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I suspect that right now, for a company going into rare disease, the juice isnt worth the squeeze. read: I dont see an easy path to profitability for companies focused on rare disease

While I agree in general, suggest you check out BCRX which got recent approval for their drug Orladeyo and seeing good results with their Factor D inhibitor. Also, Alexion (ALXN) was a company that did well with rare diseases for many years. Of course, these are not into gene therapy if that is what you meant.
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