Genethon and Hansa Biopharma launch a phase 2 clinical trial for Crigler-Najjar syndrome
The trial will be conducted in patients for whom pre-existing anti-AAV antibodies are currently a limitation to gene therapy treatment.
Genethon, the Telethon laboratory, and Hansa Biopharma, a pioneer in enzyme technology for rare immunological diseases, today announced the launch of a phase 2 trial in patients suffering from Crigler-Najjar disease, a rare liver disorder, and presenting with pre-existing neutralising antibodies against AAV vectors.
The trial will evaluate the efficacy and safety of a single intravenous injection of the gene therapy developed by Genethon, after pre-treatment with imlifidase, a unique antibody-cleaving enzyme developed by Hansa.
Overcoming the challenge of anti-AAV antibodies to broaden access to gene therapy
For diseases of the muscle, liver or vision, gene therapy today uses vectors derived from AAV viruses to transport the gene-drugs into the cells. Antibodies against these vectors represent a major challenge, as their presence in around a third of patients prevents them from taking part in gene therapy clinical trials and gaining access to available gene therapies.
For Frédéric Revah, Chief Executive Officer of Genethon, “This new clinical trial reflects Genethon’s commitment in pursuing innovative strategies to ensure and broaden access to gene therapies for patients suffering from rare diseases... The initiation of this clinical trial and the collaboration with Hansa Biopharma is a crucial step for Genethon and highlights several years of pioneering research to understand and control the immune response to AAV in order to make gene therapy more effective and to increase the number of patients able to access it.”
To learn more about the clinical trial, read Genethon's press release