Post Conference Discussion Day

Immunogenicity Discussion Day

Thursday 23rd March 2023

9:20 am Chair’s Opening Remarks

Refining Dosing and Immunosuppression

9:30 am Transferrable Lessons from a Transplant Surgeon

  • Rakesh Sindhi Co-Director, Pediatric Transplantation, Children’s Hospital of Pittsburgh of UPMC

Synopsis

A dive into immunosuppression regiments, their risks, and transferrable lessons for the gene therapy space

What might a test for gene therapy immune rejection represent as a companion diagnostic tool?

A dive into the regulatory and reimbursement perspectives of such test

10:00 am Exploring Novel Immunosuppression Regimens

  • Julie jordan Senior Vice President, Head Of Clinical Development & Operations, Homology Medicines Inc.

Synopsis

  • Exploring the latest understanding of humoral immune responses to gene therapy
  • Effective strategies for restricting CD8 T cell response to AAV vectors
  • Analysing multi-faceted immunosuppressive cocktail regimes for effectively modulating the immune response

10:00 am Morning Refreshments

Navigating Immunogenicity Into and Through the Clinic

11:00 am Minimizing the Immune Response: Overcoming the Hurdles to Successful AAV Gene Therapies

Synopsis

  • Understanding the complex mixture of innate, humoral and cellular immunogenicity concerns with AAV
  • Exploring the state of play between ‘one and done’ and redosing strategies
  • Combatting high dose AAV toxicities with new strategies

11:30 am Mastermind: Navigating Seamlessly Through the Clinic

Synopsis

This mastermind session gives different groups the same set of questions to answer on designing clinical trials to support expedited approvals of gene therapies, exploring in-depth the below considerations. Following this, insights from different groups will be collated into a single document and circulated to the entire group.

Topics to be discussed include:

  • Effectively navigating temporary holds: how can we take a collaborative with the FDA to amend protocols and swiftly lift a hold?
  • Striking a balance between efficacy and safety when determining dosing in clinical trials to maximise chances of regulatory success
  • Strategies for risk mitigation if safety signals are encountered during clinical trials
  • Comparing the dosing regimens approaches used for systemic vs local gene therapy administration

12:30 pm Lunch & Networking

Demonstrating Product Safety to the Regulators

1:30 pm Panel Discussion: Regulatory Addressing of Ongoing Complex Safety Concerns

Synopsis

  • An overview of the most common safety concerns flagged by CBER
  • Leveraging early CBER interaction to set up for success at the pre-IND stage
  • Strategies the FDA are looking for to mitigate against AAV vector safety risks
  • Top tips for demonstrating product safety with regulatory filings
  • Advocating for different safety monitoring pathways where antibody responses confer no functional side effects

2:15 pm Improving Analytical Methods for Determining AAV Full-Empty Capsid Ratios

Synopsis

  • A comparison of current available techniques for quantifying capsid ratios.
  • An overview of emerging novel analytical tools: where has the tech taken us to?
  • Assay qualification and validation for QC-friendly orthogonal methods.

2:45 pm Chair’s Closing Remarks

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