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Spark Therapeutics doses the first participant in Gene Therapy trial.

Spark Therapeutics announced on Monday that the first participant was dosed in the RESOLUTE trial. This is a Phase 1/2 dose-escalation study of SPK-3006, an investigational liver-directed adeno-associated viral (AAV) vector gene therapy for late-onset Pompe disease (LOPD).

Chief Medical Officer of Spark Therapeutics

“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.”

Enrollment of approximately 20 participants is ongoing

The RESOLUTE trial is an open-label Phase 1/2, dose-escalation gene transfer study designed to evaluate the safety, tolerability and efficacy of a single intravenous infusion of investigational SPK-3006, an AAV vector-based gene therapy, developed in collaboration with Genethon, in adults with clinically moderate LOPD currently receiving enzyme replacement therapy. The study is expected to enroll approximately 20 participants receiving the investigational gene therapy in sequential, dose-level cohorts.

“We are honored to have the first participant dosed in this clinical trial, which we hope will lead us to introduce a novel therapeutic option for patients living with late-onset Pompe disease.” said Principal Investigator Tahseen Mozaffar, M.D., University of California Irvine Health.

The full press release can be found via the following link.

Spark Therapeutics full press release