Rare Disease Research: Where Compassion Meets Commerce
This article was originally published in Start Up
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.
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Start-ups developing new drugs for rare cancers and other disorders characterized by excessive hormone production know successful treatments are likely to eventually find larger markets. Profiles of Atterocor, Crinetics Pharmaceuticals, and OctreoPharm Sciences.
Ultragenyx raised $75 million in a Series B financing led by Adage Capital Partners. The funding will support the biotech’s two lead programs that are meant to treat ultra-rare genetic disorders and will position the company for an initial public offering.
Alexion’s “hotly contested” purchase of Enobia last December didn’t get much attention, but it marked a watershed year for rare disease drug deals. Will high drug prices, skittish regulators and a gold-rush push by Big Pharma start to sour the sector?