T. Montana schreef op 12 oktober 2016 08:54:
JEFF Gene editing/therapy summit:
QURE (Buy) provided an update on its gene therapy programs utilizing AAV5 (Link). To date, ~25 pts have been treated with AAV5 across its clinical studies. While QURE has its proprietary gene therapy programs (e.g., hemophilia A, MPS IIIB), we believe the key driver for QURE valuation lies in its CV gene therapy collaboration with BMY; Ph1 for lead gene therapy product S100A1 for CHF set to begin in 2017. For its proprietary programs, Ph1/2 high-dose AMT-060 6-month data for hemophilia B will be presented at ASH 2016 (Dec. 3-6),
expecting ~5-10% FIX activity (vs. 5.4% FIX activity at low-dose). For AMT-110 for Sanfilippo B (MPS IIIB), Ph1 follow-up data is expected in 1Q17, which could be informative on clinical benefits of AMT-110.