pharmaceutical-technologyMarch 05, 2019
Tag: gene therapy , Roche , Biogen , Nightstar Therapeutics
US-based biotechnology company Biogen has signed an agreement to acquire UK-based gene therapy firm Nightstar Therapeutics for a cash consideration of $800m.
The agreement values each Nightstar Therapeutics share at $25.50.
Nightstar Therapeutics is focused on developing treatments for rare inherited retinal diseases. Its lead programme NSR-REP1 targets a degenerative, X-linked retinal disorder called choroideremia (CHM).
The inherited disorder is caused due to loss of function in the CHM gene that encodes the Rab escort protein-1 (REP-1). This loss of function results in abnormal intracellular protein trafficking.
It leads to blindness and currently lacks approved therapies.
NSR-REP1 consists of an AAV vector that delivers a functioning CHM gene and the REP-1 protein expression to restore membrane trafficking and slow, stop or potentially reverse vision deterioration.
The gene therapy company’s pipeline also includes NSR-RPGR for the treatment of X-linked retinitis pigmentosa (XLRP), which is caused by the retinitis pigmentosa GTPase regulator (RPGR) gene mutations.
Biogen expects the acquisition to support its entry into ophthalmology therapeutic area, with the addition of two gene therapy programmes to its pipeline.
"Nightstar would accelerate our entry into ophthalmology by contributing two mid to late stage gene therapy assets, with the potential to create long-term shareholder value."
Biogen CEO Michel Vounatsos said: "With this proposed acquisition, we are continuing to bolster our pipeline and further execute on our strategy to develop and expand a multi-franchise neuroscience pipeline across complementary modalities.
"Nightstar would accelerate our entry into ophthalmology by contributing two mid to late stage gene therapy assets, with the potential to create long-term shareholder value."
Nightstar Therapeutics is also developing a preclinical product called NSR-ABCA4 to potentially treat Stargardt disease, along with additional programmes for Best vitelliform macular dystrophy and other genetic forms of retinitis pigmentosa.
Nightstar Therapeutics CEO David Fellows said: "Our agreement with Biogen will give us the platform and resources to expand our mission to maintain and restore sight in patients with inherited retinal diseases."
Subject to customary closing conditions and approvals, the acquisition is expected to close in the middle of this year.
Last month, Roche signed a similar deal, agreeing to buy US-based gene therapy company Spark Therapeutics for $4.8bn.
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