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Crispr Therapeutics AG, a start-up formed by the co-inventor of the CRISPR/Cas9 genome-editing technology, closed a $25 million series A round that will fund the translation of this hugely popular approach from the academic lab to the clinic.

"The worst disease you've never heard of." That's how patient advocacy and nonprofit group Debra (Dystrophic Epidermolysis Bullosa Research Association of America) refer to epidermolysis bullosa (EB), a rare genetic disorder characterized by fragile skin that it often leaves its sufferers with chronic open wounds and blisters.

About 13 years ago, when Amber Salzman's son and two nephews were diagnosed with adrenoleukodystrophy (ALD), a genetic brain disorder, she didn't know much about rare diseases at all. That's changed for the CEO of Paris-based AAVLife, which – with $12 million in series A money – is taking aim at another: Friedreich's ataxia (FA).
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