →
avatar for Kanwaljit Singh

Kanwaljit Singh

Executive Director, International Neonatal Consortium, Critical Path Institute, United States

Wednesday June 19, 2024 1:15pm - 2:15pm PDT
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  06: TranslationalDelivery Tech-PrecMedicine, Session |   09: Regulatory, Session |   02: ClinTrials -ClinOps, Session