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Oxford Biomedica PLC
10 October 2016

Oxford BioMedica Announces Publication of Pioneering RetinoStat® (OXB-201) Phase I Study Data in the Journal, Human Gene Therapy


-- RetinoStat® met its primary endpoint, demonstrating favourable safety and tolerability profiles


Oxford, UK - 10 October 2016: Oxford BioMedica plc (LSE:OXB) ("Oxford BioMedica" or "the Group"), a leading gene and cell therapy group, today announces the publication in the journal Human Gene Therapy of the previously announced ground-breaking results from the RetinoStat® (OXB-201) Phase I study in patients with advanced wet age-related macular degeneration (AMD) on 6 May 2016. According to key published findings in the associated peer-reviewed paper, RetinoStat® demonstrated a favourable safety profile and led to robust, reproducible sustained expression of endostatin and angiostatin in the eye.


The Phase I study was primarily designed to evaluate the safety and tolerability of RetinoStat® for the treatment of severe wet AMD following a single subretinal injection and represented the first time a lentiviral based vector had been administered to the human eye. Twenty-one subjects with highly fibrotic retinas who were refractory to anti-VEGF therapy following a prior responsive history were treated. As previously announced the results of the Phase I study indicated that RetinoStat® met the primary endpoint of safety and tolerability. Importantly, therapeutic gene expression, measured in these patients as a secondary study endpoint, was found to be dose-dependent and maintained at the last measurement (2.5 years in 8 subjects and >4 years in two subjects).


Peter A. Campochiaro, the Eccles Professor of Ophthalmology and Neuroscience at the Wilmer Eye Institute was the lead author and principal and coordinating investigator. Andreas K. Lauer (Oregon Health Sciences Center of the University of Oregon) and Elliott H. Sohn (University of Iowa) were the other investigators of the study.


The online publication in Human Gene Therapy is entitled: "Lentiviral Vector Gene Transfer of Endostatin/Angiostatin for Macular Degeneration (GEM) Study". Please follow this link to read the paper in full:


Highlights from the Phase I study:


Safety: RetinoStat®, the first ocular lentiviral gene therapy to be administered in Man, has demonstrated a favourable safety profile with no serious adverse events related to the product observed to date.


Pharmacokinetics: significant expression of both therapeutic transgenes was directly measured in patient aqueous humour samples and showed a dose response that was stable and persistent in all patients (out to 4.5 years so far in the earliest patient enrolled). 


Oxford BioMedica is evaluating the optimal way to progress the clinical development for RetinoStat®.


Commenting on the publication, John Dawson, Chief Executive Officer of Oxford BioMedica, said: "Like the ProSavin® trial before it, the RetinoStat® First-in-Man study was a clinical trial of 'firsts': the first ever trial to directly administer a lentiviral vector-based product to the eye, the first directly administered lentiviral vector trial in the USA, the first direct measurement of an ocular gene therapy transgene during a study and the first reporting of data showing direct demonstration of long-lasting expression of an ocular gene therapy in human subjects.


"These peer-reviewed published results further validate the ground-breaking utility of our LentiVector® delivery platform for the treatment of chronic disease."





For further information, please contact:



Oxford BioMedica plc:

John Dawson, Chief Executive Officer

Tim Watts, Chief Financial Officer


Tel: +44 (0)1865 783 000


Financial and corporate communications enquiries:

Consilium Strategic Communications

Mary-Jane Elliott/Matthew Neal/Chris Welsh/Laura Thornton




Tel: +44 (0)20 3709 5700



About Oxford BioMedica®

Oxford BioMedica (LSE:OXB) is a leading gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford BioMedica and its subsidiaries (the "Group") have built a sector leading lentiviral vector delivery platform (LentiVector®) through which the Group develops in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology and CNS disorders. The Group has also entered into a number of partnerships, including with Novartis, Sanofi, GSK, and Immune Design, through which it has long-term economic interests in other potential gene and cell therapy products. Oxford BioMedica is based across several locations in Oxfordshire, UK and employs more than 230 people. Further information is available at



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