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Therapeutics For Rare And Neglected Diseases

NIH Translational Group To Help Biotechs Cross Valley Of Death

This article was originally published in Start Up

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Rare Disease Research: Where Compassion Meets Commerce

The advent of a new set of potential acquirers has heightened venture capital's interest in rare diseases, resulting in the creation of a new cohort of drug developers that are using cutting edge technologies to treat and in some cases cure grave illnesses. In this issue, we profile Afraxis, bluebird bio, Edimer Pharmaceuticals and Ultragenyx Pharmaceutical.

Sanofi/Genzyme: Emblematic of What Big Pharma's Buying Now

Sanofi-Aventis' much publicized pursuit - first hostile, then friendly - of Genzyme dominated biopharma news flow for more than half a year, with well timed information leaks and dramatic "he said, he said" commentary from the firms' CEOs. Such back and forth ensured continued high profile coverage until the companies officially affianced. But beyond the drama, the $20.1 billion deal, among the largest since the 2009 mega-mergers between Pfizer and Wyeth and Merck & Co. and Schering-Plough Corp., is worth discussion because it provides insight into what now drives big pharma dealmaking in the post-blockbuster era - and how companies want to pay for it.

AAVLife: Another Gene Therapy Effort To Treat Friedreich's Ataxia

With the launch of AAVLife, Versant Ventures adds to its gene therapy portfolio, and people with Friedreich's ataxia can look to another program that takes aim at the rare inherited disease.

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