Monthly Roundup: April 2024
Presented by: Wolfgang A. Miesbach, MD, PhD
The six-year follow-up report on the AMT-060 gene therapy for hemophilia B, presented at the EAHAD Congress 2024, highlights its long-term safety, efficacy, and durability. Researchers used an AAV5 vector with wild-type Factor IX to treat two dose groups in a Phase 1 trial. Patients in the higher-dose group (Cohort 2) maintained stable Factor IX levels (5.8% at six years), with no patients returning to prophylaxis, or experiencing spontaneous or joint bleeds after four and five years, respectively. The lower-dose group (Cohort 1) showed slightly lower Factor IX levels (4.6%) but still reduced prophylaxis use significantly. No new safety issues occurred during the six years. These results demonstrate that AMT-060 provides durable factor expression, reduces treatment needs, and supports long-term bleeding protection. Researchers will continue monitoring patients for up to 15 years to assess ongoing efficacy and safety.
