Gene therapy company uniQure mulling potential sale: report

According to people with knowledge of the matter, uniQure is assessing its options, including partnerships or a possible sale, amid interest from pharmaceutical firms seeking to expand in gene therapy, Bloomberg reported Sunday. The sources noted that no final decisions have been made, and there is no guarantee that negotiations will result in a deal. A representative for the gene therapy company, which has a market value of about $2.7 billion, declined to comment on the speculation.

In February, analysts at Cantor Fitzgerald suggested uniQure "could be next" to be acquired, citing major drugmakers such as Novo Nordisk, Pfizer and Sanofi as potential suitors. UniQure, which employs around 200 staff, has a pipeline of experimental treatments for haemophilia, Huntington's disease, including the gene therapy AMT-130, as well as other disorders.

EU regulators cleared uniQure’s Glybera (alipogene tiparvovec) in 2012 for patients with lipoprotein lipase deficiency, making it the first gene therapy product approved in Europe. However, due to a lack of demand for the treatment, which launched about two years later at a cost of 1.1 million euros ($1.2 million), the company eventually decided against renewing its EU marketing authorisation and has also said it would not pursue US approval of the product either.

UniQure has since focused on haemophilia treatments. Earlier this year, the company unveiled updated results from a Phase IIb study showing that the experimental AAV5-based gene therapy AMT-061 was associated with mean Factor IX activity of 38 percent of normal at 12 weeks after administration in patients with severe and moderately severe haemophilia B.

Among other transactions involving gene therapy companies are Biogen's recently finalised purchase of Nightstar Therapeutics for $800 million. Novartis bought AveXis for $8.7 billion last year, gaining spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovac-xioi), for which it plans to charge $2.1 million following its US approval last month.

Pfizer acquired a 15-percent equity stake in Vivet Therapeutics this past March, as well as an exclusive option to purchase the gene therapy company for 560 million euros ($628 million). Meanwhile, Roche is in the process of buying gene therapy developer Spark Therapeutics for $4.3 billion, although the deal is facing delays, most recently amid scrutiny from regulators in the US and the UK.  

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