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BioMarin selloff pressures other AAV gene therapy developers

August 19, 2020

UniQure (QURE -4.6%) and REGENXBIO (RGNX -4.1%) are feeling BioMarin Pharmaceutical’s (BMRN -35.0%) pain after the FDA rejected its marketing application for hemophilia A treatment valoctogene roxaparvovec, a gene therapy based on a vector called adeno-associated virus type 5 (AAV5).

QURE is also developing AA5 gene therapies while RGNX is involved with AAV types 7,8, 9 and 10.

Recombinant replication-defective adenoviruses are popular vectors for delivering gene therapies since they infect most cell types, are very efficient with no requirements for cell division and are relatively inexpensive to engineer.

https://seekingalpha.com/news/3607165-biomarin-selloff-pressures-aav-gene-therapy-developers

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