Hemophilia A   Hemophilia B
Feature Curative Treatment Valoctocogene Roxaparvovec Emicizumab EHL FVIII Etranacogene Dezaparvovec EHL FIX
One-time treatment yes yes no no yes no
Given prenatally or early neonate yes no yes yes no yes
Option for all patients yes no yes* no no no
Normal factor activity following treatment yes no no possible no possible
Hemostatic factor activity for all treated patients yes no yes$ yes$ no yes$
Predictable response yes no yes yes no yes
Sustained for a lifetime (80+ years) yes no yes yes no yes
Mean Annualized Bleeding Rate 0 1.2 2.5 0.7 1.5 1.6
Eliminate need for factor replacement for all patients in all situations yes no no no no no
Risk for unintended genome insertion no yes no no yes no
Risk for malignancy no unknown no no unknown no
Risk for hepatic inflammation no yes no no yes no
Risk for hepatic failure with vector& no yes no no yes no
Risk for TMA with vector& no yes yes# no yes no
Risk for DRG toxicity with vector& no yes no no yes no
Risk for other toxicity with vector& no yes no no yes no
Risk of reduced bone density no unknown unknown unknown unknown unknown