Spark Therapeutics Have Stopped Their Pompe Program

Spark Therapeutics Have Stopped Their Pompe Program

Posted On: 19/07/2024

Disappointing news that Spark Therapeutics will not continue with their gene therapy program in Pompe Disease.

Pompe Support Network is disappointed to learn that Spark Therapeutics have made an announcement that they have discontinued their gene therapy program in Pompe disease. The program had been paused in 2022. Participants in the study will be provided with the follow-up and support required by the study protocol. Spark Therapeutics' community update says there were no safety issues related to the decision but it was due to a portfolio review and realignment of strategy.

Please see a pdf of their update below.

Spark Therapeutics Pompe Community Letter