Europe approves high-price gene therapy

On the other, and I speak from experience, we really don't fully understand what goes on in terms of gene expression regulation. We have ideas which generally work in vitro, mostly in vivo with model organisms (drosophila, yeast, etc) but relatively little basic research looking at fundamental mechanisms of regulation is done in higher eukaryotes. The upshot is that we may have ideas of what's happening, but those ideas may be one of several possible explanations which lead to similar outcomes. Moreoever, the impact of gene therapy in terms of a long term cellular (and immune) response is poorly understood.

I'd be intrigued to see what longitudinal data have been generated to show Glybera's safety over the kinds of timeframe relevant here.

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."

Genetic improvement shouldn't be built from the ground up as the territory of the extremely wealthy alone.