RNA therapeutics for hemophilia
| Agent . | Mechanism . | Route, frequency . | Trial status . | Target group . |
|---|---|---|---|---|
| siRNA antithrombin fitusiran | AT mRNA silencing | Subcutaneous, monthly or bimonthly | Phase 1, 2, and 3 trials | HA and HB with or without inhibitors |
| siRNA PS | PS mRNA silencing | Subcutaneous | No trials to date | HA and HB with or without inhibitors |
| siRNA HCII | HCII mRNA silencing | Subcutaneous | No trials to date | HA mouse studies |
| mRNA gene therapy | Lipid-encapsulated mRNA by intracellular delivery, or transposon | IV single dose | No trials to date | HA and HB |
| Gene editing ZFN or CRISPR/Cas9 | Nuclease-driven homology-directed gene repair | IV single dose | No trials to date | HA and HB |
| Agent . | Mechanism . | Route, frequency . | Trial status . | Target group . |
|---|---|---|---|---|
| siRNA antithrombin fitusiran | AT mRNA silencing | Subcutaneous, monthly or bimonthly | Phase 1, 2, and 3 trials | HA and HB with or without inhibitors |
| siRNA PS | PS mRNA silencing | Subcutaneous | No trials to date | HA and HB with or without inhibitors |
| siRNA HCII | HCII mRNA silencing | Subcutaneous | No trials to date | HA mouse studies |
| mRNA gene therapy | Lipid-encapsulated mRNA by intracellular delivery, or transposon | IV single dose | No trials to date | HA and HB |
| Gene editing ZFN or CRISPR/Cas9 | Nuclease-driven homology-directed gene repair | IV single dose | No trials to date | HA and HB |