Spark Therapeutics Announces First Participant for LOPD

Spark Therapeutics Announces First Participant for LOPD

Posted On: 01/02/2021

Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease.

Commercial gene therapy company, Spark Therapeutics, has today announced dosing the first Pompe patient participant in the Phase1/2 RESOLUTE trial of investigational SPK-3006 for late-onset Pompe disease.

The UK has one of the 18 study centres at Salford Royal Hospital and we look forward to monitoring progress with the trial recriotment and its results later this year.


“Dosing the first participant in the Phase 1/2 RESOLUTE trial of investigational SPK-3006 for late-onset Pompe disease is an important milestone and first step to what we hope will ultimately allow us to bring an innovative gene therapy to these patients,” said Gallia G. Levy, M.D., Ph.D., chief medical officer of Spark Therapeutics. “We are deeply appreciative of the ongoing collaboration of the Pompe disease community as we continue to enroll participants in this Phase 1/2 study.”

“The International Pompe Association has been proud to collaborate with Spark Therapeutics to enhance the Pompe disease community’s understanding of gene therapy research,” said Tiffany House, International Pompe Association Board Chairman. “We look forward to the progress in the Phase 1/2 RESOLUTE trial, as well as the ongoing work aimed at developing gene therapies that have the potential to help individuals living with genetic diseases.”


For the full report please visit: https://sparktx.com/press_releases/first-patient-dosed-pompe/