Cell and gene therapy (CGT) development is entering a pivotal phase, driven by increasing reliance on integrated delivery systems, medical devices, and ancillary components. These therapies are no longer defined solely by the biologic or cellular product, but by the performance, control, and regulatory compliance of the complete combination product.
As regulatory expectations evolve across global health authorities, sponsors face growing complexity in aligning Chemistry, Manufacturing, and Controls (CMC) strategies for investigational CGT combination products. Differences in regulatory frameworks, device expectations, human factors requirements, and potency assessment approaches can introduce significant development risk if not addressed early.
This advanced, two-hour workshop provides a focused and practical exploration of global CMC regulatory considerations for investigational CGT combination products, with emphasis on FDA, EMA, and Health Canada expectations. The program is designed to equip regulatory, CMC, and technical professionals with actionable strategies to support early- and mid-stage development while minimizing downstream regulatory risk.
Key Areas of Focus
- Global regulatory expectations for investigational CGT combination products
- CMC Module 3 considerations and cross-regional alignment strategies
- Device development: Review of regulatory guidance for device development, including design controls and risk management
- Human factors and usability considerations for CGT delivery systems
- Potency strategies for cell and gene therapy products, including assay design, qualification, and validation
Learning Objectives
Upon completion of this workshop, participants will be able to:
- Interpret key CMC Module 3 expectations for CGT combination products across FDA, EMA, and Health Canada.
- Apply essential regulatory principles for medical devices, including design controls, risk management, and human factors, within CGT development programs.
- Design and justify potency strategies that meet global regulatory expectations for cell and gene therapy products.
Who Should Attend?
- Regulatory Affairs professionals supporting cell and gene therapy or combination product programs.
- CMC and Technical Development professionals contributing to global regulatory submissions.
- Device development and engineering professionals working on CGT delivery systems.
- Scientists responsible for potency assay development and regulatory justification.
- Quality and compliance professionals overseeing combination product requirements.
Audience 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.
Agenda
|
Time ET |
Topic |
|
9:00 – 9:10 am |
Welcome and Workshop Introduction |
|
9:10 – 9:30 am |
Global CMC Module 3 Expectations for CGT Combination Products |
|
9:30 – 10:05 am |
Medical Device Regulatory Principles and Human Factors Considerations |
|
10:05 – 10:35 am |
Potency Strategies for Cell and Gene Therapy Products |
|
10:35 – 11:00 am |
Case Study: Building a Global CMC Strategy for an Investigational Combination Product |
Cancellations and Refunds
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. All cancellation requests must be submitted in writing to [email protected]. Cancellations will receive a full refund minus a 20% administrative fee. RAPS is unable to accept cancellations by phone.
Substitutions
Paid registration substitutions may be accepted with written approval from RAPS for requests received before the start of the event. To transfer a registration, email [email protected] with the event title, name of the original registrant and the contact information for the new attendee.
Proof of Attendance
A certificate of attendance can be downloaded from the RAPS Learning Portal following the event.
Questions
Contact the RAPS Support Center:
Call +1 301 770 2920, ext. 200 (8:30 am–5:30 pm EST, Monday–Friday) or email [email protected].
Speakers
Sai Bhamidipati MS, RAC
