Dna (Recombinant Adeno-Associated Virus 2 Vector Lys-Saf302 Human N-Sulfoglucosamine Sulfohydrolase Cdna-Specifying)
Mucopolysaccharidosis Type 3A
Explore 5 assets in Mucopolysaccharidosis Type 3A →Floatz Rating
B57/100
Confidence
Indicative
Sponsor
LYSOGENE
Modality
gene_therapy
Development Phase
Phase 2/3
Status
Dormant
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
42Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT03612869CT.gov Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) | — | Unknown | — | — | — |
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 3A
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Dna (Recombinant Adeno-Associated Virus 2 Vector Lys-Saf302 Human N-Sulfoglucosamine Sulfohydrolase Cdna-Specifying) (this asset) | LYSOGENE | P2/3 | B · 57 |
| Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying) | Ultragenyx Pharmaceutical Inc | 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 |
| Autologous Cd34+ Cells Transduced With Tns9.3.55 | University of Manchester | P1/2 | CCC |
| dnl126 | Denali Therapeutics Inc. | P1/2 | CCC |
| gc1130a | GC Biopharma Corp | P1 | NR |
| Sobi003 | Swedish Orphan Biovitrum | 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
- Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying)Ultragenyx Pharmaceutical IncB
- Saf-301LYSOGENECCC
- Autologous Cd34+ Cells Transduced With A Lentiviral Vector Containing The Human Sgsh GeneUniversity of ManchesterCCC
- Autologous Cd34+ Cells Transduced With Tns9.3.55University of ManchesterCCC
- dnl126Denali Therapeutics Inc.CCC
Citation
Floatz Terminal. Dna (Recombinant Adeno-Associated Virus 2 Vector Lys-Saf302 Human N-Sulfoglucosamine Sulfohydrolase Cdna-Specifying) 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/dna-recombinant-adeno-associated-virus-2-vector-lys-saf302-human-n-sulfoglucosamine-sulfohydrolase-cdna-specifying-mucopolysaccharidosis-type-3a
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