UniQure in gene therapy deal with BMS

Written by Victoria English

UniQure NV has secured a gene therapy agreement with Bristol-Myers Squibb Company in cardiovascular disease, its first major commercial deal since winning regulatory approval in 2012 for Glybera, a gene therapy for lipoprotein lipase deficiency.

UniQure NV has secured a gene therapy agreement with Bristol-Myers Squibb Company in cardiovascular disease, its first major commercial deal since winning regulatory approval in 2012 for Glybera, a gene therapy for lipoprotein lipase deficiency.

Under the agreement, BMS will gain exclusive access to UniQure’s gene therapy platform for investigating targets for cardiovascular therapies. The deal includes rights to a gene therapy project aimed at treating congestive heart failure by restoring the heart’s ability to synthesise S100A1, a calcium sensor and master regulator of heart function.

UniQure acquired the programme when it bought InoCard GmbH of Germany in 2014. The technology involves delivering a gene expressing S100A1 to the heart by way of an adeno-associated viral vector. The product is still in preclinical development.

Beyond cardiovascular diseases, the agreement leaves the door open for collaborations in other disease areas. In total the two companies may collaborate on up to 10 targets including S100A1.

The financial terms of the agreement feature near-term payments by BMS to UniQure of about $100 million. BMS will acquire 5% of UniQure’s equity by 31 December and up to an additional 10% based on additional targets being introduced into the collaboration. There is also a supply contract whereby UniQure will manufacture all of the gene therapy products covered by the collaboration. Additionally, UniQure will be eligible for milestone payments of up to $254 million for the S100A1 product and up to $217 million for each of any other gene therapy products.

The two companies announced the collaboration on 6 April 2015.

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