Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying)
Mucopolysaccharidosis Type 3
Explore 3 assets in Mucopolysaccharidosis Type 3 →Floatz Rating
B59/100
Confidence
Indicative
Sponsor
Ultragenyx Pharmaceutical Inc
Modality
gene_therapy
Development Phase
Phase 2/3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
44Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04088734CT.gov Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease | — | Terminated | — | — | — |
NCT02716246CT.gov Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | — | 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 · Mucopolysaccharidosis Type 3
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying) (this asset) | Ultragenyx Pharmaceutical Inc | P2/3 | B · 59 |
| Amidase, Sulfoglucosamine Sulf- | Shire | P2 | B |
| Hydroxyurea | Masonic Cancer Center, University of Minnesota | P2 | B |
| Alemtuzumab | Masonic Cancer Center, University of Minnesota | P2 | B |
| Melphalan | Masonic Cancer Center, University of Minnesota | P2 | B |
| Clofarabine | Masonic Cancer Center, University of Minnesota | P2 | B |
| Ambroxol | Ozlem Goker-Alpan | P2/3 | B |
| Hgt-1410 | Shire | P2 | CCC |
| Duoc-01 | Joanne Kurtzberg, MD | P1 | CC |
Other indications for Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying)
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Mucopolysaccharidosis Type 3A | — | P2/3 | B · 59 |
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 (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying) in Mucopolysaccharidosis Type 3. 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-synthetic-adeno-associated-virus-9-vector-scaav9u1ahsgsh-human-n-sulfoglucosamine-sulfohydrolase-specifying-mucopolysaccharidosis-type-3
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