Content area
Full Text
N E W S Biogens anti-LINGO
promises nerve repair
Heart failure gene therapy disappoints but experts keep the faith
The largest study using gene transfer to treat advanced heart failure patients has yielded negative results, its sponsor Celladon announced in April. But experts in the field remain encouraged that the strategy used in the CUPID2 phase 2b trial is sound, and that it will reveal valuable clues for future studies through analyzing dosing and duration as well as the vector and delivery methods. Meanwhile, only days before the San Diego biotech announced its findings, Bristol-Myers Squibb announced an agreement with Amsterdam-based gene therapy pioneer uniQure (Nat. Biotechnol. 30, 807, 2012) for exclusive rights to up to ten gene therapy targets in cardiovascular and other diseases, including its program in congestive heart failure aimed at restoring a calcium sensor, S100A1. Under the collaboration, uniQure receives a $50-million upfront payment and potential milestones and royalties of up to $254 million for the S100A1 program. The New York-based big pharmas buy-in adds momentum to the notion that improvements in the design and delivery of gene therapy may provide the wherewithal to tackle a cardiac indication.
Heart failure is a leading cause of hospitalization in the US and Europe and its incidence continues to grow. This is because although
treatments to open coronary...