Syncona Limited - Nightstar initiates Pivotal Trial in Choroideremia

The full Nightstar announcement can be found here: http://ir.nightstartx.com/news-releases  

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Enquiries

Syncona Ltd
Siobhan Weaver
Tel: +44 (0) 20 7611 2031
+44 (0) 7921 822 994

Tulchan Communications
Martin Robinson
Lisa Jarrett-Kerr
Tel: +44 (0) 207 353 4200
Copies of this press release and other corporate information can be found on the company website at: www.synconaltd.com  

Forward-looking statements – this announcement contains certain forward-looking statements with respect to the portfolio of investments of Syncona Ltd. These statements and forecasts involve risk and uncertainty because they relate to events and depend upon circumstances that may or may not occur in the future. There are a number of factors that could cause actual results or developments to differ materially from those expressed or implied by these forward-looking statements and forecasts. Nothing in this announcement should be construed as a profit forecast. 

About Syncona:

Syncona is a leading FTSE250 healthcare company focussed on investing in and building global leaders in life science. Our vision is to deliver transformational treatments to patients in truly innovative areas of healthcare while generating superior returns for shareholders. Our current investment portfolio consists of seven high quality companies in life science and a leading range of fund investments. 

We seek to partner with the best, brightest and most ambitious minds in science to build globally competitive businesses. We are established leaders in gene therapy, cell therapy and advanced diagnostics, and focus on delivering dramatic efficacy for patients in areas of high unmet need.

Our market leading funds portfolio seeks to generate superior returns by investing in long only and alternative investment funds. This represents a productively deployed evergreen funding base which enables us to take a long term approach to investing in life sciences as we target the best new opportunities and support our existing portfolio companies to grow and succeed.

Syncona is aligned with two of the premium charitable funders in UK science, the Wellcome Trust, original founder of Syncona, and Cancer Research UK, both of which are significant shareholders in our business.  We make a donation of 0.3% of Net Asset Value to a range of charities each year.

About Nightstar:

Nightstar is a clinical-stage gene therapy company focused on developing and commercializing novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness

Nightstar’s most advanced programme is in clinical trials for the treatment of choroideremia, a rare disease which causes permanent loss of eyesight for which there is no treatment. Nightstar’s treatment would deliver a gene therapy by injection into the retina, providing a working copy of the disease-causing faulty gene locally in the eye. The disease modifying technology has the potential to maintain and restore sight in patients, from a single administration.

There are multiple inherited forms of blindness that are addressable with Nightstar’s gene therapy technology. Nightstar is building a pipeline of products based on its manufacturing, gene therapy and retinal surgery capability. Nightstar is listed on NASDAQ under the ticker ‘NITE’.

Syncona launched Nightstar in 2014 with Professor Robert MacLaren, Professor of Ophthalmology at the University of Oxford.

[1] ETDRS is a standardised, widely used measure of visual acuity which requires subjects to read letters on a chart.