WHile Yes they do have a GT program and its likely the bulk of their valuation I would call them a DMD company that has a GT program. I vaguely remember the story on the founding of the company. If anyone is curious on the founding/history here is a little background article.
If the stock trades at the valuation suggested I personally won't consider it will just watch but not invest (way to high if the bulk is in fact for their GT program). Guess it helps to have JP Morgan friends?
Its quite admirable what the father is doing but keep in mind doesn't always lead to success (Lysogene CEO tried/is trying the same in MPS IIIA) others with varying degrees of success UTHR (daughter never on Remodulin last I was aware), FOLD (Pompe not yet approved though daughter taking ERT), others?
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