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)
Lysosomal Storage Diseases
Spark Therapeutics, Inc.gene_therapyPhase 1/2Active
CCC
48/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 33/ 100
- Competitive Position
- 95/ 100
Competitive context
1 Active assets in the Lysosomal Storage Diseases cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
flt190 Spur Therapeutics | Phase 1/2 | CCC |
IDURONIDASE, .ALPHA.-L-(8-HISTIDINE) (HUMAN) Patricia I. Dickson, M.D. | Phase 1 | CC |
Lsd University of Chicago | Early Phase 1 | C |
Rimiducid | D | |
Clofarabine Masonic Cancer Center, University of Minnesota | N/A | D |
View all 10 →
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:
glycogen storage disease due to acid maltase deficiency, late-onsetDormant
CC
Indicative ·
47 / 100
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