With over 40 solution providers, the virtual Clinical Trials in Rare Diseases Conference 2021 will be a perfect platform to reconnect with current business partners, meet new vendors and discover which new technologies are being employed to improve Rare Disease clinical trials.

Dr Magali Taiel, Chief Medical Officer, will give an oral presentation on our lead product candidate LUMEVOQ® (GS010; lenadogene nolparvovec) as detailed below:


Tuesday 14th September 2021

Time:  9:00am – 9:30am CET

Rare disease in clinical development: learnings from development of Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy (LHON)

  • Overview: key aspects of Lumevoq Gene Therapy development
  • Regulatory pathways and challenges around documentation and working with regulatory authorities
  • Recruiting and following up with patients in order to maximise patient numbers and minimise attrition throughout the study
  • Key learnings and takeaways: what do you need to know when running a gene therapy study?