Replies to post #217941 on Biotech Values

[ghmm]

Thanks for the information!

Yes both are intended for both FDA and EMA filings.
QURE's isn't posted yet but here are (both) BMRN's (second is low dose and will have 3rd with patients with existing AAV5 antibodies
https://clinicaltrials.gov/ct2/show/study/NCT03370913
https://clinicaltrials.gov/ct2/show/study/NCT03392974


With how much factor level increases I can't imagine any of these (along with Sparks for that matter) not having a (highly) significant impact on bleeds.

Should add that BMRN is measuring baseline bleeds by patients historical records. Both companies are doing open label trials.