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


The associate professor of clinical pediatrics at Cincinnati Children's discussed the latest data from the phase 1/2 STAAR study of isaralgagene civaparvovec.

“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.

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.
Related Videos
Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital
M. Peter Marinkovich, MD, on Bringing RDEB Treatment to the Local Level
Caspian Oliai, MD, MS, the medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center
Frederick “Eric” Arnold, PhD
Genovefa (Zenia) Papanicolaou, MD, an infectious diseases specialist at Memorial Sloan Kettering Cancer Center
Jeffrey Chamberlain, PhD, on Exciting New Research at MDA 2024
Alan Beggs, PhD, on Challenges in Therapeutic Development for Rare Diseases
Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital
PJ Brooks, PhD
John DiPersio, MD, PhD, the director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine
© 2024 MJH Life Sciences

All rights reserved.