{"id":49521,"date":"2022-10-12T09:01:57","date_gmt":"2022-10-12T07:01:57","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/sangamo-therapeutics-announces-updated-preliminary-phase-1-2-data-in-fabry-disease-showing-continued-tolerability-and-sustained-elevated-%ce%b1-gal-a-enzyme-activity-in-nine-patients\/"},"modified":"2022-10-12T09:01:57","modified_gmt":"2022-10-12T07:01:57","slug":"sangamo-therapeutics-announces-updated-preliminary-phase-1-2-data-in-fabry-disease-showing-continued-tolerability-and-sustained-elevated-%ce%b1-gal-a-enzyme-activity-in-nine-patients","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/sangamo-therapeutics-announces-updated-preliminary-phase-1-2-data-in-fabry-disease-showing-continued-tolerability-and-sustained-elevated-%ce%b1-gal-a-enzyme-activity-in-nine-patients\/","title":{"rendered":"Sangamo Therapeutics Announces Updated Preliminary Phase 1\/2 Data in Fabry Disease Showing Continued Tolerability and Sustained Elevated \u03b1-Gal A Enzyme Activity in Nine Patients"},"content":{"rendered":"
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\n– Isaralgagene civaparvovec, or ST-920, continued to be generally well tolerated across four dose cohorts in the nine treated patients in the dose escalation phase.<\/i>\n<\/p>\n

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\n– All nine patients exhibited elevated \u03b1-Gal A activity, ranging from nearly 2-fold to 30-fold of mean normal, for up to 23 months post dosing, as of the last date of measurement.<\/i>\n<\/p>\n

\n– Four patients were withdrawn from enzyme replacement therapy (ERT) and demonstrated significantly elevated levels of <\/i>\u03b1-Gal A<\/i> activity up to 28 weeks post withdrawal.<\/i>\n<\/p>\n

\n– Since the cutoff date, one additional patient was withdrawn from ERT.<\/i>\n<\/p>\n

\n– The Phase 1\/2 STAAR study has progressed into the expansion phase, with four patients dosed, including the first female patient.<\/i>\n<\/p>\n

BRISBANE, Calif.–(BUSINESS WIRE)–Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced updated preliminary results 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. These latest data show that, as of the July 21, 2022 cutoff date, the investigational treatment continued to be generally well tolerated, with no treatment-related serious adverse events. The nine patients in the dose escalation phase exhibited elevated alpha-galactosidase A (\u03b1-Gal A) activity, sustained for up to 23 months for the patient with the longest follow-up.\n<\/p>\n

\nThese updated data will be shared today via an oral presentation at the 29th<\/sup> Congress of the European Society of Gene & Cell Therapy \u2013 ESGCT 2022 \u2013 from 8:30-10:40 a.m. British Summer Time (Ref: OR21). These data are also available on Sangamo\u2019s website on the Events & Presentations<\/a> page.\n<\/p>\n

\n\u201cI\u2019m encouraged by these results, showing isaralgagene civaparvovec gene therapy has reassuring safety data to date, with no requirement for corticosteroid therapy,\u201d said Dr Patrick Deegan, MD, Cambridge University Hospitals NHS Foundation Trust, and investigator of the Phase 1\/2 study. \u201cST-920 has the potential to provide an alternative to enzyme replacement therapy for patients with Fabry disease. I look forward to seeing additional data as we continue to progress this important program.\u201d\n<\/p>\n

\nAs of the cutoff date, all nine patients treated in the dose escalation phase across the four dose cohorts (0.5e13 vg\/kg, 1e13 vg\/kg, 3e13 vg\/kg and 5e13 vg\/kg), sustained elevated \u03b1-Gal A activity ranging from nearly 2-fold to 30-fold of mean normal at the last date of measurement.\n<\/p>\n

\nCohort 1<\/i>\n<\/p>\n