*All times listed are in EDT

8:25 am Chair’s Opening Remarks

NAVIGATING REGULATORY GUIDELINES, COMPLIANCE & PATIENT ENGAGEMENT

8:30 am Virtual: Exploring Regulatory Guidelines & Best Practices for Engaging Patients in Gene Therapy Development

Synopsis

• Exploring the current regulatory guidelines in incorporating patient perspectives in drug development
• Understanding how this applies in the context of genetic medicines
• How patient focused guidelines feeds into other aspects including benefit/risk guidance
• Sharing best practices of patient input at different stages of development

9:00 am Establishing Cross Functional Collaboration to Achieve Meaningful, Appropriate Interactions With Patients

Synopsis

• How can we ensure alignment across the vendors on what ‘good’ patient input looks like?
• Facilitating patient input whilst ensuring compliance
• Examples of patient input on patient recruitment and retention and overall impact

9:30 am Streamlining Gene Therapy Progress: Creating the Bespoke Gene Therapy Consortium (BGTC)

  • PJ Brooks Deputy Director, Officer of Rare Diseases Research, NIH

Synopsis

• Outlining the aims of the BGTC
• Assessing criteria for moving forward diseases to clinical trials and relation to patient communities
• Learnings from cross-collaboration between different gene therapy stakeholders

10:00 am Morning Break & Networking

DESIGNING & IMPLEMENTING MEANINGFUL GENE THERAPY PROGRAMS

11:00 am Building a Strategic Patient Council – Navigating Internal & External Needs

  • Kate Trenam Head of Patient Engagement, Early Research, UCB

Synopsis

• Building out early patient engagement within a global pharmaceutical company for gene therapy
• Developing a council for assets before candidate selection
• Sharing examples from experience at UCB

11:30 am Building a Patient-Centred Clinical Trial Protocol

  • Annie Ganot Founder & VP Patient Advocacy, Solid Biosciences

Synopsis

• Consolidating clinical trial design in conjunction with patient communities
• Ensuring patient safety is paramount and exploring other aspects to prioritise
• Exploring potential outcome measures and how this relates to regulatory guidelines

12:00 pm Lunch & Networking

TACKLING EDUCATION & MISCONCEPTIONS IN GENE THERAPY

1:00 pm Evaluating Strategies to Explain the Concept of Genomic Medicines

  • Edgar Vega Senior Manager, Patient Advocacy , uniQure

Synopsis

• Tackling differences between gene therapies, gene edited gene therapies, and other types of genomic medicine
• Creating resources to effectively explain the concept of gene therapy
• Educating on gene therapy more widely versus for specific diseases in the context of gene therapy

1:30 pm Industry’s Commitment to Patient Education & Community Resources

  • Paula Orandash Patient Engagement Liaison, Community Education Gene Therapy, PTC Therapeutics

Synopsis

• Discover how industry identifies gaps in patient education and delivers programs and resources that are understandable, comprehensive, and relevant
• Learn how to distinguish cultural learning dynamics, identify learning styles, and how to execute the strategy through a series of different assets

2:00 pm Panel Discussion: What Role & Responsibility Do Industry Have to Educate on Genetic Medicines?

  • Kristen Huehbner Associate Director, Global Patient Advocacy & Engagement, BioMarin
  • Edgar Vega Senior Manager, Patient Advocacy , uniQure
  • Paula Orandash Patient Engagement Liaison, Community Education Gene Therapy, PTC Therapeutics

Synopsis

One particular challenge of gene therapies is the way it’s understood by different communities. Ensuring there’s a good understanding is an effort involving multiple stakeholders. This panel will explore industry’s role in this challenge, including:

• How trusting are patient communities of sponsors and the information they are imparting?
• If it’s not appropriate for industry to share information, where can materials be accessed?
• Working towards a community responsibility of education from industry and how it could be achieved

2:30 pm Hearing Directly From Patients With Experience in Clinical Trials

Synopsis

• The decicision-making process; consent and risk-benefit assessment
• Expectations of gene therapy
• Commitment required to take part in a gene therapy trial
• Life before and after gene therapy

3:00 pm Chair’s Closing Remarks

LEARNING FROM PATIENTS: IMPROVING INDUSTRY & PATIENT GROUP RELATIONSHIPS