Floatz Rating
CC43/100
Confidence
Indicative
Sponsor
Joanne Kurtzberg, MD
Modality
Cell therapy
Development Phase
Phase 1
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
28Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT02254863CT.gov UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells | — | 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 |
|---|---|---|---|
| Duoc-01 (this asset) | Joanne Kurtzberg, MD | P1 | CC · 43 |
| Amidase, Sulfoglucosamine Sulf- | Shire | P2 | B |
| Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying) | Ultragenyx Pharmaceutical Inc | P2/3 | B |
| Hydroxyurea | 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 |
| Alemtuzumab | Masonic Cancer Center, University of Minnesota | P2 | B |
| Hgt-1410 | Shire | P2 | CCC |
Other indications for Duoc-01
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Primary Progressive Multiple Sclerosis | — | P1 | CC · 46 |
| Mucopolysaccharidosis Type 2 | — | P1 | CC · 43 |
| Juvenile Neuronal Ceroid Lipofuscinosis | — | P1 | 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
- Amidase, Sulfoglucosamine Sulf-ShireB
- Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying)Ultragenyx Pharmaceutical IncB
- HydroxyureaMasonic Cancer Center, University of MinnesotaB
- MelphalanMasonic Cancer Center, University of MinnesotaB
- ClofarabineMasonic Cancer Center, University of MinnesotaB
Citation
Floatz Terminal. Duoc-01 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/duoc-01-mucopolysaccharidosis-type-3
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