Gene Therapy for Rare Diseases

A San Francisco startup called Audentes Therapeutics has $30 million in funding to develop gene therapy for rare diseases

Netherlands-based UniQure broke through in November, 2012 with the first gene therapy approved in Europe, and growing scientific enthusiasm around a series of technical improvements helped propel Cambridge, MA-based Bluebird Bio (NASDAQ: BLUE) to one of the most successful biotech IPOs of the year.

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