BMS invests in gene therapy

Bristol-Myers Squibb (BMS) has agreed to invest over $100 million (£67 million) in Netherlands-based biotechnology company uniQure, which specialises in gene therapies for cardiovascular diseases.

UniQure developed alipogene tiparvovec, marketed as Glybera, which became the first gene therapy drug to be granted EU regulatory approval in 2012. Glybera treats lipoprotein lipase deficiency (LPLD), a rare inherited disease affecting one or two people in a million, which prevents the metabolism of fat particles in the blood. The drug contains a working copy of the human lipoprotein lipase (LPL) gene LPLS447X.

The deal will see BMS invest $100 million in R&D, and purchase a 4.9% stake in the company for around $32 million. The companies will collaborate on the development of new therapies, including uniQure’s gene therapy treatment programme S100A1, which is being developed for the treatment of heart failure. UniQure will continue to lead development and will manufacture clinical supplies, while BMS will be responsible for commercialising any successes. In addition to royalties for the drugs it sells, BMS has agreed to pay uniQure milestone payments in the region of $200 million for successful development outcomes, such as gaining regulatory approval for new treatments or achieving sales targets.