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Spark Therapeutics Announces Updated Phase 1/2 Study Results Supporting the Durability of Investigational Gene Therapy SPK-8011 in Patients With Hemophilia A
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Spark Therapeutics SPK-8011 Suggests Stable and Durable Factor VIII Expression in Largest Phase 1/2 Gene Therapy Study in Hemophilia A to Date
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F Hoffmann-La Roche Ltd: Roche to present new data at the ISTH 2021 Congress highlighting long-standing commitment to advancing haemophilia A standard of care
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Investegate |F Hoffmann-La Roche Ltd Announcements | F Hoffmann-La Roche Ltd: Roche to present new data at the ISTH 2021 Congress highlighting long-standing commitment to advancing haemophilia A standard of care
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Spark Therapeutics Announces Preliminary Data from Phase 1/2 Clinical Trial of SPK-8016 in .
Spark Therapeutics, Inc.February 5, 2021 GMT
Preliminary data from four participants who received investigational SPK-8016
at a dose of 5X10
11
vg/kg and have no history of FVIII inhibitors show no serious adverse events and stable and durable factor VIII (FVIII) expression at greater than 52 weeks
Data show 98-percent reduction in annualized infusion rate (AIR) and 85-percent reduction in annualized bleed rate (ABR) after a follow up of between 15 and 18 months or 5.5 total patient years