8:55 am Chair’s Opening Remarks


9:00 am A 101 in Developing Successful Educational Pieces

  • Alison Kujawski Senior Outreach Manager, American Society of Cell & Gene Therapy


• How can we develop patient materials to be conducive to a holistic knowledge of a gene therapy product and the trial process?

• The importance of developing a widely varied toolkit of materials to provide information in an accessible format

• Effectively collaborating with patient groups and experts to collate these 

9:30 am Gene Therapy Education & Decision Making From a Patient/Patient Organization Perspective


• Addressing key patient concerns and uncertainties

• Developing a comprehensive educational approach

• Ensuring all decisions made by patients are well informed

10:00 am Tackling a Field of Complicated Jargon


• Strategies to strike a balance between too much and too little information, tailored to different health literacy requirements

• Communication tools available for simplifying complex terminology

• How can we test patients’ understanding of the benefits and potential risks a gene therapy holds?

10:30 am Morning Refreshments & Speed Networking


Our speed networking is the ideal opportunity to get face-to-face time with colleagues and peers who are passionate about incorporating the patient voice into genetic therapy development. Introduce yourself to the attendees that you would like to have more in-depth conversations with, explore common challenges, and establish meaningful relationships to pursue for the rest of the conference and beyond.


11:30 am Tools & Challenges in Collecting Data to Landscape the Patient Experience


• How to partner with patient advocacy organizations to capture the full, multiphenotypic lived experience of patients

• Generating regulatory-grade data from natural history studies

• Navigating the challenge of privacy issues: who holds the data and how can it be shared?

• Compliant ways to support the patient community in patient focused drug development and listening sessions

12:00 pm Breaking Down Silos and Creating Sustainable Models for Data Collection and Sharing in Gene Therapy

  • Craig Martin President & Chief Engagement Officer, Rithm Health


  • Gene therapy offers potentially transformative impacts for patients, as well as unprecedented challenges to industry and health systems in building/sustaining long-term relationships with patient communities  
  • How can we design and implement patient-focused data collection strategies to streamline the path from disease discovery to gene therapies, and incentivize and sustain engagement and data integrity post-treatment?

12:30 pm Leveraging Real-World Evidence for Long-Term Sustainability

  • Nicole Boice Founder & Chief Mission Officer, Global Genes


• How can we design holistic RWE strategies, leveraging a pool of varied sources across a varied mix of patient types

• Effectively incorporating patient reported outcomes into a registry

• How to reduce patient burden to maximize the length of follow-up

1:00 pm Lunch & Networking


2:00 pm Roundtable Discussion: Setting Up a Team to Maintain the Patient Voice Throughout Development

  • Kristen Huehbner Associate Director, Global Patient Advocacy, BioMarin
  • Thomas Bols Head of Government Affairs & Public Policy, PTC Therapeutics
  • Daniel Leonard Senior Director of Global Patient Advocacy, uniQure


• As a gene therapy progresses through the pipeline, what role does a patient engagement department have in engaging with clinical, commercial, market access and regulatory?

• What does good teamwork look like from a patient engagement perspective?

• Building a framework to ensure the patient voice is never lost throughout the development process

• Ensuring gene therapy development begins with patient community input

3:00 pm Deploying a Consistent & Systematic Approach to Patient Engagement for Gene Therapies


• Understanding the importance of a consistent and value-driven approach

• Meeting expectations of patient-focused drug development

• Defining the priority areas of patient engagement for gene therapy

3:30 pm Afternoon Refreshments & Networking


4:00 pm Creating an Open Dialogue in the Preclinical Phase

  • Jessica Riviere Vice President - Patient Advocacy & Patient Engagement, Ultragenyx


• Understanding the importance of creating an early two-way dialogue before clinical development

• The importance of industry working with patient advocacy groups to understand the disease before designing a clinical trial

4:30 pm Navigating Patient Communication Challenges After a Clinical Hold Imposition


  • Understanding the patient impact associated with clinical holds
  •  Strategies for communicating this news to patient communities where information is potentially too sensitive to share
  • Ensuring industry pre-mortems for this occasion: how can we best prepare patients for this possibility?

5:00 pm Conveying to Patients the Unknowns

  • Brendan Hayes Director of Education, Innovative Therapies, National Hemophilia Foundation


• Exploring clearly with patients the unknown unknowns of gene therapies

• In light of this, how do we discuss with patients to find out the best time for dosing them in a trial?

• Industry’s role in effectively conveying predictability and durability of response, as well as short-term and long-term safety risks

5:30 pm Chair’s Closing Remarks

5:40 pm End of Day One