Pre Conference Workshop Day | Tuesday, June 27 2023

9:00 – 11:00 am
Workshop A

9:00 am Hitting Them With Your Best Shot: How to Determine the Most Promising Vector Designs

  • Josephine Joel Prinicipal Scientist II, Gyroscope Therapeutics
  • John Thompson Vice President, Genomics and Computational Biology, Homology Medicines Inc.
  • Sean Armour Head of Discovery, Spark Therapeutics

Synopsis

The current gene therapy landscape is a mix of novel and well-researched vector designs. Novel designs are a fresh opportunity to tackle challenges but also bring with them regulatory challenges, lack of background literature and the investment required for starting something from scratch. Hence, it is imperative to determine the most promising vector designs to invest time, money, and effort into. Is it wise to keep on

investing in new technologies? Or should we stick to what works? What kind of strategy should you have around development of technologies in this market? These are just some of the questions that will be addressed in this section.

This workshop will cover the following:

• Overview of criteria to evaluate novel and established vector designs

• How to determine the evaluation criteria pertinent to your aims

• Evaluating some novel vectors in the context of the criteria mentioned above

11:30 – 1:30 pm
Workshop B

11:30 am Finding Your Footing: How to Balance the Toxicity, Specificity and Feasibility of Vector Designs

  • Wayne Yount Senior Vice President - Analytical & Formulation Sciences, Kriya Therapeutics

Synopsis

Every vector design faces a multitude of challenges through its pipeline. These range from safety and efficiency to downstream feasibility. Vector profiles for each of these challenges vary widely and it is important to understand limits and thresholds for each one. In order to navigate the gene therapy space, one needs to take important decisions to weigh and prioritise these challenges against each other. This session will draw on the experience of key opinion leaders to review how established players in the industry solve this challenge.

This workshop will cover the following:

• Overview of the various challenges faced during vector design

• Making an informed choice about your data-readouts to prioritise challenges

• Case-study: A pre-clinical pipeline showcase followed by a critiquing session of the same

2:00 – 4:00 pm
Workshop C

2:00 pm Build vs. Buy: Should You Develop a Functionality In-House or License it from Contemporaries

  • Keith R. Lange Vice President, Head of Intellectual Property, Spark Therapeutics
  • Joseph La Barge Chief Executive Officer, Apertura Gene Therapy

Synopsis

The gene therapy industry is exploding with many new players entering the industry every year. As a result, companies are finding it increasingly difficult to navigate the IP space and make informed financial decision around building or licensing technologies. Learn from the experiences of key-opinion leaders in the space to de-risk these decisions earlier.

This workshop will cover the following:

• Navigating the IP space in gene therapy

• Considerations when building or buying a functionality or technology

• How to de-risk build vs. buy decisions based on research data