Hepastem
Urea Cycle Disorder
Floatz Rating
CCC52/100
Confidence
Indicative
Sponsor
Cellaion SA
Modality
UNKNOWN
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 |
|---|---|---|---|---|---|
NCT01765283CT.gov Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Urea Cycle Disorder
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Hepastem (this asset) | Cellaion SA | P1/2 | CCC · 52 |
| Rifaquizinone | TenNor Therapeutics (Suzhou) Limited | P1/2 | CCC |
| Glycerol Phenylbutyrate | Amgen | P1 | CC |
Other indications for Hepastem
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Metabolic Dysfunction-Associated Steatohepatitis | — | P1/2 | CCC · 50 |
| Crigler-Najjar Syndrome | — | P1/2 | NR |
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. Hepastem in Urea Cycle Disorder. 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/hepastem-urea-cycle-disorder
Are you the sponsor?
Submit your data room for a Verified Rating. The public-data rating remains visible alongside.
Contact →