Gene therapy hitting its stride as M&A slows and biosimilars challenge


Jennifer Boggs 
Managing Editor, BioWorld, Clarivate Analytics

After a few decades of fits and starts, it looks like 2017 might just go down in the books as the year gene therapy is finally ready for prime time.

The notion of treating disease by replacing a missing gene or correcting a defective one emerged in the late 1980s and 1990s as a promising approach, replete with all the accompanying hype and bioethics debates. But the tragic death of a teenage patient in a gene therapy trial in 1999 put a damper on the space. Progress came more slowly after that, and what appeared as a win in 2012 – the hard-fought approval of Glybera (alipogene tiparvovec) in Europe for an ultra-orphan disease – quietly became another disappointment. In April of this year, Glybera’s developer, Uniqure BV, axed commercial sales citing “economic reasons,” though sticker shock might have contributed – the one-off therapy came with a whopping $1.1 million price tag.

But then came September, which kicked off a flurry of gene therapy news, starting with the FDA approval of Novartis AG’s CAR T immunotherapy Kymriah (tisagenlecleucel, previously CTL-019) in children and young adults with B-cell acute lymphobla ...