Specific doses and methods of using Fidanaco-Ella Parvowitz gene therapy

Fidanacogene elaparvovec (fidanacogene elaparvovec) gene therapy is administered through a single intravenous infusion without dose adjustment. Its core mechanism is to use an adeno-associated virus (AAV) vector to deliver the functional coagulation factor IX gene to the patient's liver cells to achieve long-term treatment.该疗法为单剂量治疗，无需根据患者体重或病情严重程度调整剂量。 In the clinical trial, all participants received a single intravenous infusion. The drug introduced the gene encoding the highly active coagulation factor IX variant (FIX-Padua) into liver cells through a viral vector, allowing patients to synthesize coagulation factor IX independently, thereby reducing or eliminating dependence on external coagulation factor infusion.

药物通过静脉注射直接进入血液循环，确保病毒载体能够高效靶向肝细胞。由于基因疗法的特殊性，单次给药后即可实现长期疗效，无需重复治疗或剂量调整。

After the AAVvector carries the FIX gene into the liver cells, the gene is integrated into the host cell genome to achieve continuous expression.肝细胞开始自主合成高活性的FIX-Padua蛋白，显著提升凝血功能。 Clinical trials have shown that after a single treatment, the patient's annualized bleeding rate (ABR) was reduced by 71%, and the annualized coagulation factor infusion rate was reduced by 92%.

Fidanaco - Ella Parvowitz gene therapy is suitable for adult patients 18 years and older with moderate to severe B hemophilia, who must meet one of the following conditions:

正在接受凝血因子IX预防性治疗；

There is a life-threatening bleeding event;

Severe spontaneous bleeding occurs repeatedly.

Patient screening: Patients must pass a test approved by the FDA to confirm that there are no neutralizing antibodies against the AAVRh74var capsid in the body to avoid immune reactions affecting the efficacy.

Some patients may develop symptomsImmune response to AAVvector but can be controlled with steroid treatment. After treatment, patients need to be followed up long-term to evaluate coagulation factor IX expression levels, bleeding events and potential side effects.

fidanacogene elaparvovecGene therapy achieves long-term efficacy through a single treatment, significantly improves the quality of life of patients with B hemophilia, reduces the risk of bleeding and dependence on coagulation factor infusion, and provides patients with new treatment options.

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