Pre-Conference Workshop Day

 Workshop A: Exploring Commitment to Gene Therapy Trials & Follow-Ups: Key Considerations

Tuesday, June 7 | 9.00am - 12.00pm

One of the several unique aspects of gene therapies as a therapeutic is the requirement for many years of follow-up in clinical trials, commonly for 15 years – whereas the gene therapy is only administered once. Given this high level of commitment, it’s crucial to ensure that the follow-up process is as easy to manage as possible and reduces burden on patients, ensuring high patient retention, adequate safety monitoring, and sufficient data to submit to regulators for approval. Crucial to this process is patient understanding of what commitment to a gene therapy trial will involve.

Attend this workshop to learn about:

• Understanding potential models for gene therapy trials
• Reducing patient burden over extensive follow-ups
• Detailing what the trial process will entail to make informed decisions
• Exploring examples of remote assessment
• Communicating effectively with patients on engagement with the clinical trial
• Setting expectations with trial participants and families

Workshop Leaders:


Vivian Fernandez 
Senior Director, Patient

Untitled design (18)

Sarah Friedhoff
Associate Director, Patient
Advocacy & Clinical Affairs
Abeona Therapeutics

Jodi Wolff

Jodi Wolff
Senior Director, Patient

Workshop B: Engaging Patient Communities in Trial Design, Recruitment & Drug Development 

Tuesday, June 7 | 13.00pm - 16.00pm

Several gene therapies currently in development are targeting rare and ultrarare diseases. Given the significant challenges associated with developing trials with small patient numbers compounded with the complexity of genetic therapies, the role of patient groups in finding patients and sharing vital input on clinical trial recruitment is even more important to utilize effectively

Attend this workshop to learn about:

• Collaborating effectively with patient groups on finding patients
• Making use of patient input to streamline recruitment processes, and understand what patients need and want to make an informed decision on enrolment
• Exploring the role of industry in progressing genetic testing and newborn screening
• Developing materials in association with patient groups
• Working alongside clinicians whilst remaining compliant

Workshop Leader:


Craig Martin 
Global Genes