Biotech Values

[mcbio]

BMRN—Matt Herper opines on GT data in hemophilia-A

Some of my own questions and caveats I read from others are:

1. What is the overall impact on the other GT players focused on hemophilia A? I'm thinking in particular about ONCE. Are they too far behind in that they haven't even entered the clinic yet? Or will there still be a chance to differentiate from BMRN or at least room for other players?

2. A few questions I read were why there were still bleeding episodes despite the sharp rise in factor VIII levels in most patients. Is that a concern? Perhaps an angle where others could differentiate down the road?

3. Some questioned if it was too quick and too rapid of a rise in factor VIII levels. Could these continue to go even higher over time? And might this actually lead to LT safety issues (think thrombocytopenia was one example I saw mentioned)?