Share Name Share Symbol Market Type Share ISIN Share Description
Syncona LSE:SYNC London Ordinary Share GG00B8P59C08 ORD NPV
  Price Change % Change Share Price Bid Price Offer Price High Price Low Price Open Price Shares Traded Last Trade
  +2.00p +0.90% 225.00p 224.50p 226.00p 225.00p 225.00p 225.00p 20,760 08:30:01
Industry Sector Turnover (m) Profit (m) EPS - Basic PE Ratio Market Cap (m)
Equity Investment Instruments 28.7 5.1 0.8 288.5 1,484.89

Syncona Limited Nightstar receives RMAT designation in Choroideremia

14/06/2018 11:07am

UK Regulatory (RNS & others)

Syncona Limited 
             Nightstar receives RMAT designation in Choroideremia 
14 June 2018 
Syncona Ltd, a leading healthcare company focused on investing in and building 
global leaders in life science, today notes that its portfolio company, 
Nightstar Therapeutics Plc (NASDAQ: NITE) (Nightstar), has announced that the 
U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine 
Advanced Therapy (RMAT) designation to NSR-REP1, the company's lead product 
candidate currently in Phase 3 development for the treatment of choroideremia, 
a rare, degenerative, genetic retinal disorder that leads to blindness. 
The announcement can be accessed on Nightstar's investor website at http:// and full text of the announcement from Nightstar is 
contained below. 
Syncona Ltd 
Siobhan Weaver 
Annabel Clay 
Tel: +44 (0) 20 7611 2031 
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: 
About Syncona: 
Syncona is a leading FTSE250 healthcare company focused 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 fund investments represent 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. 
 Nightstar Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) 
                   Designation for NSR-REP1 in Choroideremia 
  * First gene therapy RMAT designation for an inherited retinal disease 
  * STAR Phase 3 registrational trial ongoing and FDA interactions planned 
  * RMAT designation enables closer and more frequent multidisciplinary 
    interaction with FDA with all of the benefits of breakthrough therapy 
WALTHAM, Mass. and LONDON - June 14, 2018 (GLOBE NEWSWIRE) - Nightstar 
Therapeutics plc (NASDAQ: NITE), a clinical-stage gene therapy company 
developing treatments for rare inherited retinal diseases, today announced that 
the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine 
Advanced Therapy (RMAT) designation to NSR-REP1, the company's lead product 
candidate currently in Phase 3 development for the treatment of choroideremia, 
a rare, degenerative, genetic retinal disorder that leads to blindness. 
"Receiving RMAT designation for NSR-REP1 highlights the potential of this gene 
therapy to maintain and improve visual acuity in choroideremia," said Dave 
Fellows, Chief Executive Officer. "This designation further underscores a 
recognition of the serious nature of choroideremia and the urgent need to 
develop new treatments for those affected by inherited retinal diseases that 
would otherwise lead to blindness. We look forward to working closely with the 
FDA to discuss the NSR-REP1 development program and to determine how we can 
accelerate the pathway for making NSR-REP1 available to choroideremia 
Established under the 21st Century Cures Act, the RMAT designation is an 
expedited program for the advancement and approval of regenerative medicine 
products. A regenerative medicine is eligible for the designation if it is 
intended to treat, modify, reverse or cure a serious or life-threatening 
disease or condition, and preliminary clinical evidence indicates that the drug 
has the potential to address unmet medical needs for such a disease or 
condition. RMAT allows companies developing regenerative medicine and gene 
therapies to work more closely and frequently with the FDA, and grants all of 
the benefits of Breakthrough Therapy Designation, including eligibility for 
priority review, rolling review and accelerated approval. In November 2017, the 
FDA expanded the RMAT designation to include gene therapies. 
RMAT designation for NSR-REP1 was based on clinical data supporting the 
maintenance and improvement of visual acuity from completed Phase 1/2 trials in 
choroideremia patients treated with NSR-REP1 and disease progression in 
untreated patients in the ongoing NIGHT natural history observational study. 
About Choroideremia 
CHM is a rare, degenerative, X-linked genetic retinal disorder primarily 
affecting males, with no treatments currently available and represents a 
significant unmet medical need. CHM presents in childhood as night blindness, 
followed by progressive constriction of the visual fields, generally leading to 
vision loss in early adulthood and total blindness thereafter. CHM is caused by 
