AMT makes important advance in the development of its gene therapy for Hemophilia B

Company Accesses Technology to Prevent Immune Response

Amsterdam, The Netherlands – Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it obtained a license from ‘TIGET’ San Raffaele Telethon Institute For Gene Therapy, Italy, to use their micro-RNA technology to prevent immune responses against gene therapy for Hemophilia B. This technology enables AMT to accelerate the development of AMT-060 (AMT’s AAV-based gene therapy for this seriously debilitating disease).

Hemophilia B occurs in 1 in 30,000 males and results in severe internal and external bleeding. The disease is caused by defective blood clotting due to an absence of clotting factor Factor IX. Gene therapy can be used to introduce the gene that produces Factor IX and thereby repair blood clotting. AMT will use the TIGET technology to prevent an immune response against the newly formed Factor IX, which the patient’s body may recognize as being foreign.

Formalizing relationship with leading micro-RNA research group of professor Naldini

Ronald Lorijn, CEO of AMT said:

“We are very excited to have licenced 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 hemophilia B with a single treatment.”

TIGET is very satisfied as this agreement represents an important development of the discoveries made by Prof. Luigi Naldini, Co-Director of TIGET and his collaborator Dr. Brian Brown, and that it will foster further industrial collaborations aimed at moving these innovative solutions from the research to the clinic.

Micro-RNA inhibition

Luigi Naldini and Brian Brown of TIGET have demonstrated 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 expression of the Factor IX gene can be selectively blocked by AMT in immune stimulating cells thus preventing the development of an immune response, and thereby enabling full effectiveness of the gene therapy for Hemophilia B.

About Amsterdam Molecular Therapeutics

AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. As such, AMT’s proprietary platform holds tremendous promise for thousands of rare (orphan) diseases that are caused by one faulty gene. AMT currently has a product pipeline with seven products at different stages of development. 


The San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) is a joint-venture between the San Raffaele Scientific Institute and the Telethon Foundation for the implementation of basic, pre-clinical and clinical research for genetic diseases. The mission of the Institute is to perform cutting edge science in the field of gene therapy and to promote the translation of basic discoveries into therapeutic advances. The genetic diseases, which are presently under investigation include primary immunodeficiencies, lysosomal storage disorders, type I diabetes, hemophilia. In addition, a primary goal of TIGET research is to improve the efficiency and safety of gene transfer by advancing the development and applications of lentiviral vector technology. For this research TIGET also received funding from the European Union (VIth Framework Program: Project RIGHT)

For information:

André Verwei
+31 20 566 5686

Rob Janssen
Director Corporate Communications & Investor Relations
+31 20 566 7509