Potential Gene Therapy SPK-8011 Fares Well in Hemophilia A Study

Potential Gene Therapy SPK-8011 Fares Well in Hemophilia A Study

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Potential Gene Therapy SPK-8011 Fares Well in Hemophilia A Study

A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical trial and its extension study. Notably, the sustained FVIII levels allowed the discontinuation of prophylaxis, or preventive FVIII therapy, and resulted in a pronounced reduction or complete…

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