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.

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.