Nightstar Therapeutics is a spinout from Oxford University and Imperial College London, with scientists from both institutions contributing to the underlying research upon which Nightstar’s product candidates have been developed.
Nightstar’s lead gene therapy product candidate is NSR-REP1, which is being developed for the treatment of choroideremia (CHM), a rare, degenerative, X-linked genetic retinal disorder primarily affecting males that is caused by a mutation in the CHM gene. The company plans to initiate a phase 3 registration study of NSR-REP1 for CHM in the first half of 2018.
CHM is caused by defects in the CHM gene on the X chromosome. The defect halts production of a protein by the CHM gene, without which pigment cells in the retina begin to stop working and eventually die. This causes progressive sight loss as the retina shrinks in size.
Professor Miguel Seabra, of the National Heart and Lung Institute, Imperial College London, identified the protein involved in CHM some 20 years ago, and he and his team at the NHLI have conducted extensive pre-clinical work enabling the project to enter the clinic. He was the lead scientist on the phase 1 clinical trial led by Professor Robert MacLaren, of the Nuffield Laboratory of Opthalmology at the University of Oxford, which led to the development of a gene therapy approach to the treatment of CHM.
Imperial Innovations acquired an equity interest in Nightstar Therapeutics as part of the original intellectual property contributions of Prof. Seabra to Nightstar.
Professor Miguel Seabra, Imperial College London, said:
“This is an exciting moment for all stakeholders involved in researching this gene therapy approach to treating choroideremia, from the researchers in the lab to the patients and clinicians involved in the early trials.
”I hope Nightstar’s fundraise represents another step towards developing this transformative treatment into a commercial product available to all patients.”
Tony Hickson, Managing Director, Imperial Innovations, said:
“The Nightstar IPO provides evidence of the strength of the UK’s science base and shows the value of academic collaboration within the ‘Golden Triangle’ of London, Oxford and Cambridge. We are delighted to see the continuing progress of Nightstar and congratulate the teams at Imperial and Oxford whose scientific research has contributed towards it.”