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DNA (recombinant adeno-associated virus vector AAV-Spark100-GAA human liver-specific expression cassette including human lysosomal ?-glucosidase codon-optimized gene GAA plus adeno-associated virus 2 inverted terminal repeat flanks)

Pompe Disease

Spark Therapeutics, Inc.gene_therapyPhase 1/2Active
CC
47/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
33/ 100
Competitive Position
89/ 100

Competitive context

8 Active assets in the Pompe Disease cohort.

Asset / SponsorPhaseRating
Avalglucosidase Alfa
Genzyme, a Sanofi Company
Phase 4A
Avalglucosidase Alfa
Genzyme, a Sanofi Company
Phase 4A
Alglucosidase Alfa
Erasmus Medical Center
Phase 4A
Albuterol
National Taiwan University Hospital
Phase 4BBB
IMMUNOGLOBULIN G 1 (HUMAN-MOUSE MONOCLONAL IDEC-C2B8 .GAMMA.1-CHAIN ANTI-HUMAN ANTIGEN CD 20), DISULFIDE WITH HUMAN-MOUSE MONOCLONAL IDEC-C2B8 .KAPPA.-CHAIN, DIMER
University of Florida
Phase 4BB

View all 28 →

Same molecule, other indications

DNA (recombinant adeno-associated virus vector AAV-Spark100-GAA human liver-specific expression cassette including human lysosomal ?-glucosidase codon-optimized gene GAA plus adeno-associated virus 2 inverted terminal repeat flanks) is also rated in:

lysosomal storage diseaseDormant
CCC
Indicative ·
48 / 100
View asset →
glycogen storage disease due to acid maltase deficiency, late-onsetDormant
CC
Indicative ·
47 / 100
View asset →

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