Devafidugene Civaparvovec
Mucopolysaccharidosis Type 1
Explore 5 assets in Mucopolysaccharidosis Type 1 →Floatz Rating
CCC48/100
Confidence
Indicative
Sponsor
Sangamo 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 |
|---|---|---|---|---|---|
NCT04628871CT.gov Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX | — | Active Not Recruiting | — | — | — |
NCT02702115CT.gov Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I | — | Terminated | — | — | — |
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 1
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Devafidugene Civaparvovec (this asset) | Sangamo Therapeutics | P1/2 | CCC · 48 |
| Aldurazyme | Genzyme, a Sanofi Company | P4 | A |
| Iduronidase, .Alpha.-L-(8-Histidine) (Human) | University of California, San Francisco | P4 | BBB |
| Valanafusp Alfa | ArmaGen, Inc | P1/2 | BB |
| Rhidu | Genzyme, a Sanofi Company | P3 | BB |
| Cyclophosphamide | Masonic Cancer Center, University of Minnesota | P2 | B |
| Busulfanum | Masonic Cancer Center, University of Minnesota | P2 | B |
| Equine Atg | Masonic Cancer Center, University of Minnesota | P2 | B |
| Serostim | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | P2/3 | B |
+7 more in the Mucopolysaccharidosis Type 1 cohort
Other indications for Devafidugene Civaparvovec
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Hemophilia B | — | Unk | D · 30 |
| Mucopolysaccharidosis Type 2 | — | Unk | D · 30 |
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. Devafidugene Civaparvovec in Mucopolysaccharidosis Type 1. 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/devafidugene-civaparvovec-mucopolysaccharidosis-type-1
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