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 Disease
Explore 2 assets in Lysosomal Storage Disease →Floatz Rating
CCC48/100
Confidence
Indicative
Sponsor
Spark Therapeutics, Inc.
Modality
gene_therapy
Development Phase
Phase 1/2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
33Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04093349CT.gov A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Lysosomal Storage Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| 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) (this asset) | Spark Therapeutics, Inc. | P1/2 | CCC · 48 |
| flt190 | Spur Therapeutics | P1/2 | CCC |
| Iduronidase, .Alpha.-L-(8-Histidine) (Human) | Patricia I. Dickson, M.D. | P1 | CC |
| Lsd | University of Chicago | Early P1 | C |
| Cyclosporin A | Masonic Cancer Center, University of Minnesota | N/A | D |
| Clofarabine | Masonic Cancer Center, University of Minnesota | N/A | D |
| Melphalan | Masonic Cancer Center, University of Minnesota | N/A | D |
| Campath-1H | Masonic Cancer Center, University of Minnesota | N/A | D |
| Ald-101 | Aldagen | P3 | NR |
Other indications for 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)
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Glycogen Storage Disease II | — | P1/2 | CC · 47 |
| Glycogen Storage Disease Due To Acid Maltase Deficiency, Late-Onset | — | P1/2 | CC · 47 |
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. 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) in Lysosomal Storage Disease. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/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-disease
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