Venture capitalists are giving the risky field of gene therapy a new dose of confidence—and cash. Genetix Pharmaceuticals, a 17-year-old developer of gene therapies, has replenished its coffers with a $35 million Series B round of venture capital.
The Cambridge, MA-based company attracted the fresh capital after a study in France showed that one of the firm’s gene therapies blocked the progression of a debilitating brain disorder called adrenoleukodystrophy (ALD) in two children. This is the disease that was featured in the acclaimed 1992 film “Lorenzo’s Oil,” the true story of a husband and wife who searched for a cure for their son with the crippling illness. While much testing will be required to bring the gene therapy to market, Boston’s Third Rock Ventures and Cambridge-based biotech giant Genzyme were convinced that there was enough promise in the data to make big bets on the company.
Third Rock and Genzyme Ventures, the venture unit of Genzyme (NASDAQ:[[ticker:GENZ]]), are the new investors in Genetix’s second-round financing, according to the company. The round includes investments from the firm’s previous VC backers Easton Capital, Forbion Capital Partners, and TVM Capital. The new funding came with major management changes at Genetix: Third Rock partner Nick Leschly is leading the firm as interim president; Phil Reilly, a venture partner at Third Rock, has become chief medical officer; and Mitchell Finer, a veteran biotech executive, is the new chief scientist. Genetix CEO Alfred Slanetz is leaving the firm.
Gene therapies, which typically use viruses to deliver healthy genes into cells to treat diseases, have never been approved for the market nor lived up to the hype they initially generated about two decades ago. Genetix is one example of renewed faith in the science in some scientific and investor circles. The company, one of hundreds like it that formed in the 1990s to develop gene therapies, was recapitalized in 2004 and licensed technology from the French
