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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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A major hurdle to bringing novel medical products and devices developed in research laboratories to patients in our hospitals is the FDA approval process.

This FDA Roundtable aimed to jumpstart and centralize efforts among CHOP faculty and staff who struggle with 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

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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 by Sarah McCague MS


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

Part 3 - CHOP FDA Roundtable: Stephan Grupp, MD, Sarah McCague, MS, and Chip Smith, DVM (17 min)

Related and Referenced Chapters

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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


Referenced Resources and Centers at CHOP

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