adeno-associated viral vector composed of a bioengineered AAV capsid and a codon-optimized expression cassette encoding a high-specific activity variant of human coagulation factor IX | |
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Trade Name | |
Orphan Indication | Hemophilia B |
USA Market Approval | USA |
USA Designation Date | 2015-09-21 00:00:00 |
Sponsor | Spark Therapeutics, Inc.;3737 Market Street, Suite 1300;Philadelphia, Pennsylvania, 19104 |