Hansa Biopharma and Genethon are planning to test imlifidase as a gene therapy pre-treatment for patients with a type of antibody resistance.
The collaboration will investigate Hansa’s imlifidase as a pre-treatment for Genethon’s gene therapy GNT-0003 in patients with the ultra-rare disease Crigler-Najjar syndrome. The two companies will specifically target patients with pre-existing neutralising antibodies to adeno-associated virus serotype 8.
Most gene therapies rely on adeno-associated virus (AAV) vectors to deliver genes, but many patients have antibodies against AAV vectors. Imlifidase could clear anti-AAV antibodies from the body, opening the door for these patients to receive gene therapy treatment.
GNT-0003 is currently in a registrational Phase I/II trial in Europe for Crigler-Najjar syndrome (NCT03466463). Under the new collaboration, a similarly designed study will investigate patients in this indication with anti-AAV8 antibodies who are pre-treated with imlifidase.
Imlifidase, which is marketed as Idefirix for kidney transplant rejection in Europe and Israel, is an antibody-cleaving enzyme that can suppress immune response. Hansa is based in Lund, Sweden, and Genethon is a non-profit biotherapy company headquartered in Paris, France.
Gene therapies for ultra-rare diseases
The new collaboration targets Crigler-Najjar syndrome, an ultra-rare autosomal disorder characterized by high levels of the toxic substance bilirubin in the blood and occurs in less than one in a million cases per year.
Targeted approaches like gene therapies and antisense oligonucleotides have emerged as potential treatment options for ultra-rare genetic diseases. But controlling costs and getting drugs to patients can prove a difficult task, as a bioethicist explained to Clinical Trials Arena.
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