AMT Makes Important Advance in the Development of its Gene Therapy for Hemophilia B -Company Accesses Technology to Prevent Immun...( AMSTERDAM The Netherlands June 10...)

AMSTERDAM, The Netherlands, June 10 /PRNewswire-FirstCall/ -- 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
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