Floatz Rating
CCC52/100
Confidence
Indicative
Sponsor
UniQure Biopharma B.V.
Modality
gene_therapy
Development Phase
Phase 1/2
Status
Dormant
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
37Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT03300453CT.gov Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome | — | Completed | — | — | — |
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 3B
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| AAV-2 (this asset) | UniQure Biopharma B.V. | P1/2 | CCC · 52 |
| Lesinidase Alfa | Alexion Pharmaceuticals, Inc. | P1/2 | CCC |
| Raav9.Cmv.Hnaglu | Abeona Therapeutics, Inc | P1/2 | CCC |
| ax 250 | Allievex Corporation | P4 | NR |
| Tralesinidase Alfa | Spruce Biosciences | P3 | NR |
Other indications for AAV-2
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Hemophilia A | — | P1/2 | CC · 43 |
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. AAV-2 in Mucopolysaccharidosis Type 3B. 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/aav-2-mucopolysaccharidosis-type-3b
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