PRE-CONFERENCE WORKSHOP DAY
Pre-Conference Workshops | Tuesday 6 June, 2023
9:00 – 12:00 pmWorkshop A
Strategizing to Gain EMA Regulatory Approval & Aligning Regulatory and Payer Expectations
Synopsis
The European market is a notoriously tricky area to navigate for gene therapy products, and there is a significant disconnect between what regulators require for a product to gain approval, and what payers require from a product.
This workshop will provide a discussion-based deep-dive into the best strategies to build adequately robust data packages for European regulatory approval, and how to please payers to help save time and money in developing gene therapies for neurological disorders.
This session will include:
- Hearing about the requirements to gain regulatory approval in Europe for gene therapies for neurological disorders
- Discussing how to best prove efficacy and safety to please payers in Europe
- Debating how to better align the data requirements for regulatory approval and payer expectations for neurological gene therapies
1:00 – 4:00 pmWorkshop B
Improving Biodistribution Studies for Neurological Gene Therapies
Synopsis
Understanding biodistribution in gene therapies for the CNS is holding back many therapies from regulatory approval and commercialisation so presents itself as an area in dire need for better understanding.
In this workshop session, join experts in bioanalytics and biodistribution to uncover recent bioanalytical advancements in animal models to build sufficient biodistribution data to apply within your own development for your gene therapy neurological products.
This session will include:
- Improving understandings how to undertake biodistribution studies on mice, rat, or non-human primate models for CNS gene therapies
- Advancing the analytical methods used for preclinical biodistribution assessments in Neurological disorders
- Improving access to non-human primate models as the best template for biodistribution assessments in Europe