Pre-Conference Workshops

Morning Workshops

WORKSHOP A: 9am-12pm GMT

Demonstrating Comparability to Meet Regulatory Requirements

As more gene therapies are progressing through the clinic, the ability to understand and implement comparability studies are more important than ever. By drawing on experience in biologics and building on existing knowledge of gene therapies, this workshop will provide a platform to explore comparability and give you meaningful insights to take back to your own gene therapy programs.

Attendees will discuss:

• Establishing clinical and commercial comparability
• Complications associated with changing platforms
• Discussing analytical methods to assess comparability effectively
• Regulatory requirements in the context of comparability

Workshop Leaders:

Eduard Ayuso, Head of Innovative Vectorology and Scientific, Director of the Translational Vector Core, University of Nantes
Claire Davies, Associate Vice President, Sanofi
Andrea Challand, Group Lead, Technical Regulatory Affairs, Roche

WORKSHOP B: 9am-12pm GMT

Accelerating the Transition from Early Stage Research to Clinical Trials

Gene therapy offers transformative treatment for patients who often have little or no other options. Therefore, the ability to close the gap between research in labs and entering phase I trials is paramount to improving patient lives. By translating early promise into clinical success through successful partnerships, gene therapies with meaningful impacts can reach those patients more quickly and efficiently.

Attendees will discuss:

• Patient groups and labs - optimum meaningful partnership
• Improving the efficiency of pre-clinical studies
• Extrapolating effectively to clinical trials

Workshop Leader:

Gilles Atlan, Vice President, NF2 BioSolutions

WORKSHOP C: 9am-12pm GMT

Exploring the Value of Gene Therapies for Reimbursement

Understanding the real value of gene therapies is vital to any conversation around pricing and reimbursement decisions. By utilizing health economics and outcomes research to illustrate value to different stakeholders, including patients, payers and regulators, this information can then be utilized to develop different potential models for reimbursement that are based on evidence. This workshop will explore the challenges of gene therapy reimbursement, the role that value can play in influencing product decisions and developing models that reflect this approach.

Attendees will discuss:

• Understanding the value of health economics and outcomes research in the gene therapy space
• Using real-world data to assess unmet needs in the gene therapy landscape
• Developing economic models to understand product value
• Deciding on reimbursement strategies based on the available data

Workshop Leader:

Grace Hampson, Associate Director, Office of Health Economics

Afternoon Workshops


Establishing and Maintaining a Good Working Relationship with Contract Manufacturing Services

Transitioning from small-scale academic labs to large-scale commercial manufacturing is becoming more commonplace as the gene therapy field continues to grow. Therefore, facilitating successful relationships with contract manufacturers who are providing these capabilities is essential for anyone looking to bring a gene therapy through clinical trials and into market as a commercial product.

Attendees will discuss:

• Understanding the criteria to use to assess suitability of a partnering organization
• Establishing mutual priorities for greater clarity across companies
• Managing day-to-day interactions to support overall company manufacturing aims

Workshop Leaders:

Christina Broomes, Director, Contract Manufacturing, Ultragenyx
Andreas Kouri, Vice President, Global Supply Chain, bluebird bio


Exploring Next Generation Technology for Effective Targeting & Reduced Immune Response

Gene therapy delivery is continuing to evolve to increase safety and efficiency of administration. Immune response to vectors continues to be a challenge for several reasons, including eligibility of patients, safety, and the potential tore-dose. Therefore, investigating novel technologies which
look to solve some of these challenges will be paramount to developing a more robust, applicable, and safe gene therapy.

Attendees will discuss:

• Discussing main challenges immunogenicity presents for successful gene therapy administration
• Exploring the latest advances in gene therapy delivery to mitigate or evade immune response
• More efficient targeting to cells

Workshop Leader:

Kei Kishimoto, Chief Scientific Officer, Selecta Biosciences


Evaluating the Importance of Product Differentiation in a Growing Market

As more gene therapies progress through the clinic and to approval, the ability to differentiate your gene therapy in this space will be key to long-term success, whether in terms of presenting data to regulators, or framing your product to patients. Establishing a clear profile for your product and presenting relevant information to stakeholders in an accessible way will set you up for success when transforming your clinical candidates into a commercial reality.

Attendees will discuss:

• Developing strategies to showcase unique product properties
• Creating a suitable Target Product Profile (TPP) and what data this should be based on
• Framing your product to patients, payers and regulators

Workshop Leader:

Sven Kili,  Principal, Sven Kili Consulting