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
9:45 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
10:15 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
Focusing on Pre-Clinical Challenges for Cell & Gene Therapies such as IND-Filing & Dosing to Unravel Bottlenecks & Share Experiences
11:15 am Securing IND-Approval by Leveraging Lessons from Common Challenges in IND-Filing to Advance Towards the Clinic
Synopsis
- Discussing a case study to dissect past experiences and common challenges overcome during IND application
- Classifying the pre-clinical models and data required for IND filing to ensure criteria are met
- Establishing achievable and clear goals for progressing toward IND filing to map out next steps
11:45 am Translating Pre-Clinical Doses into Efficacious Human Dosages in the Clinic & Reviewing Recent Dosing Guidance
Synopsis
- Clarifying regulatory expectations for translating pre-clinical doses into efficacious human dosages
- Investigating immunogenicity and the impact it has on dosing
- Reflecting on recent FDA guidance from Project Optimus and its impact on oncology dosing
12:15 pm Lunch Break & Networking
Finding Solutions to Regulatory Bottlenecks to Advance Gene Therapies Through the Clinic
1:15 pm 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
1:45 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
2:30 pm Afternoon Break & Networking
Finding Solutions to Regulatory Bottlenecks to Advance Allogeneic Cell Therapies & In Vivo Gene Editing Through the Clinic
3:30 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
4:00 pm Demystifying the Regulatory Pathways for In Vivo Gene Editing to Advance Pipelines to the Clinic
Synopsis
- Exploring the challenges in clarifying regulatory pathways for in vivo cell and gene editing pipelines
- Investigating what first-in-human data packages should look like and the nuances and risk benefits in patient populations
- Determining clinical follow-up plans that will be required by regulatory bodies