Padua gambit seems to have worked as new gene therapy looks the equal of Pfizer/Spark Therapeutics.
As data on Amicus Therapeutics' clinical-stage Pompe disease candidate roll in, the group locks up preclinical gene therapy assets.
Companies that want to play in rare diseases need to shop for gene therapies, but price tags are looking rich.
Reaction to data from Sangamo's ongoing genome-editing trial shows that small numbers can equal big risks.
Results of a phase I/II trial in Hunter syndrome will have implications for Gliead, and a Cushing's candidate will be watched for side-effects.