University College London, Moorfields Eye Hospital and Targeted
Genetics Initiate Phase I/
LONDON and SEATTLE, WA -- (MARKET WIRE) -- May 01, 2007 -- University College London's (UCL) Institute of Ophthalmology, Moorfields Eye Hospital and Targeted Genetics Corporation (NASDAQ: TGEN) today announced the initiation of a Phase I/II clinical trial to test an innovative approach to treating a form of childhood blindness. The trial, funded by the UK Department of Health is the first of its kind and could have a significant impact on future treatments for eye disease.
The trial involves adults and children who have a progressive deterioration in vision caused by an abnormality in the RPE65 gene. In affected individuals, this defect prevents normal function of the retina, the light-sensitive layer of cells at the back of the eye. There are currently no effective treatments for this condition.
The approach being evaluated in this clinical trial utilizes an adeno-associated virus (AAV) vector to deliver a normal copy of the RPE65 gene into the cells of the retina to help them to function normally. Targeted Genetics, the leader in the development and manufacture of AAV-based product candidates, manufactured the vector that will be used in this trial.
"We have been developing gene-based treatments for eye disease for almost 15 years but until now we have been evaluating the technology only in the laboratory," said Professor Ali. "Testing it for the first time in patients is very important and exciting and represents a significant step towards establishing gene therapy for the treatment of many different eye conditions."
Work using animal models has demonstrated that this AAV-mediated delivery of RPE65 can improve and preserve vision. The purpose of this clinical study is to evaluate the safety and efficacy of this approach in patients.
The team from UCL Institute of Ophthalmology and Moorfields Eye Hospital, led by Professor Robin Ali, includes leading eye surgeon Mr. James Bainbridge and leading retinal specialist Professor Tony Moore.
"So far the operation has been performed in young adult patients who developed the condition as children," said Mr. James Bainbridge, who leads the surgical team. "The data available thus far, although preliminary, suggest that AAV-based delivery of genes to extremely fragile sites within the eye can be accomplished without significant complications."
"Additional data will be needed in order to assess the outcome of this approach fully," said Professor Moore, "However we expect that the best outcomes will be observed in younger patients."
About University College London
UCL Institute of Ophthalmology is one of a number of specialised research centres linked to University College London and is, together with Moorfields Eye Hospital, one of the leading centres for eye research. The Institute scored a 5*A (highest point) in the last Research Assessment Exercise. The Institute is committed to a multi-disciplinary research portfolio that furthers an understanding of the eye and visual system linked with clinical investigations targeted to specific problems in the prevention and treatment of eye disease. The combination of the Institute's research resource with the resources of Moorfields Eye Hospital, which has the largest ophthalmic patient population in the Western World, opens the way for advances at the forefront of vision research.
About Moorfields Eye Hospital
Moorfields Eye Hospital NHS Foundation Trust is the world's oldest specialist eye hospital. It is a world-renowned centre for ophthalmic treatment, teaching and research.
About Targeted Genetics
Targeted Genetics Corporation is a biotechnology company committed to the development of innovative targeted molecular therapies for the prevention and treatment of acquired and inherited diseases with significant unmet medical need. Targeted Genetics' proprietary Adeno-Associated Virus (AAV) technology platform allows it to deliver genes that encode proteins to increase gene function or RNAi to decrease or silence gene function. Targeted Genetics' product development efforts target inflammatory arthritis, AIDS prophylaxis, congestive heart failure and Huntington's disease. To learn more about Targeted Genetics, visit Targeted Genetics' website at www.targetedgenetics.com.
Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:
This release contains forward-looking statements regarding the data to be collected in this trial, the establishment or determination of efficacy endpoints from the data collected in the trial, the timely and complete accrual of patients in the trial and our ability to commercialize and other statements about our plans, objectives, intentions and expectations. These statements, involve current expectations, forecasts of future events and other statements that are not historical facts. Inaccurate assumptions and known and unknown risks and uncertainties can affect the accuracy of forward-looking statements. Factors that could affect our actual results include, but are not limited to, our ability to obtain, maintain and protect our intellectual property, our ability to raise capital when needed, our ability to recruit and enroll suitable trial participants, the timing, nature and results of research and clinical trials, potential development of alternative technologies or more effective processes by competitors, and our ability to obtain and maintain regulatory or institutional approvals, as well as other risk factors described in Item 1A. Risk Factors in our report on Form 10-K for the year ended December 31, 2006. You should not rely unduly on these forward-looking statements, which apply only as of the date of this release. We undertake no duty to publicly announce or report revisions to these statements as new information becomes available that may change our expectations.
For further information, or to arrange an interview with Professor Robin Ali, please contact Ruth Metcalfe at UCL Media Relations on: +44 (0)20 7679 9739, +44 (0)7990 675 947 (mobile) or in out of hours 44 (0)7917 271 364 Notes to editors:
1. The team is supported by funding from the Department of Health, Sir Jules Thorn Charitable Trust, The Wellcome Trust, The British RP Society, The European Union (EVI Genoret and Clinigene programmes), The Medical Research Council, Foundation Fighting Blindness USA, Fight for Sight and The Ulverscroft Foundation.
2. Robin Ali is Professor of Human Molecular Genetics at UCL Institute of Ophthalmology and Head of Division of Molecular Therapy.
3. James Bainbridge is a Wellcome Trust Advanced Fellow at UCL Institute of Ophthalmology and Consultant Ophthalmologist at Moorfields Eye Hospital
4. Tony Moore is Professor of Ophthalmology at UCL Institute of Ophthalmology and Consultant Ophthalmologist at Moorfields Eye Hospital and the Hospital for Children Great Ormond St London.
Targeted Genetics' Investor and Media Contact Stacie D. Byars Director, Communications Targeted Genetics Corporation (206) 521-7392
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