The partnership, in a clinical trial, will assess the efficacy and safety of imlifidase, Hansa Biopharma’s antibody cleaving enzyme, as a pre-treatment prior to the administration of Genethon’s gene therapy product candidate GNT-0003 in Crigler-Najjar syndrome in patients with pre-existing neutralizing antibodies (NAbs) to adeno-associated virus serotype 8 (AAV8).
The company stated that patients with circulating NAbs are today excluded from clinical trials of potentially curative gene therapies as well as from access to approved gene therapies.
Hansa Biopharma president and CEO Søren Tulstrup said: “Genethon is a pioneer at the cutting-edge of research and development of gene therapies for rare diseases and we are thrilled to be collaborating with them.
“This research collaboration further validates Hansa’s commitment in gene therapy and underscores the important role that our antibody-cleaving enzyme technology can play in ensuring that even more patients can benefit from life-saving gene therapies”.
Imlifidase is originated from Streptococcus pyogenes that targets IgG specifically and inhibits IgG-mediated immune response.
The European Medicines Agency (EMA) granted PRIME (PRIority MEdicines) status to GNT-0003, which is now being assessed in a pivotal clinical trial in the Netherlands, Italy, and France.
The company stated that Crigler-Najjar patients with pre-formed antibodies to AAV8 will be enrolled in a trial with similar design, through this collaboration.
The trial will assess imlifidase as a pre-treatment to enable gene therapy treatment with GNT-0003.
Hansa Biopharma expects the findings from the ongoing GNT-0003 clinical trial will be the basis for a biologics license application (BLA) in the US or marketing authorisation applications (MAA) in Europe.
Genethon CEO Frédéric Revah said: “Patients with pre-existing neutralizing antibodies against AAV vectors cannot today benefit from gene therapy.
“This collaboration with Hansa Biopharma is thus an important next step in the development of our gene therapy treatment for Crigler-Najjar syndrome, Hansa Biopharma’s proven enzyme technology coupled with its scientific expertise will help us advance the critical research we are conducting in Crigler-Najjar and could enable gene therapy treatment for patients who are today not eligible because of their immunological status.”