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Mergers & Acquisitions

Biogen to buy the retinal gene therapy maker Nightstar

The $800 million purchase is the third gene therapy acquisition announced in a week

by Ryan Cross
March 4, 2019

A photo of a surgeon injecting Nightstar's gene therapy into a person's eye.
Credit: Nightstar Therapeutics
A surgeon at Oxford Eye Hospital performs surgery to inject Nightstar’s gene therapy for X-linked retinitis pigmentosa into a person’s eye.

Biogen is the third drug company in a week to open its wallet to buy a gene therapy firm. Biogen says it will acquire UK-based Nightstar Therapeutics for $800 million, a 68% premium over the firm’s stock market value of about $500 million on March 1, before it was announced.

The news “clearly reinforces the notion that gene therapy continues to be a ‘hot’ therapeutic area” for drug company acquisitions in 2019, analysts from J.P. Morgan wrote in a note to investors.

Last week, Roche announced that it will purchase the gene therapy company Spark Therapeutics for $4.8 billion. The muscular dystrophy drug firm Sarepta Therapeutics recently said it will pay $165 million to acquire a small Ohio-based gene therapy start-up called Myonexus Therapeutics.

And in January, Johnson & Johnson agreed to pay MeiraGTx $100 million in cash to develop and commercialize two retinal gene therapy programs.

As experimental gene therapy programs progress through late-stage clinical studies, large drug companies are increasingly spotting value in gene therapies. In a report after the Spark purchase was announced, analysts from the investment firm SVB Leerink suggested that more such deals could be coming, naming Nightstar, Audentes Therapeutics, BioMarin Therapeutics, and Uniqure as possible targets.

Nightstar won’t be Biogen’s first foray into gene therapy. In 2015, it struck a deal with Applied Genetics Technologies Corp. (AGTC) to develop gene therapies for several eye diseases. AGTC stood to receive more than $1 billion in milestone payments, but Biogen pulled the plug on the collaboration in December 2018 after a clinical trial showed one of AGTC’s gene therapies was ineffective.

Biogen will be giving gene therapies for the eye another try with Nightstar, which is focused on rare inherited retinal disorders. It is running advanced-stage clinical trials for choroideremia and X-linked retinitis pigmentosa, diseases that are caused by mutations that lead to the degeneration of photoreceptors. Nightstar is using adeno-associated viruses to deliver corrective pieces of DNA into the eyes of people with the conditions.


Several other companies are developing gene therapies for eye diseases. That’s partly because it seems to be easier and safer to inject gene therapies into the eye than into other parts of the body and partly because of precedence from a commercial product. The only gene therapy currently approved in the US is Spark’s Luxturna, which treats a rare form of inherited blindness.

After the Spark acquisition and J&J’s partnership with MeiraGTx, Biogen “may have felt the need to pull the trigger before a more competitive bidding process ensued,” the SVB Leerink report states. The current moment in gene therapy is “reminiscent of the early days of biologics,” which spurred a bounty of deals between biotech and larger drug companies, the report says.

Biogen anticipates that Nightstar’s therapies for both conditions could be approved and on the market in the early 2020s.


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