A little bit of background in the form of an Editorial from Molecular Therapy, a well-regarded gene therapy journal (pay-walled, I assume): http://www.nature.c

A little bit of background in the form of an Editorial from Molecular Therapy, a well-regarded gene therapy journal (pay-walled, I assume): http://www.nature.com/mt/journal/v20/n10/full/mt2012194a.html http://www.nature.com/mt/journal/v20/n10/full/mt2012194a.htm... An interesting paragraph from the editorial, indicating that the regulatory process for this sort of gene therapy is still quite difficult: "This lengthy and tortuous approval process raises several questions. Frequent reapplications are very time-consuming and expensive, which in this case led to the demise of Amsterdam Molecular Therapeutics, although thankfully private investors were able to continue the process. Nevertheless, investors will shy away from supporting biotech companies unless greater clarity and predictability can be achieved in the process of regulatory evaluation and approval. The Glybera saga also highlights problems specific to ultra-orphan drugs—because obtaining large-scale phase III data with a very limited number of patients is virtually impossible, procedures to handle these indications must be further developed."