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In ALD, which usually strikes boys during early childhood, bone marrow transplants have been effective in holding back the disease’s devastating symptoms such as the deterioration of brain function. Genetix’s treatment could treat the majority, or about 70 percent, of ALD patients, for whom there are no matching donors to give them bone marrow transplants, according to the company.
Genetix itself is now getting the Third Rock treatment, which is typically an infusion of both fresh capital and members of the venture firm to run the operation on an interim basis. This formula has been applied to other biotech companies in the three-year-old venture firm’s portfolio, including Boston-area biotech startups Agios Pharmaceuticals, Constellation Pharmaceuticals, and Eleven Biotherapeutics. Genetix is much older than other companies to get investments from Third Rock’s maiden fund of $378 million, but the company won the firm’s confidence with its strong clinical data, Leschly says.
On the other hand, it’s no surprise to see Genzyme in the mix at Genetix. Genzyme, which is the world’s largest provider of enzyme-replacement drugs for genetic diseases, has been pursuing gene therapy research consistently since the early ’90s without a late-stage product to show for it. The firm’s most advanced gene therapy is one in Phase I trials for Parkinson’s disease. Leschly says that Genzyme is only a financial investor in Genetix and has no rights to its intellectual property, yet the biotech powerhouse obviously wants to keep tabs on Genetix’s progress.
Amid the excitement around the firm’s recent progress, Leschly knows that Genetix has much more work to do before its treatments are available to patients. “There is a sense of humility around here; we do understand that this isn’t going to be a cakewalk, not all things are going to work, and patients aren’t going to have access to this tomorrow,” he says. “These are early data, but they are a launching pad, and we’re very excited about that.”
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