Component Type: Tutorial
CE: ACPE 2.75 Application UAN: 0286-0000-25-504-L04-P; CME 2.75; IACET 2.75; RN 2.75
Pre-registration required and is an additional fee.
*Please note: Short Courses are stand-alone events. Registration for the main conference, forum, etc. are not mandatory. Already registered? Log in to DIA's
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This Short Course will be offered virtually – Join from anywhere! Cell and Gene Therapies (CGTs) are a rapidly expanding area in the pharmaceutical sector, offering the potential to address serious unmet medical needs in many indications. Yet some of the conceptual frameworks in this field differ dramatically from those of traditional pharmaceutical medicines, specifically with regard to CMC, Non-clinical and Clinical requirements and Health Authority expectations. This short course will help those who have pharmaceutical sector experience but are newcomers to the CGT field ‘get into the right frame of mind’ to think about CGT development. Participants will be introduced to requirements for each of the major disciplines for regulatory submissions: CMC, Non-clinical and Clinical. The course will also explain the rationale behind the CMC approach to CGT manufacture and basic CMC regulatory expectations for CGT products, including process and product characterization, comparability and process validation. Biological mechanisms for CGTs will also be discussed, with a focus on how mechanistic understanding can inform the design of in vitro and animal studies to provide non-clinical evidence of safety and efficacy. Key principles for clinical development of CGTs will be presented, with an emphasis on clinical study design elements that are critical for Health Authority evaluation of benefit-risk. Case studies of recent FDA approvals will be showcased to highlight key clinical considerations and changes in the regulatory environment for CGTs. The course features ex-Health Authority Regulators presenters who have a combined 20 years of experience at the FDA and EMA. Receive
$50 off your Global Annual Meeting total purchase by registering for two or more Short Courses. Purchases must be made at the same time to receive the discount. Discounts will be reflected on the last page of the cart. Registered attendees for this virtual Short Course will receive access to the course recording for 2 full months post-course! This allows you to remain flexible with your schedule and not worry if you need to step out momentarily. Have a conflict with the dates of the course, but are interested in the content? Register anyway and you will receive access to the recording!
Who should attend? Professionals involved in or interested in gaining an understanding of the basics of cell and gene therapy development, including regulatory personnel, clinical operations personnel, researchers and investigators, medical writers, and commercial specialists.
Learning Objectives - Demonstrate why cell and gene therapies (CGT) require a different and very specialized regulatory approach compared to small molecules and biologics
- Develop knowledge of CGT-specific considerations and requirements for CMC, Non-Clinical, and Clinical development
- Analyze important regulatory considerations for CGT development programs
