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.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

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.

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