Replies to post #204095 on Biotech Values

[ghmm]

Any hard numbers out there on estimates of number of patients AAV5 vector could work in versus AAV8? Also, curious what competition looks like for QURE in hemophilia B with AAV5 vector specifically if you know.

This analysts report estimates 40%. I don't know if Spark has revealed their screen fail rate which would help to confirm this number. For UniQure I believe I've seen a figure of 90% should be eligible (10% having anti-aav5 antibodies) but can't recall if that was company figure or elsewhere. They have said no patients (small numbers) in the Hemophilia trial that have been screened had anti-aav5 antibodies. It should be cautioned that perhaps someone develops a way around this (like UniQure working on retreatment) still nothing I am aware is far along.
https://twitter.com/Liquid_Biopsy_/status/761164927809753088/photo/1
https://pbs.twimg.com/media/CpAzajVWcAA_abK.jpg

For other Hemophilia B work in AAV5. BMRN hasn't announced a program but have said should be able to translate work done for their A program to a B program. There are other Hemophilia B programs like DMTX (I believe its a different vector) too but sorry don't have a list handy.

[smittypa]

According to the Wedbush analyst, about 40% of Hemo b patients will be unable to take SPK9001, due to antibodies. Average age of the (4) Spark Hemo B patients was 35.5, while the (5)QURE patients averaged age 60, although it's much too early to tell if age matters, in terms of efficacy. While the ONCE results are great, durability of response is still a big open question. Watch for an update on the first (6 patients) cohort of the UCSF AAV2-GDNF Parkinson's trial, on Sept. 12, in Switzerland. QURE owns all rights to this university sponsored trial. First cohort took 2.5 years to complete, but the second cohort enrolled in six months, hopefully a good sign.