GSK Is First Big Pharma To Seek Gene Therapy Approval, Plans More
This article was originally published in The Pink Sheet Daily
Executive Summary
Britain’s biggest drug maker is seeking regulatory approval in Europe to use gene transfer technology in children born with ADA-SCID and has further programs using the approach in two other rare diseases - metachromatic leukodystrophy and Wiskott-Aldrich syndrome - with both currently in clinical trials.