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.
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