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12:00 AM
Apr 28, 2014
 |  BioCentury  |  Emerging Company Profile

NightstaRx: Correcting choroideremia

NightstaRx focusing on AAV gene therapy for degenerative retinal conditions

NightstaRx Ltd. is developing an adeno-associated viral vector-based gene therapy to treat choroideremia, a rare X-linked hereditary retinal dystrophy for which there are no disease-modifying treatments and no other disclosed clinical programs.

The company was spun out of the University of Oxford and its technology transfer company, Isis Innovation Ltd., in late January with a £12 million ($19.8 million) series A round led by Syncona Partners and an exclusive, worldwide license to IP from the university covering AAV-REP1.

AAV-REP1 is an AAV serotype 2 vector encoding Rab escort protein 1 (CHM; REP1), which facilitates the passage of nutrients across cells.

In choroideremia, a deletion or loss-of-function mutations in the gene encoding REP1 result in gradual degeneration of the choroid and the retinal pigment epithelium, which supply oxygen and nutrients to the retina. Eventually, photoreceptors began to degenerate as well.

Over several decades, usually beginning in the teenage years, patients progress from night blindness...

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