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Hemophilia (Hemophilia) is a type of inherited bleeding disorders in which patients suffer from severe coagulation disorders due to the lack of certain coagulation factors in the blood.
Hemophilia A, Hemophilia B, and Hemophilia C
The overwhelming majority of hemophilia types A and B are X chromosome recessive inherited diseases.
Due to insufficient coagulation factors, patients with hemophilia are prone to bleeding and difficult to coagulate.
For hemophilia patients, gene therapy is almost the only effective treatment
November 18, 2021, the University of Pennsylvania and Spark Therapeutics researchers in the "New England Journal of Medicine" (NEJM) published a report entitled: Clinical Trial Results Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A 1-2 period
Spark Therapeutics
In this phase 1-2 clinical trial, the research team injected SPK-8011 with adeno-associated virus type 5 (AAV-5) as a vector for 18 patients with hemophilia A to evaluate the safety of SPK-8011 Sexuality and preliminary efficacy, as well as the expression and persistence of coagulation factor Ⅷ
The median observation time was 36.
The experiment was divided into four different doses, which were 5×10E11 viral vector genomes (vg) per kilogram of body weight, and 1×10E12, 1.
Among the 18 treated hemophilia patients, 16 patients had long-term stable expression of coagulation factor Ⅷ, and the annual bleeding event was reduced by 91.
In addition, the number of annual coagulation factor Ⅷ injections decreased from a median of 57.
A total of 33 treatment-related adverse events occurred in 8 participants, of which 17 were related to AAV viral vectors, including 1 serious adverse event, and the other 16 were related to glucocorticoids
In summary, this phase 1-2 clinical trial showed that 16 of the 18 hemophilia A patients who received this AAV gene therapy successfully continued to express coagulation factor Ⅷ, and significantly reduced bleeding events, and there was no Report major safety issues
Original source:
Original source:Lindsey A.
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
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