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FDA Roundtable: Demystifying the FDA for Biologics Development

Recording of a Learn and Connect event sponsored by Children's Hospital of Philadelphia Research Institute's Innovation Ecosystem

Published onMay 16, 2022
FDA Roundtable: Demystifying the FDA for Biologics Development
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The FDA approval process can be a significant and necessary hurdle to bringing novel medical products and devices developed in research laboratories to patients in our hospitals.

This FDA Roundtable aimed to jumpstart and centralize efforts among CHOP faculty and staff who must navigate the FDA approval process. This session focused on novel drugs and biologics, and provided time at the end to network and connect with colleagues.

Our panelists played vital roles in CHOP’s efforts to achieve FDA approval of two groundbreaking biologic therapeutics, Kymriah and Luxturna. Kymriah is the first FDA-approved CAR-T therapy, and Luxturna is the first FDA-approved in vivo gene therapy.

This Event took place on May 11, 2022 at the Children’s Hospital of Philadelphia.

Presenters


Moderator:
Dr. Chip Smith, DVM, MS Technical Director of the Comparative Medicine Services Core

Panelists:
Dr. Stephan Grupp, PhD, MD, Director of Translational Research in the Center for Childhood Cancer Research

Sarah McCague, MS, Clinical Research Program Manager Clinical In-Vivo Gene Therapy Team

Presentation


Each presenter’s video and the Q&A session are listed below:

Kymriah with Dr. Stephan Grupp

Part 1: Kymriah - First FDA Approved Gene Therapy, Dr. Stephan Grupp [20 min]

Luxturna with Sarah McCague, MS

Part 2: Gene Therapy Approval - Site Perspective, Sarah McCague, MS

Q&A With Dr. Chip Smith, Dr. Stephan Grupp and Sarah McCague, MS

Part 3: CHOP FDA Roundtable ft. Stephan Grupp, MD, Sarah McCague, MS, and Chip Smith, DVM [17 min]

Related and Referenced Chapters


Overview of Drug Development

Gooneratne, N. (2019). Overview of Drug Development. In Academic Entrepreneurship for Medical and Health Sciences. https://doi.org/10.21428/b2e239dc.84c3ee98

Orphan Drugs: Understanding the FDA Approval Process

Srivastava, G., & Winslow, A. (2019). Orphan Drugs: Understanding the FDA Approval Process. In Academic Entrepreneurship for Medical and Health Sciences. https://doi.org/10.21428/b2e239dc.381f7d37

Preclinical Animal Models

Park, S. E., & Schaer, T. P. (2019). Preclinical Animal Models. In Academic Entrepreneurship for Medical and Health Sciences. https://doi.org/10.21428/b2e239dc.034dc5a5

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