What challenges do hemophilia gene therapy need to overcome to achieve the dream of curing patients?

source: | author:webadmin | release time: 2020-07-16 | views:64

For every patient with serious diseases, to be cured is their ultimate dream!
Hemophilia patients are no exception. When the cascade of controlling coagulation was clarified in 1964, it was optimistic that the goal of curing hemophilia would be achieved within five years. Many 5 years have passed, although the treatment of hemophilia has made great progress, but the curative treatment still has not appeared.
However, since the successful cloning and expression of coagulation factor VIII (FVII) and IX (fix) genes, the development of hemophilia curative therapy for 35 years is expected to end. Valoctocogene roxaparvovec, a bio Marin based gene therapy for hemophilia A, may be approved by FDA this summer as the first gene therapy for hemophilia A on the market. It can provide significant protection for some hemophilia patients, and achieve the effect of functional cure.
As the new era of hemophilia treatment is approaching, Dr. Glenn Pierce, vice president of the World Hemophilia Alliance (WFH), wrote an article in haemophilia magazine, reviewing the development history of hemophilia gene therapy, and expounding our attitude towards hemophilia gene therapy.

In the past 30 years, gene therapy has made significant progress in enabling hemophilia A and hemophilia B patients to obtain near normal or normal coagulation factor activity. However, there are still many questions about its efficacy and safety.

Dr. Pierce said embracing gene therapy as a medical model for "curing" hemophilia means recognizing the uncertainty of gene therapy, and pursuing the answers to these questions with an uncompromising spirit and unremitting efforts. This will run through the process that the first generation of hemophilia gene therapy is gradually applied in more patients.