Advancing the Frontiers of Gene Therapy and Rare Diseases. Day 1- Lessons learnt in Gene Therapy & Rare Disease Development

Tuesday 10 May 2022

This practical and educational session will deliver an introduction and overview on the use of evolving gene therapies in the rare disease space and the challenges faced in clinical development, including discussion on use of biomarkers, novel complex endpoints and innovative trial designs.  Insights will be provided into ‘lessons learnt’ from the perspectives of four highly experienced experts in the field.

These events are for professionals working across the science and practice in rare diseases and gene therapies and have been organised by the FPM Rare Disease and Gene Therapy Expert group.

Day 1

Lessons learnt in Gene Therapy & Rare Disease Development 

10 May 16:00-18:00


Day 2

Lessons learnt in Gene Therapy & Rare Disease Patient Engagement and Access

12 May 16:30-18:00



See below for Day 1 tickets, as well as multi-day discounts.

Booking is now closed

*Reduced rates are available for:
  • PMST trainees
  • People from low and lower-middle income countries
  • Unemployed
  • Part-time employed (earning less than £15,000 per year)
  • People on Parental Leave
  • People who are Out of Practice & Retired
  • Charity representatives


Dr Zoya Panahloo – Welcome.

Prof Alan Boyd – Innovative trial designs and regulatory approached in rare diseases – how to maximise chances of success.

Dr Anthony Hall – The use of “basket trials”, biomarkers and novel complex endpoints.

Dr Arseniy Lavrov – Considerations in Gene Therapy development: highlighting successes and challenges.

Dr Suyash Prasad – What are the lessons we have learnt so far in this newly developing field?

Panel discussion and Q&As

Mapping the PMST curriculum:

  • CiP 5 “Identifies, manages and mitigates risk in special populations” 
  • CiP 11 “remains up to date with research and best practise” 
  • Market Access, Engagement with Key Stakeholders, Patient Organisations 

Day 1 - Speakers and Chair

Dr Zoya Panahloo


Dr Zoya Panahloo a physician by background, is currently Senior global Medical Director in rare neurological diseases at UCB and Chair of the FPM Rare Disease and Gene Therapy Expert Group of FPM. She has over 15 years’ experience in both large Pharma and small biotech, specifically in Global Medical Affairs roles and in rare neurological Diseases area.

Booking Information

FPM will close bookings no later than 13:00 on 10 May 2022

Queries: If you are unable to complete your booking online or you have questions, please email

Joining instructions will be emailed to attendee’s registered e-mail address, with a reminder sent on 10 May 2022.

This event will be held on Zoom.


If you attend this course you can use it as CPD for your appraisal. To do so you’ll need to provide:

  • evidence (certificate of attendance), and
  • reflection, specifically a couple of paragraphs on what you learnt, what effect it will have on your current practice and how it relates to Good Medical Practice.

Upload these into your portfolio and self-allocate your CPD points at 0.25 credits per 15 mins.

Refund policy

  • For cancellations up to 1 week before the start date a full refund will be given.
  • For cancellations between 1 week and 1 day before the start date a 50% refund will be given.
  • Refunds cannot be given after 13:00 10 May 2022.
  • In the event of cancellation by FPM a full refund will be given.

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The views, information, or opinions expressed during FPM events and training are those of the individuals involved and do not necessarily represent those of the Faculty of Pharmaceutical Medicine. We value inclusivity, equality and diversity, and work hard to promote these whenever possible in all of our activities. We welcome your comments and feedback: