Gene therapy enablement

Currently, it is estimated that antibodies prevent up to 1 in 3 people from benefiting from gene therapy treatments.^1-4

For people living with rare, monogenic diseases, gene therapies may offer a cure to a life-long condition by introducing genetic material that compensates for a defective gene. However, if the therapy is based on the use of Adeno Associated Viruses (AAV) vectors, the immune system may carry antibodies that counteract the gene therapy treatment preventing its success.^2,5-6 Understanding how to overcome the action of anti-AAV antibodies is crucial to ensure all patients in need can receive gene therapy treatments.

Our commitment

Bridging the access to gene therapies for more patients

We are collaborating with gene therapy companies at the forefront of innovation to evaluate the potential of our technology as a pre-treatment to gene therapy. The hope is that by creating an IgG-free window in those patients with anti-AAV antibodies, it would allow for the successful administration of a gene therapy treatment. 

Partnerships

Sarepta Therapeutics

AskBio

Généthon

We work with Généthon to assess the use of imlifidase as a pre-treatment preceding the administration of Généthon’s gene therapy in Crigler-Najjar syndrome in patients with antibodies targeting adeno-associated virus (AAV). The collaboration, set as a preclinical and clinical feasibility program, evaluates the safety and efficacy of imlifidase in this setting.