Cooking with the Genzyme Recipe: New Players Funding Rare Disease Drugs in Boston

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fill their pipelines to continue to evolve,” says Nick Leschly, a partner at Third Rock Ventures, “and nobody’s scoffing at $150 million, $250 million, or $500 million markets anymore.”

Third Rock has hired experts on rare genetic diseases as part of its strategy to find investment opportunities in this field over the past couple of years, Leschly says. He credited Philip Reilly, one such expert who joined the firm in 2009, for finding a promising experimental therapy for X-linked hypohidrotic ectodermal dysplasia in Switzerland. The rare genetic disease, which affects 1 in 17,000 people, robs them of their ability to sweat, and stunts the normal development of teeth and hair, according to the National Institutes of Health. Third Rock gained rights to the Swiss therapy and formed Cambridge-based Edimer Pharmaceuticals last year to advance it into clinical trials.

Edimer quietly raised more than $20 million in a round of funding from Third Rock and the Swiss venture firm VI Partners last year, according to Leschly, a member of the firm’s board of directors. Last month he also led his firm’s investment in the $35 million second-round funding for Genetix Pharmaceuticals, a Cambridge-based developer of a gene therapy for a rare brain disorder called adrenoleukodystrophy. Genzyme Ventures, the investment arm of Genzyme, is another new investor in Genetix.

Rare disease deals aren’t limited to the Boston area. Last month Carlsbad, CA-based Isis Pharmaceuticals pocketed $35 million in upfront cash from Glaxo in a deal that is worth up to $1.5 billion to discover new gene-silencing drugs for rare diseases and other serious illnesses.

“There’s a shift away from the blockbusters like anti-cholesterol and anti-diabetes drugs towards some of the orphan disorders,” says Michelle Dipp, a vice president in charge of Glaxo’s U.S. Center for Excellence in External Drug Discovery in Cambridge, MA. “So people are trying to build a mini-Genzyme … Next Page »

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One response to “Cooking with the Genzyme Recipe: New Players Funding Rare Disease Drugs in Boston”

  1. Jerry Jeff says:

    Well, here’s hoping for an end to these captive markets. All those billions come out of our health insurance premiums, after all. And what’s up with that picture?