EMBARK.....As in "Here we go! All aboard! The journey begins." | SRPT Message Board Posts


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Msg  14489 of 14500  at  11/15/2022 3:08:08 PM  by

schmiggins


 In response to msg 14488 by  schmiggins
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Re: EMBARK.....As in "Here we go! All aboard! The journey begins."

Doug Ingram -- Chief Executive Officer

I apologize, Hartaj -- I'm sorry, Louise. I think, Louise is cutting out a little bit. I'll just repeat less artfully what I think Louise was saying, which was -- look, first I will repeat what Louise said, firmly, we love rh74. We are excited.

That is really the platform right now for our gene therapy focused on neuromuscular and neuro and frankly, cardiomyopathies as well. And there is no -- from our perspective, at least, there is no better current serotype from a safety perspective, coupled with a tropism perspective than rh74. So this isn't about any negativity on rh74. We are thrilled with it.

Obviously, there is an enormous amount of future value to patients and to the breadth of patients if one can find ways to decrease dose, which would make therapies more affordable around the world, etc., and probably increase safety and the like. We have a long way to go. We haven't dosed any patients or even healthy human volunteers with MyoAAV, but at a preclinical and animal model perspective, we're seeing some things that really excite us about the potential at least for MyoAAV. Could it be literally an order of magnitude more tropic than any of the current serotypes, which would really be extraordinarily meaningful.

It would be meaningful around the world in places like India and Africa and other places where we're going to have to find a different kind of cost of goods to really have the full breadth of the benefits of our therapies, including 9001. And it probably takes us to many other kinds of disease states that would be challenging today. At these cost of goods with gene therapies currently today, there are probably certain kinds of ultra-rare diseases that become very difficult. And that's -- frankly, that's not acceptable.

That's tragic. If we have the ability to treat even very, very rare diseases, we ought to be thinking about ways to do it. And MyoAAV -- if not MyoAAV, maybe some other capsid, but currently the best thing we've seen and we've seen everything, is MyoAAV. It could really be a transformative next-generation approach to gene therapy.

So we are in love with rh74, but we are excited about the potential of MyoAAV down the road.


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