With only limited regulatory guidance on gene therapies, many drug manufacturers struggle to develop a strategy for chemistry, manufacturing and controls (CMC) that will be acceptable by regulatory agencies.
The workshop will engage participants with learning exercises to gain a thorough understanding on extrapolating current regulatory guidance on sterile injectable aseptic processing for gene therapies. Detailed examples and case studies of specific drug substance and drug product CMC section content for module 3 of the Common Technical Document (CTD) will be reviewed, keeping lifecycle management in mind. Based on the type of therapeutic modality an manufacturing approach, attendees will learn the advantages of utilizing specific regulatory pathways and tools.
The workshop will also present a consolidation of helpful tips based on approved siRNAs, viral vectors, mRNA and associated delivery systems (e.g., lipid nanoparticles) for regulatory submissions, allowing you to implement this information effectively for your own clinical trial or marketing application.
Topics will cover manufacturing, raw materials, in-process controls, analytical procedures, impurities, specification setting and container closure system information. Key challenges of potency testing and compatibility studies for gene therapies will be priority focus areas.
The workshop will conclude by highlighting risk-based approaches for product lifecycle management, based on ICH Q12 and other associated regulatory guidance, to deal with manufacturing changes likely to occur during different phases of gene therapy development.
Registration Fees & Deadlines
February 1, 2024 – April 15, 2024: Member $285.00 | Nonmember $335.00
April 16, 2024 – May 16, 2024: Member $335.00 | Nonmember $395.00
Learning Objectives
- Develop a CMC regulatory strategy for specific gene therapy modalities utilizing available guidance and industry expertise
- Justify sufficient information in CMC section submissions to assure regulatory reviewers that product quality, safety and efficacy is maintained, utilizing analytical testing, risk assessments, and Quality by Design
- Manage challenges and changes to gene therapy CMC information by utilizing regulatory tools and guidance for effective planning, early communication, and notification
Who Should Attend?
Regulatory CMC Regulatory Affairs Quality Assurance Quality Control professionals
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
| 10:00 – 10:30 am |
Regulatory Submissions and Pathways Overview
|
|
10:30 – 11:15 am
|
Synthetic oligonucleotide gene therapy CMC submissions
|
|
11:15 – 12:00 pm
|
Group Exercise
|
|
12:00 – 12:30 pm
|
Lunch Break
|
|
12:30 – 1:00 pm
|
Viral Vector CMC considerations with submission draft examples
|
|
1:00 – 1:30 pm
|
mRNA CMC considerations with submission draft examples
|
|
1:30 – 1:45 pm
|
Gene therapy product lifecycle management
|
|
1:45 – 2:00 pm
|
Review of the Day: Tying it All Together
|
|
2:00 pm
|
Adjourn
|
Speakers
Janmeet Anant
Mililpore Sigma, Senior Regulatory Consultant
Janmeet Anant, PhD, PMP, RAC serves as a Senior Regulatory Consultant at MilliporeSigma, focused on pharmaceutical manufacturing, specifically working on sterile injectables. Janmeet has been a key member of the Paradigm Change in Manufacturing Operations (PCMO), which is a Quality Risk Management (QRM) team of the Parenteral Drug Association (PDA). In addition, Janmeet served as an Executive Board Member for the Bioprocess Systems Alliance (BPSA, subcommittee Vice-Chair of the American Society of Methanical Engineers - Bioprocessing Equipment (ASME-BPE) standard setting organization, and a member of the Regulatory Governance Team at BioPhorum. With over 20 years of experience, Janmeet has been consulting on CMC submissions for CDMO clients focused on recombinant proteins and gene therapies, leveraging skills from his Project Management Professional (PMP) and Regulatory Affairs Certification (RAC). Janmeet has a Bachelor of Science degree in Chemistry and a PhD in Pharmacology.
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 support@raps.org. 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 support@raps.org 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
RAPS@raps.org