mutations in the CHM gene, which encodes REP1, a protein that plays a key role 
in intracellular protein trafficking and the elimination of waste products from 
retinal cells. Absence of functional REP1 leads to death of the RPE cells and 
degeneration of the overlying retina, which contains the retinal photoreceptors 
required to convert light into visual signals. Thus, the loss of REP1 function 
in retinal cells caused by CHM results in progressive vision loss and 
About Nightstar and NSR-REP1 
Nightstar is a leading 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 lead product candidate, NSR-REP1, is comprised of an 
AAV2 vector containing recombinant human complementary DNA, or cDNA, that is 
designed to produce REP1 inside the eye. NSR-REP1 is currently in Phase 3 
development for the treatment of patients with choroideremia, a rare, 
degenerative, genetic retinal disorder that has no current treatments and 
affects approximately one in every 50,000 people. Positive results from Phase 1 
/2 trials of NSR-REP1 were published in The Lancet in 2014 and in The New 
England Journal of Medicine in 2016. In data from 32 patients treated with 
NSR-REP1 across four open-label Phase 1/2 clinical trials, over 90% of treated 
patients maintained their visual acuity over a two-year follow-up period. In 
some cases, substantial improvements in visual acuity were also observed. 
Nightstar's second product candidate, NSR-RPGR, is currently being evaluated in 
a clinical trial known as the XIRIUS trial for the treatment of patients with 
X-linked retinitis pigmentosa, an inherited X-linked recessive retinal disease 
that affects approximately one in every 40,000 people. 
For more information about Nightstar or its clinical trials, please visit 
Cautionary Language Concerning Forward-Looking Statements 
This press release contains "forward-looking statements" within the meaning of 
the Private Securities Litigation Reform Act of 1995. The words "believe," 
"anticipate," "intend," "estimate," "will," "may," "should," "expect" or other 
similar expressions are intended to identify forward-looking statements, 
although not all forward-looking statements contain these identifying words. 
All statements contained in this press release other than statements of 
historical facts are forward-looking statements, including, without limitation: 
statements about our planned and ongoing clinical trials for NSR-REP1, 
including our Phase 3 STAR trial in choroideremia, the continued clinical 
development of our pipeline, the timelines associated with our research and 
development programs including the timing of patient enrollment and the release 
of data from ongoing clinical trials and studies, whether the receipt of 
regenerative medicines advanced therapy designation for NSR-REP1 in 
choroideremia will meaningfully impact the development and review of NSR-REP1 
by the FDA or the likelihood that the product candidate will be found safe and 
effective, the prevalence of patient populations for our targeted indications, 
and the utility of prior preclinical and clinical data in determining future 
clinical results. These forward-looking statements are based on management's 
current expectations of future events and are subject to a number of involve 
substantial known and unknown risks, uncertainties and other factors that may 
cause our actual results, levels of activity, performance or achievements to be 
materially different from the information expressed or implied by these 
forward-looking statements, including the risks and uncertainties set forth in 
Item 3.D. "Risk Factors" section of our Annual Report on Form 20-F for the year 
ended December 31, 2017 and subsequent  reports that we file with the U.S. 
Securities and Exchange Commission We may not actually achieve the plans, 
intentions, estimates or expectations disclosed in our forward-looking 
statements, and you should not place undue reliance on our forward-looking 
statements. Actual results or events could differ materially from the plans, 
intentions, estimates and expectations disclosed in the forward-looking 
statements we make. We anticipate that subsequent events and developments will 
cause our views to change. We are under no duty to update any of these 
forward-looking statements after the date of this press release to conform 
these statements to actual results or revised expectations, except as required 
by law. You should, therefore, not rely on these forward-looking statements as 
representing our views as of any date subsequent to the date of this press 
Senthil Sundaram, Chief Financial Officer 
Brian Luque, Sr. Manager, Investor Relations 

(END) Dow Jones Newswires

June 14, 2018 06:07 ET (10:07 GMT)

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