Confusing data from the first in vivo genome editing trial and the decision to move to next generation editing technology pushed the Phase 3 decision for MPS II into 2020.
Hemophilia A gene therapy is progressing quickly per accounting disclosures. It remains under the radar due to a lack of disclosure of "competitive data".
Clinical initiations for beta-thalassemia and hemophilia B were delayed for most of 2018. Patients are now being dosed.
The investigational new drug application for Fabry, expected in early 2018, was delayed for undisclosed reasons. It was approved by the FDA in January 2019 with initiation expected by year end.