Amsterdam Molecular Therapeutics in US gene therapy tie-up

Collaboration agreed

Amsterdam Molecular Therapeutics, a spin-out from the Amsterdam Academic Medical Centre, The Netherlands, has agreed a collaboration with St Jude Children's Research Hospital in Memphis, Tennessee, USA, to develop a gene therapy treatment for haemophilia B. Under the deal, AMT will receive the exclusive commercial rights to the final product. The combination of this gene with AMT’s proprietary adeno-associated virus (AAV) gene therapy platform could potentially cure this seriously debilitating disease with a single administration of the product, says the company.

Haemophilia B is characterised by severe episodes of external and internal bleeding that result in significant morbidity, cause long-term damage in the joints and may be fatal when occurring in tissues like the brain. Current treatments require up to three injections a week to stop or prevent bleeding, which is a burden for young children, may be associated with abnormal blood clotting and does not prevent recurrent bleeding episodes. It is a rare disease, occurring in 1 in 30,000 people, almost always in males. The total number of patients in Europe and the US is estimated between 35,000 and 40,000.

Under the agreement AMT will sponsor research in haemophilia B at St Jude. Ronald Lorijn, CEO of AMT said: “The importance of this collaboration with renowned St Jude Children’s Research Hospital stretches beyond its scientific and business aspects. This collaboration will bring to these patients the hope that a real cure is on the horizon. Access to the Factor IX gene therapy programme perfectly complements our gene therapy platform allowing us to develop an effective and long-lasting therapy for haemophilia B. Dr Arthur W. Nienhuis and his group at St Jude have done very important scientific work on this disease and we really look forward to collaborating with them.”

Amit Nathwani, who began his work on Factor IX while in Nienhuis’ lab, has continued to collaborate with St Jude faculty Andrew Davidoff and John Gray in developing a novel AAV vector that produces therapeutic levels of Factor IX when given intravenously in animal models. This vector has been licensed to AMT.

Haemophilia B is characterized by defective blood clotting due to an absence of functional clotting Factor IX. The gene with the information for the Factor IX protein is mutated in Hemophilia patients, resulting in the production of non-functional protein. AMT uses gene therapy to introduce the functional gene into the patient's cells and thereby restore blood clotting.

AMT recently acquired a licence from TIGET, the San Raffaele Telethon Institute For Gene Therapy, Italy, to use its micro-RNA technology to prevent immune responses against gene therapy for haemophilia B. 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.