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.