" class="no-js "lang="en-US"> Sangamo Announces Benefit in Study in Fabry Disease
Sunday, March 03, 2024

Sangamo Therapeutics Announces Evidence of Clinical Benefit in Phase 1/2 STAAR Study in Fabry Disease

Sangamo Therapeutics, a genomic medicine company, today announced updated preliminary data as of the October 20, 2022 cutoff date from the Phase 1/2 STAAR clinical study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease. This data, which present new biomarker data and results from the first kidney biopsies in this study, indicate evidence of clinical benefit for isaralgagene civaparvovec in Fabry disease.

As of the November 15, 2022 supplemental cutoff date, 13 patients across the dose escalation and expansion phases exhibited supraphysiological levels of α-Gal A activity, sustained for over two years for the patient with the longest follow-up. All five patients who began the dose escalation phase on ERT had been successfully withdrawn from ERT and continued to exhibit supraphysiological levels of α-Gal A activity following withdrawal. No patient has required the resumption of ERT treatment to date.

Importantly, there is evidence of significant Gb3 substrate reduction at six months in one of the first kidney biopsies taken from this study, along with a significant reduction in urine podocyte loss. In addition, the study observed a clinically meaningful and statistically significant increase in mean general health scores, as measured by the SF-36 General Health survey.

“Fabry is a debilitating disease with life-long impact,” said Dr. Robert Hopkin, MD, Cincinnati Children’s Hospital Medical Center, and investigator of the Phase 1/2 study. “The combination of the first kidney biopsy results and the associated urine podocyte data are highly encouraging and compelling. As a whole, this exciting dataset shows that ST-920 has the potential to improve the lives of patients without the need for burdensome ERT treatment.”

  1. Eloxx Pharmaceuticals Announces Final Data Assessment from Phase 2 Combination Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis Patients Read more
  2. Verge Genomics Announces Positive Safety and Tolerability Data from the Phase 1 Clinical Trial of VRG50635, a Potential Best-in-Class Therapeutic for All Forms of ALS Read more
  3. DEM BioPharma Appoints Wendy Young, Ph.D., to Scientific Advisory Board Read more
  4. Confo Therapeutics Enters into Research Collaboration for GPCR-Targeting Antibody Discovery with AbCellera Read more
  5. Vyriad Announces Expansion of T-Cell Lymphoma Trial at Mayo Clinic Read more