8:25 am Chair’s Opening Remarks


8:30 am Navigating End-to-End Patient Input: Gene Therapy Case Studies

  • Amy Fisher Head of Patient Advocacy, Spark Therapeutics


• Determining key milestones of patient input across clinical development
• When were patients initially engaged in the process and was this the optimal set-up?
• What are the key learnings to take away for future therapies?

9:00 am Unique Characteristics of Gene Therapy & Their Ethical Implications For Clinical Trial Participation & Post-Approval Access


  • Exploring the ethical considerations relevant to gene therapy in research and clinical practice
  • Specific ethical issues relevant to conducting gene therapy research in children
  • Navigating the balance between gene therapy progress and meeting needs
    of patients, caregivers, and patient communities

9:30 am Panel Discussion: What Do Patients Want & Need from Industry?

  • Amy Fisher Head of Patient Advocacy, Spark Therapeutics
  • Pat Furlong Founding President & CEO Parent Project Muscular Dystrophy, Parent Project Muscular Dystrophy
  • Tom Croce VP, Patient Advocacy, bluebird bio


For any relationship between patients and industry to work, it’s well established that the patient voice needs to be heard. However, implementing this and incorporating that voice into gene therapy development is not always actioned effectively.
This panel will discuss:
• Fostering collaboration between patient groups and industry
• Understanding what patients are looking for and how industry can provide for those needs effectively
• Sharing examples of effective and less effective collaboration to streamline future efforts
• What’s different about interactions on gene therapy compared to other modalities?

10:00 am Morning Refreshments & Structured Networking


Our structured 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.


11:00 am Setting & Managing Expectations of Gene Therapy Within The Patient Community

  • Jennifer Helfer Head, Patient Advocacy & Engagement, Encoded Therapeutics


• Educate along the entire research, development, and commercialization continuum
• Create a two-way dialog
• Transparency and the words we use matter
• Don’t go it alone, work with Patient Advocacy Organizations and others

11:30 am Explaining the Nuances of Gene Editing & Preparing For First-in-Human Trials

  • Jordanna Mora Senior Director, Patient Advocacy & Centricity, Beam Therapeutics


• Several transformative medicines are currently in development so understanding the differences between them is key
• Working with patient communities to develop information that is accessible, understandable, and relevant
• Optimizing the patient experience for first-in-human trial using base editing

12:00 pm Roundtables: Managing Expectations at Different Stages of Gene Therapy Development


One of the biggest challenges for genetic medicines in particular is effectively managing expectations of the therapy, from the perspectives of different stakeholders, functions, and at each stage of gene therapy development. This roundtable session will delve into some of those key milestones in development to be able to share best practice and examples of effective managing expectations, including:


12:30 pm Lunch & Networking


1:30 pm Helping Patients Understand Gene Therapy: Process & Decision Points

  • Tom Croce VP, Patient Advocacy, bluebird bio


• How patients consider gene therapy
• Strategies for compliant, patient-focused education on gene therapy process and support services
• Resources available that facilitate patients’ informed decision making

2:00 pm Building a Patient-Driven Informed Consent Process for a Gene Therapy

  • Pat Furlong Founding President & CEO Parent Project Muscular Dystrophy, Parent Project Muscular Dystrophy


• Ensuring that the informed consent process has been built using patient input
• Understanding the power of language and how we use it in the context of gene therapy, including expectations and assumptions
• How can we be confident that consent is fully informed?

2:30 pm Mastermind: The (Mis)conception of Gene Therapies as a Cure


One of the biggest misconceptions in the genetic medicines field is the idea that it is a cure. When the word ‘cure’ itself means different things to different people, one of the biggest challenges patient advocacy and engagement functions face when communicating on gene therapies is what they are, and what they are not. This is then compounded by different outcomes in different disease areas, different types of genetic medicines, and awareness in different patient communities.

This mastermind will give each group the same set of questions surrounding this idea, and then answers will be collated at the end of the session and distributed to all attendees post-conference to gain insights into current perspectives in the field, and suggestions and ideas of relevant actions we can take as a collective to navigate misconceptions in gene therapy.

Topics to cover include:
• What do people think of when we use the word ‘cure’?
• How can there be a ‘best practice’ to describe what gene therapies are, and what they are not?
• What are some of the other misconceptions people have about gene therapy?
• How can we tackle misconceptions in-house between different departments?
• Any examples of strategies and tools to effectively explain the concept of gene therapies to share with the group?

3:00 pm Afternoon Refreshments & Networking

3:30 pm Aligning Internally to Establish Early Patient Engagement Processes

  • Wendy Borsari Senior Manager, Patient Advocacy, Tenaya Therapeutics


• Building out a patient advocacy function early on in gene therapy development
• Sharing examples of strategies to align on this internally, including patient visits to the lab
• Working with R&D functions & manufacturing on effective patient input & understanding of the unique aspects of gene therapy drug development


4:00 pm Exploring Opportunities to Establish a Pan-Therapeutic Strategy For Gene Therapy


• Examine the need to develop a global, pan-therapeutic strategy to support the unique aspects of gene therapies
• Discuss opportunities and best practices to glean patient advocate and community member insights on gene therapy
• Share engagement and collaboration models for collaborating with rare disease communities to build consensus and prioritize and address unmet needs

4:30 pm Building Trust With Patient Groups: Case Study in Sickle Cell Disease

  • Sara Davis Senior Director, Patient Advocacy & Government Affairs, CRISPR Therapeutics


• Working with different patient groups on a state-by-state basis, and exploring different outreach methods
• Bringing patients to speak to the wider company and understand their perspectives
• Bringing these perspectives through to clinical trial design and implementation

5:00 pm Fireside Chat: Partnering With the Patient Community to Support Access to Gene Therapies


• Understanding patient concerns with respect to ease of access to gene therapies
• Bringing the patient voice into novel approaches to support access (i.e. financing models)
• Partnering with the patient community to shape the policy environment to ensure patient access
• What role do industry have in sharing and conveying this information?

5:30 pm Chair’s Closing Remarks