BMS inks gene therapy deal with uniQure worth up to $2bn

By Dan Stanton

- Last updated on GMT

A new gene is inserted into a cell using an AAV vector. Once inside the nucleus, the new gene makes functional protein to treat a disease.
A new gene is inserted into a cell using an AAV vector. Once inside the nucleus, the new gene makes functional protein to treat a disease.

Related tags Gene therapy

Bristol-Myers Squibb will use uniQure’s insect-cell based technology and manufacturing facilities to develop gene therapies for cardiovascular diseases in a deal potentially worth $2bn (€1.8bn).

The deal gives Bristol-Myers Squibb (BMS) exclusive access to uniQure’s gene therapy technology for up to nine targets in cardiovascular diseases and was described by uniQure’s CEO Joern Aldag yesterday as “one of the largest and most comprehensive collaborations in the gene therapy sector to date.”

uniQure, which saw its stock rise more than 50% on Monday, has gained regulatory success with Glybera, its gene therapy treatment for the rare disease that compensates for lipoprotein lipase deficiency (LPLD), and speaking on a conference call Aldag said this was a major reason behind BMS’s decision to select the platform.

“We have the experience of bringing a gene therapy product all the way to market and we have the most comprehensive platform of manufacturing technologies and vectors to do it again,”​ he said.

BMS will take over uniQure’s lead programme for congestive heart failure, S100A1, plus a further nine targets in a deal that could net uniQure up to $2bn. BMS will pay an upfront payment of $50m, plus a further $32m for a 4.9% equity stake in uniQure which could increase to nearly 20% as the collaboration progresses.

“We will lead the discovery efforts and be responsible for manufacturing of clinical and commercial supplies using our vector technology and our proprietary insect-cell based manufacturing platform. BMS will be responsible for all R&D costs, lead development, regulatory activities and commercialisation.”

Tech and manufacturing

The technology has been licensed and enhanced by uniQure from the National Institutes of Health (NIH), and is comprised of therapeutic gene cassettes (that carry a transgene that provides the blueprint for the expression of a therapeutic protein), coupled with an AAV-based vector system and administration technologies which deliver the relevant transgene into the tissues and organs.

The company uses insect cells and baculoviruses to manufacture these AAV-based gene therapies - which Aldag said makes it highly scalable and cost effective - from its facility in Amsterdam, but following an investment in 2013, a second 53,000sq ft site in Lexington, Massachusetts is set to be operational later this year.

“Today we have what we believe to be the largest, most versatile modular gene therapy manufacturing facility in the world in Lexington, Massachusetts,”​ Aldag said, adding the build out of the plant is complete and GMP production is expected to commence in the autumn.

“Combined with our Amsterdam facility we are proud to say there is no other company with the manufacturing expertise, capacity and flexibility of uniQure.”

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