Conference Day One - Tuesday | October 29, 2024
8:00 am Check-In & Coffee
8:50 am Chair’s Opening Remarks
Reflecting on the Most Pressing Challenges & the Future of the Cell & Gene Therapy Space
9:00 am Industry Leaders Fireside Chat: Insights from Leading Experts on Overcoming Current & Future Regulatory Challenges in the Cell & Gene Therapy Field
Synopsis
- What are the key obstacles challenging the cell and gene space now?
- Where will the cell and gene space be in 5 years, and how will this impact the regulation of these treatments?
- How can service providers assist biopharma in paving the way forward?
Driving Accelerated Approval by Dismantling Criteria for this Pathway to Fast-Track Cell & Gene Therapies Through Pipelines
10:00 am Enlightening the Regulatory Community on Accelerated Approvals to Break Down Criteria & Expectations
Synopsis
- Evaluating the starting point for achieving accelerated approvals to guide companies pursuing this pathway
- Exploring scenarios where accelerated approvals are suitable and unsuitable to define who this would be relevant to
- Outlining how the timeline works for accelerated approvals to aid future planning efforts
10:30 am Morning Break & Speed Networking
Synopsis
As this community unites, this session will provide valuable networking time with your peers to foster new and lasting connections
Finding Solutions to Regulatory Bottlenecks to Advance Gene Therapies Through the Clinic
11:30 am Exploring the Development of Novel Endpoints & Negotiating Endpoint Approval with Regulatory Bodies
Synopsis
- Examining how endpoints are determined by considering starting points, planning, and use of biomarkers
- Discussing the iterative process that must be done to validate the endpoints
- Sharing advice on negotiations with regulatory bodies to facilitate more efficient conversations
12:00 pm Panel Discussion: Navigating Various Assigned Designations like Orphan, Prime, & More to Clarify Criteria & Distinctions
Synopsis
- Breaking down criteria for different platform designations to streamline decisions on the most suitable designation for your therapy
- Emphasizing specific advantages of certain designation allocations to rationalize plans for application
- Tackling challenges in applying for platform designations to streamline the application and approval process
12:45 pm Lunch Break & Networking
1:45 pm IND-Enabling Toxicology and FIH Dose Extrapolation Considerations
Synopsis
- Regulatory expectations for nonclinical package to support AAV-based gene therapy IND
- Investigating immunogenicity and the impact it has on dosing
- FIH Dose Extrapolation Considerations
Finding Solutions to Regulatory Bottlenecks to Advance Allogeneic Cell Therapies & In Vivo Gene Editing Through the Clinic
2:15 pm Differentiating Between Autologous & Allogeneic Clinical Regulation to Spotlight Allogeneic Therapies
Synopsis
- Comparing regulatory requirements for allogeneic versus autologous therapies to highlight the key differences
- Examining the use of platform technology in starting materials, donor screening, and selection for allogeneic therapies
- Delving into the manufacturing process of allogeneic therapies and how this is impacted by policy