After decades of unfulfilled promises and setbacks, the field of gene therapy broke through with three FDA-approved products last year, ushering in what is likely to be a rapid escalation of new treatments for some of the rarest and most debilitating diseases.

“Part of the idea of the Human Genome project was that once we had the identity of all the genes, it would be important and straight forward for us to development more therapeutic options for people with serious inherited diseases, said Katherine High, M.D., president and director of research and development for Philadelphia-based Spark Therapeutics. “But it turned out this took a little longer to do than the Human Genome project.” Continue reading →