San Raffaele outlicenses micro-RNA technology

Licensing deal

TIGET, the San Raffaele Telethon Institute for Gene Therapy, in Italy, has granted the Dutch gene therapy specialist Amsterdam Molecular Therapeutics (AMT) the right to use its micro-RNA technology to prevent immune responses against gene therapy for haemophilia B.

This technology will enable AMT to accelerate the development of its treatment for the condition, in which the blood does not clot properly. This is caused by an absence of the clotting factor Factor IX.

Gene therapy is used to introduce the gene that produces Factor IX, and AMT will use the TIGET technology to prevent an immune response against the newly formed Factor IX, which the patient’s body may recognise as being foreign.

Ronald Lorijn, CEO of AMT said, “We are very excited to have licensed this groundbreaking technology, which has been shown to prevent immune responses effectively. Access to TIGET’s micro-RNA technology perfectly complements our gene therapy approach to cure haemophilia B with a single treatment.”

TIGET says this agreement represents an important development of the discoveries made by Luigi Naldini, Co-Director of TIGET and his collaborator Brian Brown, and will foster further industrial collaborations.

Naldini and Brown have shown in mouse models that naturally occurring micro-RNAs can be exploited to stringently regulate the expression of genes delivered by vectors used in gene therapy.

With this approach AMT’s hopes to selectively block the expression of the Factor IX gene in immune stimulating cells, thus preventing the development of an immune response.