First participant dosed in the RESOLUTESM trial, a Phase 1/2 dose-escalation study of SPK-3006
Enrollment of approximately 20 total study participants is ongoing
PHILADELPHIA, February 1, 2021 (GLOBE NEWSWIRE)— Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. company dedicated to challenging the inevitability of genetic disease, today announced the dosing of the first participant in the Phase 1/2 RESOLUTESM trial of SPK-3006, an investigational liver-directed adeno-associated viral (AAVA harmless virus commonly used as a delivery system in gene therapy to carry healthy genes into cells.) vectorA delivery system, often based on a modified virus, used to carry genetic material into cells. gene therapy for late-onset Pompe disease (LOPD)A form of Pompe disease that begins after infancy and usually progresses more slowly., a rare, inherited lysosomal storage disorder.
“Dosing the first participant in the Phase 1/2 RESOLUTE trial of investigational SPK-3006 for late-onset Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart. 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 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 infusionA method of delivering medication through an IV. of investigational SPK-3006, an AAV vector-based gene therapy, developed in collaboration with Genethon, in adults with clinically moderate LOPD currently receiving enzymeA protein that helps the body carry out chemical reactions. replacement therapy. The study is expected to enroll approximately 20 participants receiving the investigational gene therapy in sequential, dose-level cohorts. Additional details are available on ClinicalTrials.gov (NCT04093349).
“We are honored to have the first participant dosed in this clinical trialA research study that tests new treatments or approaches in people., 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.