Is this the end of the line for Celladon after its gene therapy flop?
- Celladon specializes in gene therapy. But its key therapy, Mydicar, failed in a mid-stage trials earlier this year, and now the firm is saying it may be forced to liquidate if it cannot find a buyer.
- If Celladon liquidates, it may occur in September, and there will be a payout to shareholders of $25 million to $30 million.
- Celladon has also announced a workforce reduction, which will cut its 34-person staff in half.
The one-time cure of hard-to-treat diseases is the holy grail of gene therapy researchers. However, in the last 20 years, efforts to develop a safe, effective therapy have often been met with acute and long-term failures. Despite this, new companies continue to enter the gene therapy arena, and there have been some recent succeses.
Unfortunately, Celladon was not one of them. In April, its once-promising gene therapy candidate, Mydicar, a virus designed to deliver a gene that reconstitutes an enzyme reponsible for heart muscle contraction, failed in mid-stage trials.
Recent successes in this space include UniQure NV, Spark Therapeutics, and Bluebird Bio, all of which have recently had successful IPOs. Bluebird has continued to make headlines as it ushers its gene therapy for beta-thalassemia and sickle cell disease through the development process. And now Bluebird has successfully negoatiated an expedited approval pathway with the FDA.