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recombinant adeno-associated viral (AAV) vector that contains a bio-engineered capsid (AAV-Spark100) and a codon-optimized expression cassette to drive expression of a secretable form of human acid a-glucosidase (GAA)
Trade Name not avaiable
Orphan Indication Pompe Disease
USA Market Approval
USA Designation Date 01-02-2019
Sponsor Spark Therapeutics, Inc.
3737 Market Street, Suite 1300
Philadelphia, Pennsylvania, 19104
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