*All Times Listed are in GMT

9:20 am Chair’s Opening Remarks


9:30 am Confirming the Value of Gene Therapies Through Real-World Data – From Accelerated Approval to Proven Long-Term Outcomes


  • Making gene therapies quickly available for patients through accelerated approvals based on limited clinical data
  • Post-approval real-world evidence (RWE) seeks to characterise safety and efficacy parameters in clinical practice and in the long term
  • Using RWE to underline the value proposition and sustained benefit of gene therapy for patients and families

10:00 am Panel Discussion: Payer Perspective on Gene Therapy Pricing & Reimbursement Interactions so far in Europe


  • Learnings from Zynteglo market access approach in Europe, including Germany response
  • Incorporating different perspectives from key European players on how to work with sponsors
  • Where is there room for improvement in the current market access process, and how will this be expected to be implemented?

10:30 am | Morning Break & Group Networking

Establishing Effective Analytical Tools

11:00 am Optimizing Full to Empty Capsid Ratios for a Purer, Higher Yield Product


  • Effectively characterizing for full, empty, and partially filled capsids
  • Exploring ways to consistently measure viral titre
  • How can the ratio of fully to empty be further optimised?

11:30 am Analytics & Characterization of Gene Therapy Products


  • Establishing key analytical assays for ophthalmic gene therapy development
  • Characterizing products – highlighting the importance of quality
  • Learnings from SparingVision’s approach for a successful manufacturing strategy

12:00 pm Key Considerations for Lentiviral Vector Analytical Methods


  • Comprehensive analytics are required by regulatory agencies to ensure quality, safety, and efficacy of gene therapies
  • Characterizing drug products
  • Assessing the challenge of a lack of reference standards and how to approach this in development
  • Defining critical quality attributes for lentiviral and assays to measure them

Aligning Stakeholders for Approval: What Needs to Happen?

11:00 am Exploring the Link Between Innovative Payment Schemes & Real World Evidence (RWE) in Supporting Gene Therapy Value & Approval Processes

  • Paolo Morgese EU Director Market Access & Member Relations, Alliance for Regenerative Medicine (ARM)


  • Setting the context of innovative payment model approaches in Europe and links to RWE
  • Exploring how recent pan-European legislation relates to gene therapy market access
  • How are different European countries approaching market access in practice?

11:30 am Demonstrating Value in a ‘Crowded’ Market: Ensuring Optimal Patient Outcomes

  • Paul Cox VP Global Commercial, Freeline Therapeutics


  • Developing a gene therapy where current standard of care exists, or where other gene therapies are also being developed, poses unique considerations from a commercial perspective
  • Understanding evidence-based patient decisions for choosing gene therapy (or between different gene therapies)
  • Predictability of gene therapy outcomes can be variable, so how does this feed into value demonstration?

12:00 pm Exploring Opportunities to Build Novel Payment Models: Beyond Annuity Payments

  • Michela Gabaldo Head of Translational Project Management & Regulatory Affairs , Fondazione Telethon


  • Exploring different types of value based agreements that could be possible
  • How is Italy approaching alternative payment models?
  • Discussing where innovation is coming from and if it’s being put into practice
  • Bridging market access and patient access

12:30 pm | Lunch & Networking

Making Strategic Manufacturing Decisions

1:30 pm Contract Manufacturing in the Development of New Gene Therapies for Rare Disorders

  • Nicholas Medcalf ISCF Deputy Challenge Director, Medicines Manufacturing, Innovate UK


  • A choice of approaches is possible depending on the business model adopted
  • Design early for manufacture and consider the operability of the process at the proof of concept stage
  • The European CDMO system is healthy and there are things that public sector organisations can do to make it better still

2:00 pm Roundtables: Scaling Up Versus. Scaling Out: Key Considerations


This virtual roundtable session will be the opportunity to discuss the key strategic manufacturing decisions involved in scaling up and scaling out gene therapy manufacturing. Share audio and video and discuss this key topic with peers, including:

  • Preference tends to be to scale up versus scale out to keep processes as similar as possible
  • Where is scale out the viable option?
  • Accommodating for any manufacturing process changes as early on as possible

Building a Streamlined Global Clinical Trial

1:30 pm Lumevoq Gene Therapy in LHON


  • LHON disease
  • Overcoming mitochondrial mutations with gene therapy
  • Lumevoq regulatory pathways in Europe and the US

2:00 pm Defining Clinical Endpoints for Rare Diseases


  • Incorporating patient perspectives to define a suitable primary endpoint
  • Assessing measurability as a factor in endpoint choice
  • Navigating challenges of clinical trial design with a small patient population

2:30 pm | Afternoon Break & Networking

3:00 pm Engineering Lentiviral Vectors for In Vivo Gene Therapy Applications


  • Reviewing efforts to modify lentiviral vectors
  • Reducing immunogenicity and improving cell and organ-specific targeting
  • Exploring recent preclinical work which highlights the potential of modified lentiviral vectors

3:30 pm Promising Next Generation Vector Technology to Improve Gene Therapy Safety


  • Improved gene therapy vectors have the potential to lower doses and therefore toxicity risks
  • Highlighting key novel vector approaches with lower immunogenicity and higher specificity
  • How translatable are these vectors to current manufacturing approaches?

4:00 pm Chair’s Closing Remarks & End of Summit