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 (Late-onset)
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
11 Active assets in the Pompe Disease (Late-onset) cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Alglucosidase Alfa Erasmus Medical Center | Phase 4 | BBB |
Cipaglucosidase Alfa-ATGA Amicus Therapeutics | Phase 3 | BB |
Avalglucosidase Alfa Iris Plug | Phase 4 | BB |
Miglustat Amicus Therapeutics | Phase 3 | B |
at2221 Amicus Therapeutics | Phase 3 | B |
View all 13 →
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:
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