Floatz Rating
B59/100
Confidence
Indicative
Sponsor
Spark Therapeutics, Inc.
Modality
gene_therapy
Development Phase
Phase 3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
52Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT06297486CT.gov Study of a Gene Therapy Treatment for Hemophilia A | — | Withdrawn | — | — | — |
NCT03432520CT.gov Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A | — | Active Not Recruiting | — | — | — |
NCT03003533CT.gov A Gene Transfer Study for Hemophilia A | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
73High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Hemophilia A
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Dirloctocogene Samoparvovec (this asset) | Spark Therapeutics, Inc. | P3 | B · 59 |
| Turoctocog Alfa | Novo Nordisk A/S | P4 | A |
| Eptacog Alfa (Activated) | AryoGen Pharmed Co. | P4 | A |
| Rurioctocog Alfa Pegol | Takeda | P4 | BBB |
| bay94-9027 | Bayer | P4 | BBB |
| N8-GP | Novo Nordisk A/S | P3 | BBB |
| Immunoglobulin G4 [230-Proline,De-449-Lysine], Anti-(Human Blood-Coagulation Factor Ix)(Human Clone 0365-0001-9985 ?4-Chain), Disulfide With Human Clone 0365-0001-9985 ?-Chain, (228?226?),(231?229?)-Bis(Disulfide) With Anti-(Human Blood-Coagulation Factor X) Immunoglobulin G4 [228-Proline,405-Leucine,409-Lysine,De-447-Lysine] (Human Clone 0365-0001-8174 ?4-Chain) Disulfide With Human Clone 0365-0001-8174 ?-Chain | Novo Nordisk A/S | P3 | BBB |
| Emicizumab | Emory University | P4 | BBB |
| Fitusiran | Sanofi | P4 | BBB |
+36 more in the Hemophilia A cohort
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. Dirloctocogene Samoparvovec in Hemophilia A. 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/dirloctocogene-samoparvovec-hemophilia-a
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