Robert J. Hopkin, MD, on Safely Delivering Gene Therapy for Fabry Disease

“One of the big issues with gene therapy is questions of safety. And in many of the gene therapy trials, we see inflammatory reactions that involve the liver, kidneys, heart or other organs. And this trial, we have had really very little in the way of inflammatory reactions. We see some fever, in the first day or 2, we had some reports of headaches and cold like symptoms. But that resolves early. And then the safety has been quite stable.”

Isaralgagene civaparvovec gene therapy (ST-290; Sangamo Therapeutics) was well-tolerated in adult patients with symptomatic Fabry disease and yielded durable, supraphysiological levels of α-Gal A activity for up to36.2 months. In 13 participants with at least 12 months of follow-up, renal function remained stable, Fabry outcome survey Mainz Severity Score Index improved, and 38% of participants on enzyme replacement therapy (ERT) improved in disease severity category. Furthermore, all 12 participants that discontinued ERT remain off ERT for up to 19 months as of data cut off.

These data, from the phase 1/2 STAAR study (NCT04046224), were presented by Robert J. Hopkin, MD, associate professor, clinical pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, at the 2024 WORLDSymposium, held February 4-9, in San Diego, California.

CGTLive spoke with Hopkin to learn more about the results. He went over the highlights of the updated biomarker data from STAAR. He shared the encouraging safety data and noted that no steroid or immunosuppressants were used in the course of the study, unlike with other gene therapy trials, and no serious complications have been seen so far.

REFERENCE

Hopkin RJ. Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing Phase 1/2 study (STAAR). Presented at: WorldSymposium; February 4-9; San Diego, California.