*All Times Listed are in GMT

7:50 am Chair’s Opening Remarks


8:00 am Keynote: Effectively Launching a Gene Therapy in Europe: Key Learnings


  • Understanding the complex processes involved in bringing a gene therapy from phase I trials through to market
  • Exploring the unique considerations for product launching in Europe
  • Navigating the pathway to approval and highlighting potential obstacles that must be overcome

8:30 am Gene Therapy Landscape Review of 2020


  • Therapy landscape – unique features of assets making it to phase III
  • Trial landscape – trial status and progress
  • COVID-19 gene therapy landscape – what do the results look like?
  • Where is gene therapy heading? A look into what the future may hold for gene therapy and how we can overcome some of challenges met in the clinic

9:30 am Speaking position reserved for BIA Separations

10:00 am Morning Refreshments & Speed Networking


Making Strategic Manufacturing Decisions for Gene Therapies


Exploring the European Regulatory Landscape


Understanding the Payer Perspective to
Gene Therapy Products

11:30 am Utilising Contract Development and Manufacturing Organisation (CDMO) to Supply Clinical and Commercial Autologous Cell and Gene Therapies
• Manufacturing drug product in-house versus CDMO
• Key considerations for selecting a CDMO
• Establishing and maintaining a good working relationship with CDMOs
Lee Markwick, Associate Director, External Development & Manufacturing, Orchard Therapeutics

11:30 am Outlining Key Regulatory Pathway Differences in the US & Europe - Learnings from the development of Lumevoq Gene Therapy in LHON subjects
• Comparing and contrasting EMA & FDA regulatory pathways
• Working effectively with regulators to meet their requirements
• Outlining the advantages of more common guidances between different regulatory agencies
Magali Taiel, Chief Medical Officer, Gensight Biologics

11:30 am The Evaluation of Cell & Gene Therapies: Perspective from National Institute for Health and Care Excellence (NICE)
NICE regenerative medicine study
• Outlining current guidance from NICE on cell and gene therapies
• Highlighting case studies
• NICE services to support appropriate evidence development
Nick Crabb, Programme Director, Scientific Affairs, National Institute for Health and Care Excellence (NICE)

12:00 pm Speaking position reserved for Brammer Bio

12:00pm Speaking position reserved for The Jackson Laboratory

12:00 pm Preparing for a New Treatment Paradigm – Supporting Access to Gene Therapies in Europe

Talk details to be confirmed

Jim Lennertz, Senior Vice President, Commercial Operations, EUMEA, BioMarin






12:30 pm Lunch & Networking


Effectively Scaling Up to Meet Gene Therapy Demands 


Optimising Clinical Trial Design For Rare Disorders


Evaluating Potential Pricing Models & Strategies 

2:00 pm Implementing an Effective Scaling Strategy for Gene Therapy
• identify the needs for scaling-up processes
• Anticipate and adjust your development strategy with your scale-up
• Highlighting considerations when working in rare diseases with larger patient populations
Pierre-Albert Colcomb, Business Development Director, Genethon


2:00 pm Considerations for Selection of Novel Endpoints and Appropriate Controls
• Discussing some of the creative manners gene therapy developers have used to optimize clinical trial design
• Collecting the patient and caregiver perspective as a valuable source of information
• Understanding an individual’s trajectory to inform treatment expectations
Samantha Parker, Senior Vice President & Chief Patient Access Officer, Lysogene

2:00 pm Innovative Payment Models: Italian Experience with the First Registered ATMP
• Lessons from recent ATMPs launches In Italy: High value with significant costs; often one-time treatment, with challenge of significant budget impact at administration and effectiveness overtime
• Italian model to balance uncertainties:
+ Payments based on patient outcome
+ Special funding mechanisms
Michela Gabaldo, Head of Alliance Management & Regulatory Affairs, Fondazione Telethon

2:30pm Investigating Technical Advances to Increase Scaling Efficiency
• Increasing efficiency of the upstream scaling process to scale up versus scale out
• Developing industrialised scaling processes
• Highlighting novel transfection methods that are effective at large scales
Speaker TBC

2:30 pm Establishing a Clinical Trial in Europe: Key Considerations
• Understanding operational considerations of running a clinical trial in Europe
• Planning effectively where clinical trial sites should be
• Exploring logistical implications of running a clinical trial at multiple sites
Magali Gibou, Vice President, Regulatory Affairs, Sangamo Therapeutics

2:30 pm The European Health Technology Assessment (HTA) and Reimbursement Landscape for Gene Therapies – Current Status and Future Evolution
• Exploring differences between the European reimbursement system and the US for gene therapies
• Evidence requirements for HTAs in Europe and how this affects overall P&R strategy
• What kind of funding and contracting mechanisms could work in Europe?
Simon Shohet, Head of International Market Access, Amicus Therapeutics

3:00 pm Panel Discussion: Scaling for a Commercial Product: Evaluating Best Approaches and Practices
This panel discussion will address:
• Scaling up from phase I to phase III
• Scaling between different indications
• Making the transition to industrialised processes with increases yield and purity
Hosted by Christina Broomes, Director, Contract Manufacturing, Ultragenyx

3:00 pm Panel Discussion: Overcoming Hurdles Associated with Clinical Trial Design for Rare Diseases
This panel discussion will address:
• Choosing the most suitable comparator
• Utilizing natural history studies effectively
• Using appropriate controls
Hosted by Mindy Leffler, President & Chairman, Casimir

3:00 pm Panel Discussion: Evaluating the Advantages
of Different Launch Locations in Europe
This panel discussion will address:
• Discussing differences in launch strategy depending on launch location
• Highlighting regulatory, cultural, and operational differences that affect launch location decisions
• Is there one country in Europe that should be launched to first?
Hosted by Sven Kili, Principal, Sven Kili Consulting

3:45 pm Afternoon Refreshments & Scientific Poster Session


4:45 pm Panel Discussion: Exploring the Industry Response to COVID-19 & Identifying Key Learnings


It is no secret that the COVID-19 pandemic has been one of the biggest challenges gene therapy companies have faced, affecting progress in multiple areas from early-stage research to patient follow-ups and GMP manufacturing. This panel will bring together industry leaders to discuss how companies have coped with the pandemic’s impact on development and ignite useful discussions on how COVID-19 has changed approaches. Topics to discuss include:

• Impacts on clinical trials and patient follow-ups

• Impacts across manufacturing, including supply chain management

• Responses to the crisis, highlighting effective strategies and actions

• Outlook on the landscape post-crisis – how will approaches change?

5:15 pm Keynote: Leveraging Real-World Evidence (RWE) for Successful Product Launch in Gene Therapy

  • Ramin Farhood Vice President, Head of Global Medical Affairs , AveXis


• Developing RWE objectives and scope that addresses RW data gap and supports clinical value proposition
• Operationalizing a global RWE registry across the world with high-quality and clinically relevant data
• Utilizing RWE for discussion with external stakeholders (i.e. payers, regulators, physicians) – pre and post launch

5:45 pm Chairs’ Closing Remarks

6:00 pm Close of Day One