Focus on Fidanako-Ella Parvowitz gene therapy: What diseases can be overcome

Fidanacogene elaparvovec (fidanacogene elaparvovec) gene therapy is suitable for adults with a specific type of hemophilia B (congenital factor IX deficiency). Specifically, the therapy targets the following groups:

The first is adult patients who have been diagnosed with moderate to severe B hemophilia and are currently receiving preventive treatment with factor IX; the second is patients who have experienced life-threatening bleeding events or have repeated and severe spontaneous bleeding episodes. In addition, patients need to confirm the absence of neutralizing antibodies against the capsid of adeno-associated virus serotype Rh74var (AAVRh74var) through an FDA-approved test to ensure the safety and effectiveness of treatment.

This therapy uses genetic engineering technology to deliver functional Factor IX genes to the patient's liver cells, allowing them to independently synthesize Factor IX protein, thereby reducing or eliminating bleeding events and significantly improving the patient's quality of life. It can achieve long-term effects with a single treatment, providing new treatment options and hope for patients with hemophilia B. Adeno-associated virus (AAV) vector is used to deliver the highly active human coagulation factorIX gene (FIX-Padua) to the patient's liver cells. After gene integration, liver cells can continue to synthesize FIX protein independently, reducing or eliminating dependence on external coagulation factor infusion.

Phase III clinical trial (BENEGENE-2) showed that patients’ annualized bleeding rate (ABR) was reduced by 71%. The annualized coagulation factor transfusion rate was reduced by 92%. A single intravenous infusion can achieve long-term efficacy, avoid frequent injections of coagulation factors, and significantly improve patients' quality of life.

In clinical trials, some patients developed an immune response to the liver cells targeted by the therapy, but this could be effectively controlled with steroid treatment.

Long-term safety requires further monitoring, and current data show no treatment-related deaths.

Reference materials:https://en.wikipedia.org/wiki/Fidanacogene_elaparvovec