Uniqure Comp | SGMO Message Board Posts

Sangamo Therapeutics, Inc.

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Msg  141803 of 144030  at  2/18/2020 5:56:27 AM  by


 In response to msg 141793 by  rynotheknife
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Re: Uniqure Comp

rynotheknife msg 141793:

"hemo a will cash flow about $100 million a year"... yeah that's the bull thesis and I'd love to agree...

but I now think that the market, John Q Public, and the FDA all think that bleeding is the Hemo A goal line...not factor durability

and if that's true, clearly there's a huge first mover advantage to ValRox... again "if'


How do you think the maintenance of reduced bleeding will be achieved in hemophilia A patients if FVIII levels are not maintained?

Do you think that hemophilia patients are not interested in factor durability?

Do you think that hematologists are not aware of the implications of durability?

Do you think insurance companies are not interested in durability?

Above all, are you suggesting that the FDA is only interested in the initial bleeding events and not maintenance of factor levels?

Valrox (BMN 270) may well get approved, but if SB-525 can demonstrate durability then Biomarin will ‘have a hell of a fight on their hands’. Do you think Pfizer will stay quiet if SB-525 proves to be a superior treatment?

100% of patients in Sangamo’s high dose cohort (3e13 vg/kg) have had no bleeds to date (from 21 days post dosing). Admittedly n=5, but so far perfect. Onset of efficacy, factor levels and consistency with dosage all suggest that SB-525 may well be a much better treatment option. Durability is a key issue and I think you are naïve if you think the market will not recognise this.


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