FDA suspends Sigilon’s hemophilia cell therapy clinical trials

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FDA suspends Sigilon’s hemophilia cell therapy clinical trials  due to safety issues

FDA suspends Sigilon’s hemophilia cell therapy clinical trials  due to safety issues.   A few days ago, the FDA suspended Sigilon Therapeutics’ clinical trials of encapsulated cell therapy for the treatment of hemophilia type A.　　

Sigilon Therapeutics developed the hemophilia type A cell therapy numbered SIG-001, which aims to restore the patient’s ability to produce coagulation factors. This therapy encapsulates the coagulation factor VIII-producing cells in a semi-permeable protective film. It protects it from the immune system and prevents scar tissue from forming around it, and continues to produce coagulation factor VIII.

However, in this clinical trial involving 3 hemophilia A patients, the patients who received the highest dose of treatment showed antibodies against coagulation factor VIII, which means that the patient’s own immune system treats it as a foreign substance. Attacked.

Sigilon said that the FDA has requested more information about the possible causes of patients’ antibodies, including their family history and recent vaccination status.

Sigilon’s technology platform

Sigilon’s R&D pipeline

Hemophilia gene therapy

For hemophilia, BioMarin and Roche subsidiary Spark Therapeutics are both conducting research on gene therapy. In 2017, the FDA suspended Sanofi’s hemophilia gene therapy clinical trials due to the death of a patient.

Among them, the most rapid progress is BioMarin. It is understood that BioMarin has submitted a marketing application for the AAV therapy to the European Medicines Agency and the US FDA.

　　 Picture source: NEJM

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