Floatz Rating
CC45/100
Confidence
Indicative
Sponsor
Children's Hospital of Fudan University
Modality
UNKNOWN
Development Phase
Early Phase 1
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
33High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT06479616CT.gov A Long-term Follow-up Study in Participants Who Received CS-101 | — | Recruiting | — | — | — |
NCT06328764CT.gov CS-101 in Patients With β-thalassemia | — | Enrolling By Invitation | — | — | — |
NCT06065189CT.gov Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major | — | Completed | — | — | — |
NCT06024876CT.gov A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
81High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Beta Thalassemia
+42 more in the Beta Thalassemia cohort
Other indications for cs-101
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Beta-Thalassemia Major | — | P2 | B · 60 |
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
- Desferrioxamine BAin Shams UniversityBBB
- DeferasiroxKevin H.M. Kuo, MD, MSc, FRCPCBBB
- Autologous Cd34+ Cells Isolated From Mobilised Peripheral Blood By Positive Selection, Modified By Crispr/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats/Crispr-Associated Protein 9) Mediated Gene Editing Consisting Of A Guide Rna (Grna) Introduced Transiently As Ribonucleoprotein (Rnp) Complex, Targeting The Erythroid Lineage-Specific Enhancer Region Of Bcl11A (B-Cell Lymphoma/Leukemia 11A). The Site-Specific Cleavage By Cas9 Forms A Double Strand Break (Dsb), Which Is Subsequently Repaired By Nonhomologous End-Joining (Nhej), Leading To The Transcriptional Repression Of Bcl11A, A Repressor Of ?-Globin Gene TranscriptionDaniel BauerBB
- HydroxyureaNational Heart, Lung, and Blood Institute (NHLBI)BB
- DeferiproneCarelon ResearchBB
Citation
Floatz Terminal. cs-101 in Beta Thalassemia. 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/cs-101-beta-thalassemia
Are you the sponsor?
Submit your data room for a Verified Rating. The public-data rating remains visible alongside.
Contact →