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Fundraisings and IPOs

Date: 2014-01-30

Type of information: Financing round

Company: Nightstar (UK)

Investors: Syncona (UK)

Amount: £12 million

Funding type: seed financing round

Planned used:

Nightstar will focus on the development and commercialisation of therapies for retinal dystrophies.The company’s first programme is a gene therapy for an inherited form of progressive blindness called choroideremia developed by Professor Robert MacLaren at Oxford’s Nuffield Laboratory of Ophthalmology. The therapy has shown extremely promising results in
a clinical trial published in the Lancet in mid-January. The gene therapy uses a small modified virus, AAV.REP1 to deliver the correct version of the choroideremia (CHM) gene to cells in the retina of the eye. The Lancet reported that six months after treatment with this therapy, the first six patients showed improvement in their vision in dim light and two of the six were able to read more lines on the eye chart. The vector used to treat choroideremia, AAV.REP1, was developed by a team of
researchers, led by Professor Robert MacLaren of the University of Oxford, and is currently being studied in a 12 patient Phase I clinical trial supported by the Wellcome Trust and Department of Health. Nightstar has an exclusive licence from Isis Innovation to the intellectual property which underpins this program.

Others:

* On January 30, 2014, Syncona LLP, an independent subsidiary of the Wellcome Trust, announced a £12 million investment in NightstaRx, a spin-out from the University of Oxford and its research commercialisation company Isis Innovation. Chris Hollowood, partner at Syncona and Nightstar’s Chairman, commented: “The recent data publication in the Lancet is very compelling and we are delighted to be working with Professor MacLaren to provide the support required to bring this important therapy to choroideremia patients. We have appointed Dr. Melanie Lee as CEO of Nightstar; an experienced industry professional with both scientific and business acumen and we will augment the team over the coming weeks.”

Therapeutic area: Ophtalmological diseases

Is general: Yes