Floatz Rating
CC47/100
Confidence
Indicative
Sponsor
4D Molecular Therapeutics
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.
33Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05629559CT.gov 4D-310 in Adults With Fabry Disease and Cardiac Involvement | — | Recruiting | — | — | — |
NCT04519749CT.gov An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Fabry Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Duvalgagene Otiparvovec (this asset) | 4D Molecular Therapeutics | P1/2 | CC · 47 |
| Recombinant Alpha-Galactosidase A | Shire | P4 | A |
| Agalsidase Beta | Sanofi | P4 | A |
| Pegunigalsidase Alfa | Chiesi Italia | P3 | BBB |
| Migalastat | Amicus Therapeutics | P3 | BBB |
| Montelukast | Sanofi | P4 | BB |
| Fexofenadine | Sanofi | P4 | BB |
| Loratadine | Sanofi | P4 | BB |
| Lucerastat | Idorsia Pharmaceuticals Ltd. | P3 | BB |
+28 more in the Fabry Disease cohort
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. Duvalgagene Otiparvovec in Fabry 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/duvalgagene-otiparvovec-fabry-disease
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