Three patients who received SPK-8011 — a gene therapy developed by Spark Therapeutics — for hemophilia A show increased production of clotting factor VIII without any safety issues reported, says the company.
The patients are the first to receive the treatment as part of a Phase 1/2 clinical trial, with early data showing they have not yet experienced spontaneous bleeding episodes.
“The encouraging start of our SPK-8011 clinical trial reinforces the strength of our gene therapy platform, delivers human proof-of-concept in a second liver-mediated disease — a significant achievement in the gene therapy field — and positions us well to potentially transform the current treatment approach for this life-altering disease with a one-time intervention,” Katherine A. High, president and CEO of Spark Therapeutics, said in releasing second-quarter 2017 financial results.
Source: Hemophilia News Today