About Event

Gene Therapy for Rare Disorders is a uniquely focused conference that will unite the leaders in this field to identify and discuss the critical factors that will influence genetic treatments most significantly in the near future.

Bringing together leading experts from innovative biotechs, large pharma, academia and key service provider companies, Gene Therapy for Rare Disorders will present a comprehensive insight into the cutting edge progress of gene therapies combatting rare disease.

See the full highlights from our 2018 meeting below:


Companies in attendance included:

Spark Therapeutics – FDA – uniQure – Pfizer – GSK – Rocket Pharma – Sanofi-Genzyme
Bluebird Bio – Adverum – Aquilo Capital Management – REGENXBIO
Adrenas Therapeutics – BioMarin – Bioverativ – Dimension Therapeutics

NORD – Milo – Shire – Voyager – AvroBio – Burns & Levinson LLP – Harvard Medical School
Noah’s Hope – Sarepta Therapeutics – University of Massachusetts Medical School

Solid Biosciences – Atlas Venture – Voisin Consulting – Abeona Therapeutics – Agilis

Join your colleagues at Gene Therapy for Rare Disorders to accelerate the progress of the next generation of gene therapies that show improved efficacy, enhanced safety and commercial viability.

Secure your place for the 2019 meeting now to gain an exclusive early booking rate!