Spark Therapeutics' Hemophilia Gene Therapy Achieve Stable, Durable FVIII Expression

Spark Therapeutics, a unit of Roche Holdings AG RHHBY, has announced updated data from the ongoing Phase 1/2 trial of SPK-8011 gene therapy in hemophilia A.
Data were presented at the International Society of Thrombosis and Hemostasis (ISTH) 2021.
These data demonstrated that administration of SPK-8011 resulted in sustained factor VIII (FVIII) expression in 16 of 18 participants with up to 4 years of follow-up, as of the May 3, 2021 data cutoff.
Eighteen participants in the Phase 1/2 trial received a single administration of investigational SPK-8011 in four dose cohorts, ranging from 5x1011 vg/kg to 2×1012 vg/kg. In the 16 patients with sustained FVIII expression, there was a 91.2% reduction in annualized bleed rate (ABR) and a 97% reduction in annualized FVIII infusion rate (AIR) after vector administration.
SPK-8011 showed an acceptable safety profile with no deaths and no FVIII inhibitor development with up to 4 years of follow-up.
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