*All times listed are in EST

WELCOME TO GENE THERAPY PATIENT ENGAGEMENT

7:45 am Online Registration

8:20 am Chair’s Opening Remarks

EVALUATING THE CURRENT PATIENT-CENTRIC VALUES IN THE GENE THERAPY COMMUNITY

8:30 am Setting the Scene: Progress of Gene Therapy so Far in the Context of Patient Centricity

  • Jill Dolgin Executive Director, Global Patient Advocacy, AGTC

Synopsis

  • Patients are central to any drug development process – what makes gene therapy unique in terms of incorporating the patient voice effectively?
  • Understanding the increased industry presence particularly within rare disease, and how this has altered stakeholder perspectives and priorities
  • Achieving success (or failure) of gene therapy programs is impacted by how effectively patient values are both heard and actioned

9:00 am Patient Advocacy and Gene Therapy: What’s Different?

  • Tom Croce Vice President, Patient Advocacy, bluebird bio

Synopsis

  • Understanding how to ensure patient advocacy and patient engagement reflect the unique challenges of gene therapy
  •  Identifying how to generate actionable insights along the development and commercialization continuum
  • Spotlighting internal and external partnerships that tap patients and caregivers’ knowledge, experience and preferences
  • Examining how companies have met the challenge of gene therapy-related patient advocacy and patient engagement

9:30 am PANEL DISCUSSION: Bringing all Stakeholders in Alignment to Work Towards a Common Goal

  • Tiffany Cook Senior Director, CureDuchenne Cares
  • Nicolas Garnier Director of Patient Advocacy, Global Product Development, Pfizer
  • Tom Croce Vice President, Patient Advocacy, bluebird bio

Synopsis

  • Understanding who the stakeholders in gene therapy development are in the context of incorporating patient views
  • What does ‘good’ collaboration look like between sponsors, patient advocacy groups, and patients themselves?
  • Strategies to understand the needs and concerns of stakeholders, and ways these can realistically be addressed and
    solved

10:00 am Virtual Speed Networking

Synopsis

Grab a cup of coffee from the comfort of your own kitchen and jump straight into this exclusive virtual speed networking! This session is the ideal opportunity to meet face-to-face (albeit 2D faces) with many of the brightest minds in the industry to establish meaningful business relationships.

EXPLORING EFFECTIVE STRATEGIES IN PATIENT IDENTIFICATION & ENROLMENT

11:00 am Collaborating with Patient Advocacy Groups on Resources to Identify Patients

  • Tom Pulles Vice President, Head of Medical Affairs & Patient Advocacy, EMEA, Ultragenyx

Synopsis

  • Making the most of existing natural history studies & patient registries
  • Building out resources to find patients
  • Strengthening patient and industry collaboration is key, particularly where the
    space is ‘crowded’

11:30 am Exploring Several Avenues to Maximize Patient Outreach

  • Kyle Bryant Spokesperson, Friedreich’s Ataxia Research Alliance (FARA)

Synopsis

  • Identifying where social media can play a potential role in raising awareness
  • Does presence at patient meetings make a positive impact on the patient
    community?
  • Understanding the importance of sponsor/clinician rapport to provide
    patients with the most relevant information

12:00 pm Lunch & Networking

1:30 pm Developing a Newborn Screening Toolkit for Adrenoleukodystrophy: Key Learnings

  • Emily Fields Associate Director, Patient Advocacy, bluebird bio

Synopsis

  • Understanding the importance of newborn screening in the context of gene therapy
  • Collaborating with patient communities on development of an advocacy toolkit specifically focused on newborn screening
  • Exploring what went well, but also what improvements could be made to the process for future projects given what is known now

2:00 pm Improving Diversity in Clinical Trials to More Accurately Reflect the Patient Community

Synopsis

  • Building trust more effectively between sponsors and patient communities
  • Investigating the reasoning behind eligibility criteria and whether those limitations are necessary
  • Engaging with community partners to better understand the patient community

2:30 pm Afternoon Break & Networking

DISCUSSING APPROACHES TO MANAGE EXPECTATIONS ACROSS STAKEHOLDERS

3:00 pm Communicating Across Functional Departments on Patient Centricity Beyond the Patient Advocacy Team

  • Emily McGinnis Chief Patient Officer & Head of Government Affairs, Taysha Gene Therapies

Synopsis

  • Exploring ways to ensure departments who are not necessarily patient-facing are able to maintain a patient-centric approach
  • Collaborating with different teams to ensure the patient voice is heard for every function, including manufacturing, clinical development, medical affairs
    & market access
  •  Highlighting examples of internal alignment on patient engagement, and identifying where there is room for improvement

3:30 pm PANEL DISCUSSION: Hearing the Needs of the Patient Community: What do Patients Want to See from Industry?

  • Susan Walther Director of Patient Engagement, Friedreich’s Ataxia Research Alliance (FARA)
  • Allyson Berent Chief Scientific Officer, FAST

Synopsis

  • The problem with positioning gene therapy as a cure
  • How can we hear the perspectives of the everyday patient?
  • Listening to patients and making decisions based on their input are two different things. How can we close that gap?

4:00 pm Chair’s Closing Remarks

4:10 pm Close of Day One