Spark Therapeutics Stock Forum

The safety risks of AAV are so persistent that last year Nat Bio's aAV8/9 animal trials saw significant integration into the genome, and now BMRN's AAV5 has been hammered and clinically suspended, feeling increasingly firm (at least for existing wild serotypes).
BMN307 is an aAV5-based gene therapy targeting the phenylalanine hydrogenase gene for the treatment of phenylketonuria, currently undergoing phase 1/2 clinical trials. However long before clinical data showed that high dose group of 7 has 6 in rat liver tumor, and integrated into the genome of evidence is clear, the mouse is the most terrible of lesions, and 24 weeks are not observed in 52 weeks discovered tumorigenic anatomy, this led to Biomarin pushed to the first, after finding that these risks are clinical...
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