*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 Lumevoq Gene Therapy in LHON


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

10:30 am | Morning Break & Group Networking

Establishing Effective Analytical Tools

11:00 am How Important are the Full to Empty Capsid Ratios?


  • Effectively characterizing full, empty, and partially filled capsids
  • How does measured viral titre relate to other attributes
  • Analysing the actual content of filled viral particles

11:30 am 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

12:00 pm 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

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 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

Building a Streamlined Global Clinical Trial

1:30 pm Choosing a Suitable Comparator in a Rare Disease Trial


  • Regulators require comparators but often placebo controls in rare disease trials are not ethical or practical
  • Exploring the role natural history studies can play
  • Understanding regulatory requirements of suitable comparators

2:00 pm Panel Discussion: 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 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

3:00 pm Chair’s Closing Remarks & End of Summit