Tackling the CMC Challenges for Cell and Gene Therapy Product Development

RAC Credits
Virtual Programs Virtual Programs
Wednesday, 03 August 2022 (12:00 PM) - Wednesday, 03 August 2022 (2:00 PM) Eastern Time (US & Canada)

The curative potential of cell and gene therapies (CGTs) has led to an explosion of interests and investment for this novel therapeutic modality, and there are currently over 2,400 active INDs in various stages of clinical development. While parallels can be drawn with small molecule drugs and protein therapeutics, there are a number of unique and significant challenges for CGT manufacturing: variability in source materials, limited material for testing, absence of reliable reference standards, maintaining the chain of identity and custody, and the human cost of manufacturing failures, just to name a few. Furthermore, many CGTs are on an expedited development path, leading to compressed CMC development timelines and possible misalignment with clinical development. As a result, establishing proper manufacturing control can become bottlenecks for product registration. This unusual situation was best captured by former FDA Commissioner Scott Gottlieb in his speech at the Alliance for Regenerative Medicine's Annual Board Meeting: “In contrast to traditional drug review, where 80 percent of the review is focused on the clinical portion of that process, and maybe 20 percent is focused on the product issues, I’d say that this general principle is almost completely inverted when it comes to cell and gene therapy.”

Although each product has its own unique challenges, there are common themes in successful CMC development for diverse CGT products. In this symposium, key opinion leaders from academia, industry, and a regulatory agency will discuss these strategies in addressing CGT manufacturing challenges to bring transformative medicines with consistent quality to patients. Hot topics include potency assays, comparability studies, and common quality issues for CGT manufacturing.

Registration Fees & Deadlines

1 June 2022 – 3 July 2022: Early Bird $195 member | $230 nonmember
4 July 2022 – 3 August 2022: Regular $230 member | $270 nonmember

Learning Objectives

After the event, the attendees should be able to

  • describe the unique challenges in cell and gene therapy manufacturing
  • understand strategies in CMC development of cell and gene therapy investigational products

Learning Level

Intermediate: Content is designed based upon the assumption that individuals have basic knowledge of the topic(s) and/or demonstrated competence related to the topic(s). Higher-level concepts are introduced during lectures; exercises requiring synthesis and/or application of concepts are incorporated into the activity.

Who Should Attend?

Regulatory professionals working or interested in Cell and Gene therapy investigational products.


Bruce Levine

Bruce Levine
University of Pennsylvania School of Medicine

Steven Oh

Steven Oh
U Food & Drug Administration

Chuck Calderaro

Chuck Calderaro
Kite Pharma

Don Fink

Don Fink
Dark Horse Consulting Group

Cancellation Policy

RAPS reserves the right to cancel this program at its sole discretion. RAPS will not be responsible for travel or other costs incurred due to cancellation. To transfer a registration, email support@raps.org with the event title, name of the original registrant and the contact information for the new attendee.

Proof of Attendance

RAPS will send an electronic letter or a certificate of attendance at the following the workshop.


Contact the RAPS Support Center:
Call +1 301 770 2920, ext. 200 (8:30 am–5:30 pm EST, Monday–Friday) or email support@raps.org

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