Biogen bags UK company Nightstar for US$877m

The US company paid $877m, a sixth of the price Roche paid for gene therapy pioneer Spark Therapeutics this month.

Nightstar currently has seven ophtalmological programmes under development, two thereof in clinical phases. The company will shortly start a Phase III trial with its lead candidate NSR-REP1, an adeno-associated virus (AAV) expressing the CHM gene, to treat choroideremia, a X-chromosomally inherited retinal defect of the CHM gene that affects 13,000 male patients in the US. Expression of the CHM gene boosts production of the Rab escort protein-1 (REP-1), which plays a role in intracellular protein trafficking and the elimination of waste products from retinal cells.

Furthermore, the company is in Phase I/II testing of NSR-RPGR to treat X-linked Retinitis Pigmentosa (XLRP), a disorder currently without a cure that is caused by a mutations in the RPGR gene resulting in photoreceptor degeneration. NSR-RPGR is an AAV8 vector overexpressing a codon-optimised form of the RPGR (retinitis pigmentosa GTPase regulator) gene. The gene therapy has successfully passed dose-escalation.

Single administration-gene therapies for ophthalmological diseases are expected to outperform antibody-based treatments because they would not require lifelong treatment.