Autologous Cd34+ Cells Transduced With Tns9.3.55
Mucopolysaccharidosis Type 3A
Explore 5 assets in Mucopolysaccharidosis Type 3A →Floatz Rating
CCC48/100
Confidence
Indicative
Sponsor
University of Manchester
Modality
unclassified
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.
33Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04201405CT.gov Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95Moderate confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Mucopolysaccharidosis Type 3A
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Autologous Cd34+ Cells Transduced With Tns9.3.55 (this asset) | University of Manchester | P1/2 | CCC · 48 |
| Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying) | Ultragenyx Pharmaceutical Inc | P2/3 | B |
| Dna (Recombinant Adeno-Associated Virus 2 Vector Lys-Saf302 Human N-Sulfoglucosamine Sulfohydrolase Cdna-Specifying) | LYSOGENE | P2/3 | B |
| Saf-301 | LYSOGENE | P1/2 | CCC |
| Autologous Cd34+ Cells Transduced With A Lentiviral Vector Containing The Human Sgsh Gene | University of Manchester | P1/2 | CCC |
| dnl126 | Denali Therapeutics Inc. | P1/2 | CCC |
| Sobi003 | Swedish Orphan Biovitrum | P1/2 | NR |
| gc1130a | GC Biopharma Corp | P1 | NR |
Other indications for Autologous Cd34+ Cells Transduced With Tns9.3.55
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Severe Combined Immunodeficiency | — | P1/2 | BB · 64 |
| Beta-Thalassemia Major | — | P1/2 | CCC · 54 |
| Sickle Cell Disease | — | P2 | CCC · 52 |
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
- Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying)Ultragenyx Pharmaceutical IncB
- Dna (Recombinant Adeno-Associated Virus 2 Vector Lys-Saf302 Human N-Sulfoglucosamine Sulfohydrolase Cdna-Specifying)LYSOGENEB
- Saf-301LYSOGENECCC
- Autologous Cd34+ Cells Transduced With A Lentiviral Vector Containing The Human Sgsh GeneUniversity of ManchesterCCC
- dnl126Denali Therapeutics Inc.CCC
Citation
Floatz Terminal. Autologous Cd34+ Cells Transduced With Tns9.3.55 in Mucopolysaccharidosis Type 3A. 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/autologous-cd34-cells-transduced-with-tns9355-mucopolysaccharidosis-type-3a
Are you the sponsor?
Submit your data room for a Verified Rating. The public-data rating remains visible alongside.
Contact →