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Gene therapies are a highly promising and rapidly expanding new class of biotherapeutics able to treat and potentially cure diseases that currently have few or no treatment options. The major challenge facing commercialisation of gene therapies is the manufacture and supply of products at a commercial scale. Critical to the success of gene therapies is the ability to develop robust and scalable manufacturing process that can meet anticipated commercial demand. Platform processes will enable more rapid development and commercialisation of gene therapies at lower risk and cost. This 12 month project brings together the significant AAV process development expertise and capability of two leading national research organisations, CPI and the National Research Council of Canada, to rapidly optimise a new manufacturing platform for the benefit of companies developing gene therapies (particularly SMEs) and accelerate these important new treatments to market.
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