7:00 am Registration & Breakfast Briefing Hosted by Project Farma


7:50 am Chair’s Opening Remarks

Moving Towards Post-Approval Commercialization: Where is the Field Going?

8:00 am Generating Post-Approval Real World Evidence (RWE) to Support Overall Value Proposition


  • Comprehensive RWE data collection can support pricing decisions, particularly on outcome-based agreements
  • More long-term data is needed to back up overall gene therapy safety and effectiveness
  • Exploring the best tools and strategies to collect RWE

8:30 am Supply Chain Stability vs Instability in 2022 and Beyond


  • What it takes for successful material management and execution in a Covid Era
  • In the face of uncertainty, how to accelerate timelines to manufacturing by up to 70% w/high quality GMP plasmid manufacturing

9:00 am Developing a Differentiated Gene Therapy for Sickle Cell Disease: Key Considerations


  • Aligning with regulatory requirements for quality of gene therapies, across CMC & clinical development
  • Standing out from both a safety and efficacy perspective
  • Developing therapies in a ‘crowded’ market – with respect to gene therapies in development and existing standards of care

9:30 am Analytical Testing Toolbox for AAV Characterization and Lot Release: Selected Application of Biophysical, HPLC, Immunoassay, and Mass Spectrometry Methods

  • Mark Jones Site Director Biologics , Charles River


  • Intended Use of AAV test methods for characterization, identity, product and process impurities, content and potency
  • Identity test method options
  • HPLC and Mass Spectrometry-based impurity methods: PEI, Triton-X, Host Cell Proteins
  • ELISA for impurities including HCP and AAVx
  • Content assays for excipients, poloxamer, etc.
  • Biophysical methods including AUC, SEC-MALLS
  • Pathways from initial Characterization through Method Development into Qualification and ICH Validation

10:00 am Morning Break & Refreshments

12:30 pm Lunch Briefing Hosted by Resilience


High-Throughput Platforms for Gene Therapy Programs

  • Future proofing your gene therapy
  • Forward thinking in process development including an introduction to new platforms and technology modules
  • Fit for purpose design
  • High throughput process and analytical development concepts
  • How partnering with Resilience will change the way you consider service providers and industry partners

3:00 pm Networking & Afternoon Refreshments

Integrating the Patient Voice Across Every Stage of Gene Therapy Development

3:45 pm Accelerating Gene Therapies for Rare Disorders Utilizing Real-World Evidence

  • Nancy Yu Co-Founder & CEO, All Stripes Research


  • Establishing more appropriate stratification of patient cohorts
  • How a clinical development strategy aligns with patient journey and burden of illness
  • Ensuring better alignment with regulatory agencies on outcome measures for trial design

4:15 pm Incorporating Patient Perspectives from the Very Beginning of Development

  • Jennifer Helfer Senior Director, Patient Advocacy & Engagement , Encoded Therapeutics


  • Including patient advocacy alongside the R&D function and ensuring cross-functional collaboration
  • Managing expectations between different stakeholders and clarifying knowns and unknowns
  • Developing tools to engage with the patient community early on – what are the key learnings?

4:45 pm Channelling Patient Input in Clinical Development: Key Considerations

  • Jessica Riviere VP, Global Patient Advocacy & Patient Engagement, Ultragenyx


  • Engaging with patient communities and care partners to ensure their perspectives drive internal decision making in key areas of clinical development through commercialization
  • Balancing pro-active versus reactive engagement with patient communities regarding clinical trials
  • Partnering with patient communities to publish patient data to advance the understanding of a disease

5:15 pm Gaining Insights into the Patient & Caregiver Perspective: Sharing Experience Engaging with Gene Therapy


  • Understanding the unmet needs for patients and caregivers with respect to gene therapy
  • Understanding and alleviating the patient and caregiver burden?
  • Identifying where sponsors have a positive meaningful impact on patient and caregiver burden

5:45 pm PANEL DISCUSSION: How Prepared is the Industry for More Gene Therapy Approvals on the Horizon?


As more gene therapy companies are entering or are in late-stage phase III trials, the industry need to prepare to navigate the challenges this will bring, including demands on manufacturing capacity, coming to agreements with payers, and ensuring patients are able to access these therapies. This panel will discuss:

  • Current payer perspectives into gene therapy pricing and reimbursement
  • Building out internal manufacturing capacity for commercial products
  • Exploring the concept of centres of excellence versus multi-centre gene therapy

6:15 pm Chair’s Closing Remarks