Genetix raises $35M to advance gene therapies

Cambridge, MA-based Genetix Pharmaceuticals has completed a $35 million Series B financing with new investors Third Rock Ventures and Genzyme Ventures joining TVM Capital, Forbion and Easton Capital. The company was founded in 1993, but in 2004 Genetix was re-started and recapitalized in a Series A investment by TVM, Forbion, and Easton, and has raised a total of $23 million in Series A and bridge funding since then. "The whole point of this financing is to (1) advance the programs in the clinic, (2) expand and broaden the platform of the company, and (3) expand the team," explained Genetix President Nick Leschly (also of Third Rock Ventures) in an interview with FierceBiotech. He said this round will give the company a cash runway of three or more years.

Genetix has two lead programs in the Phase II trials. Lenti-D is an experimental therapy for Adrenoleukodystrophy (ALD), a severe neurodegenerative disorder that affects one in 17,000 males. Its other program is Lentiglobin for beta-Thalassemia, one of the most prevalent human genetic disorders. Both therapies use Genetix's gene therapy platform to re-engineer patient's own cells to treat the rare and potentially fatal diseases. And Leschly notes that its technology is being explored for other genetic conditions. The funding will be used to immediately move both lead programs into pivotal trials.

Genetix currently has 15 employees but expects to double over the next 12 to 18 months. And it's already getting that process under way. In addition to today's financing, the company has announced that it named Mitchell Finer, Ph.D. as chief scientific officer. Finer is a former Novocell exec with years of experience in gene therapy and regenerative medicine. As Genetix news CSO, he'll help guide the company's existing programs while exploring other applications for its technology.

"The intent of the company is to create a collaborative networks with both disease foundations, academic institutions, and industry," explained Leschly. "We're in ongoing discussions with a number of folks who are interested in our programs." And with a disease-altering technology platform, you can bet Big Pharma will take notice. Leschly wouldn't give specifics on any companies that would be interested in Genetix technology, but he did point out that developers like Biomarin, GlaxoSmithKline, Shire and Novartis have all expressed interested in pursuing treatments for rare genetic diseases or severe diseases. And investor Genzyme Ventures is the VC arm of Genzyme, which has built its business on treating rare diseases.

- here's Genetix's release on the funding
- and take a look at the announcement on Finer's appointment