Seeking Alpha: Bluebird Takes Flight On Very Promising, Very Early Data

Investors have had good reason to be skeptical of Bluebird Bio (BLUE). Gene therapy was the "next big thing" in the mid-to-late 1990s and while it is too negative to say it has thus far produced almost nothing of value, it certainly hasn't lived up to "the medicine of tomorrow, today!" hype. While I was bullish on Bluebird Bio back in February, I wasn't entirely surprised to see the shares of this very early-stage, high speculative stock sell off when the biotech sector started wheezing shortly thereafter.

Now, investors have some enticing data to mull over. Over the weekend, Bluebird Bio presented data on two patients in its HGB-205 study of LentiGlobin gene therapy in beta-thalassemia, and while two bluebirds may not make a summer, the surprisingly strong response in these two patients suggests that Bluebird may just have something big on its hands.

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Bluebird Takes Flight On Very Promising, Very Early Data

Seeking Alpha: PTC Therapeutics Looks Like A High-Risk Play On Rare Diseases

With money and momentum flowing out of the biotech sector, there are going to be a lot of beaten-down diamonds in the rough amidst the wreckage. I'm not entirely convinced PTC Therapeutics (PTCT) is a diamond, or at least not yet. While the company's lead compound ataluren has a lot of promise, there some serious questions and concerns about the drug. Bulls are right about the high-end potential here, but prior trial failures shouldn't be ignored.

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PTC Therapeutics Looks Like A High-Risk Play On Rare Diseases