When you look at the agenda, you will see 3 different session types:

Gene Therapy Medical Affairs Discover
Gene Therapy Medical Affairs Develop
Gene Therapy Medical Affairs Action

Discover Sessions

Offering you the chance to hear the most admired medical affairs professionals from the gene therapy world share the stories and case studies of what they’ve done to solve their more pressing challenges.

Develop Sessions

Providing you with the unique opportunity to have your burning questions answered in a virtual room filled with your industry peers, and move beyond the “what” and the “why” to get to the “how”.

Action Sessions

Designed to help you work with your team to set a clear, bespoke action plan of what you are going to change when you get back to your organization to ensure you see a fast ROI.

8:15 am Online Registration & Virtual Coffee Networking


Get the jump-ahead by kick starting your conference with a virtual networking experience. This is your chance to make those all-important first connections. Grab your morning cup of coffee, sit back and join the conversation.

8:50 am Chairs Opening Remarks


9:00 am Discover: The Critical Role of the Gene Therapy Platform Medical Affairs Team


• Building a medical affairs team focused on the gene therapy platform in rare disease
• Upskilling gene therapy platform capabilities internally
• Ensuring external stakeholders understand the science of gene therapy

9:20 am Discover: Harnessing Relationships with Patient Advocacy Groups to Support Operational Excellence in Gene Therapies

  • Tom Pulles Vice President Medical Affairs, Ultragenyx


• Exploring the impact patient organizations have on trial recruitment, and on programs overall
• Collaborating with patient groups to raise awareness, and understanding existing differences
• Understanding operational considerations and challenges of partnerships with advocacy groups in a European landscape

9:40 am Discover: Role of Medical Affairs in KOL Mapping and Identification A Critical Success Factor for Successful Gene Therapy Launch

  • Peter Mooney Senior Director Medical Affairs, Orchard Therapeutics


• Building your KOL Management Database to identify the right KOLs in each country/market
• Understanding and defining what are your key parameters for a KOL
• Taking the theory into practice

10:00 am Gene Therapy Communications: Avoiding ‘It’s All Greek to Me’


• How can we align internally to effectively communicate externally?
• What can we do to ensure our audiences understand gene therapy?

10:30 am Develop: What Are the Keys to Success to Ensure Effective Collaboration Between Medical Affairs Teams and Both Internal and External Bodies?


This is your opportunity to have the speakers answer your questions!

11:00 am Action: What Are You Now Going to Change About Your Approach to Collaboration or Partnerships With Internal or External Bodies to Accelerate Opportunities For Your Medical Affairs Team in The Future?


11:20 am Virtual Speed Networking & Morning Break


Recreating face-to-face networking in the virtual world. We will pair you up with fellow attendees
so you can get face-to-face time with the brightest minds working in the gene therapy field in
order to establish meaningful business relationships to pursue for the rest of the conference.

11:50 am Discover: Dravet ENGAGE: A Patient Centric Approach in the Clinical Development of the First One Time Gene Therapy for SCN1A+ Dravet Syndrome


• A case study on how to design a clinical development plan with a patient-centric approach
• Discussion on the role of qualitative research insights to inform clinical development
• Generation of evidence of the “patient/caregiver voice” in interactions with regulatory authorities and IRBs/Ethics Committees

12:10 pm Discover: Complementary Medical Affairs Approach to Patient Engagement

  • Angela Tom Medical Director, Orchard Therapeutics


• Meeting the informational needs of patient organizations
• Multi-dimensional patient experience
• Insight informed decision making

12:30 pm Discover: Medical Affairs as Partner to Empower Patients Throughout the Drug Development Process


• Incorporating patient engagement into medical affairs strategic thinking
• Patient centric data generation
• Approaches to removing knowledge barriers for patient access to trials

12:50 pm Develop: What Are the Keys to Success in Developing a Drug Development Process that is Truly Patient Centric


This is your opportunity to have the speakers answer your questions!

1:10 pm Action: What Are You Now Going to Change About Your Approach to Patient Engagement to Successfully Adapt for the Future?

1:30 pm Lunch & Virtual Networking

2:30 pm PANEL DISCUSSION: Engaging Patients Through the Medical Affairs Role: Challenges & Best Practice

  • Jodie Gillon Vice President Patient Advocacy & Clinical Affairs, Abeona Therapeutics
  • Daniel Snyder VP Medical Affairs, Urovant
  • Rafael Escandon Senior Vice President - Medical Affairs, Policy & Patient Engagement , Bridge Bioscience Corp


3:00 pm Medical Affairs and Medical Communications: Considerations for Cell and Gene Therapy Development


  • Medical communication agency services
  • Medical communication initiatives essential to the development of cell and
    gene therapies
  • A medical communication agency’s role in supporting Medical Affairs

3:10 pm Building a Dynamic Global Medical Training Program in Gene Therapy

  • Jennifer Aikin Head of Global Medical Affairs Training, Novartis Gene Therapies


  • Identify the elements of an effective Global Medical Affairs training program
  • Describe how a dynamic training program can support the business priorities for a Medical Affairs organization
  • Outline the unique challenges and opportunities when training on gene therapy for rare diseases

3:30 pm Discover: The shift from rare disease approach to prevalent conditions


  • What best practices from gene therapy in rare disease can transfer to prevalent conditions
  • What are the pitfalls and the opportunities
  •  How Medical Affairs can facilitate the shift

3:50 pm Discover: Review Aligning Medical Affairs and (Real-World) Evidence Generation Strategies – Better Together”


• Explore unique gene therapy real-world evidence generation needs
• Optimize medical affairs resources in support of RWE generation
• Discuss best practice examples and what can go wrong

4:10 pm How to hire top talent in a niche field while balancing your budget and saving time


  • Discussion on how partnering with a specialized contingency firm can help with building out a Medical Affairs division.
  • Insight for managing interview timelines, candidate and company expectations and competitive intelligence while also saving your company money and time.

4:20 pm Develop: What is the Role and Responsibility of Medical Affairs Professionals in Developing Successful and Effective Scientific Communication Strategies

4:50 pm Action: What Will You Now Change About Your Approach to Medical Communication and Education to Better Engage with Stakeholders Within the Gene Therapy Community?


This is your opportunity to have the speakers answer your questions!

5:10 pm Chair’s Closing Remarks