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NEJM: SPK-8011 gene therapy for hemophilia A

 Last Update: 2021-12-01
 Source: Internet
 Author: User

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Hemophilia A is a blood clotting factor 8 gene due to defects caused by blood diseases, one person every 5000-10000 men worldwide suffer from type A hemophilia

The researchers recently published the results of clinical studies Spk-8011 treatment of hemophilia A

In the newly conducted phase I-II clinical study, 18 hemophilia A patients participated and received 4 doses of Spk-8011.

Prevent immunization

The average observation time of participants during the safety period was 36.

The expression time of 12 people lasted for more than 2 years, and the activity of factor 8 did not decrease over time.

After Spk-8011 treatment, the patient's bleeding risk is greatly reduced

After Spk-8011 treatment, the bleeding risk of patients was greatly reduced.

SPK-8011 can achieve continuous factor 8 expression in patients with hemophilia A, reducing bleeding risk by more than 90% SPK-8011 can achieve continuous factor 8 expression in patients with hemophilia A, reducing bleeding risk by more than 90%

Original source:

Lindsey A.

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.

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