Due to safety issues, the FDA suspends a clinical trial of cell therapy for hemophilia, Sigilon's stock price plummets

Written | Edited by Wang Cong | Nagashi Typesetting | Hemophilia is a rare type of hereditary bleeding disorder that causes severe coagulopathy in patients caused by the lack of certain coagulation factors in the blood.
It can occur in both men and women.
However, since the overwhelming majority of hemophilia A and B are X-chromosome recessive inherited diseases, women only have two mutations to develop the disease, while men only have one mutation to develop the disease.
Therefore, most patients with hemophilia For men
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Hemophilia is the most common congenital bleeding disorder, and bleeding is the main clinical manifestation of the disease
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Patients are often prone to bleeding and difficult to coagulate due to insufficient coagulation factors.
Therefore, most patients have long-term restrictions on activities, unable to go to school or go out to play, etc.
, and even some patients with severe hemophilia need to stay in bed for a long time, and the quality of life is extremely poor.

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Recently, FDA suspended the encapsulated cell therapy clinical trials Sigilon Therapeutics company's treatment of hemophilia A type
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Affected by this, Sigilon's stock price plummeted by 25%
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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 membrane.
It protects it from the immune system and prevents scar tissue from forming around it, and continues to produce coagulation factor VIII
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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
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Sigilon said that the FDA has requested more information about the possible causes of patients' antibodies, including their family history and recent vaccination status
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Sigilon CEO Dr.
Rogerio Vivaldi said that he is currently working with regulatory agencies and company consultants to conduct a thorough investigation of the incident to confirm whether there is a causal relationship between the production of antibodies and SIG-001 therapy
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Sigilon was established in 2015 in Cambridge, Massachusetts, USA.
With the support of the well-known venture capital firm Flagship Pioneering, Sigilon has developed rapidly and will be listed on the Nasdaq in December 2020
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At the beginning of the listing, the market value once exceeded $1 billion, but it has now fallen by 80%
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Sigilon's technology platform Sigilon's R&D pipeline hemophilia gene therapy For hemophilia, BioMarin and Roche's subsidiary Spark Therapeutics are both conducting research on gene therapy
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In 2017, the FDA suspended Sanofi's hemophilia gene therapy clinical trials due to the death of a patient
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Among them, the most rapid progress is BioMarin.
In January 2020, the New England Journal of Medicine (NEJM) published a multi-year follow-up survey of BioMarin's hemophilia type A gene therapy
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15 patients with severe hemophilia type A who received AAV gene therapy, after 2-3 years of treatment, the effect was very significant, and there were no other serious side effects
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It is understood that BioMarin has submitted a marketing application for the AAV therapy to the European Medicines Agency and the US FDA
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Image source: NEJM Reference materials: https://sigilon.
com/science/https:// Open for reprint