Targeted Genetics Announces Data From First Gene Therapy Trial for Leber's Congenital Amaurosis in New England Journal of Medici
28 4월 2008 - 5:30AM
Marketwired
FORT LAUDERDALE, FL and SEATTLE, WA today announced encouraging
results from a Phase 1/2 clinical study demonstrating that
treatment of three young adults with Leber's congenital amaurosis
(LCA) with an Adeno-Associated Virus (AAV) vector containing the
RPE 65 coding sequence resulted in improved visual function in one
patient. The study, titled "Effect of Gene Therapy on Visual
Function in Leber's Congenital Amaurosis," by Bainbridge, et al.,
conducted in collaboration with University College London and
Moorfields Hospital, will appear in the May 22 issue of the New
England Journal of Medicine.
This ground-breaking data are also being presented at the
Association for Research in Vision and Ophthalmology (ARVO) 2008
Annual Meeting in Fort Lauderdale, Florida. A presentation will be
given by Dr. James W. B. Bainbridge, the study surgeon, at 10AM
Monday, April 28th, in Room 305 of the Broward County Convention
Center. Dr. Bainbridge and the study's Chief Investigator, Dr.
Robin R. Ali, will be available for questions after the
presentation.
LCA is an inherited retinal degenerative disease characterized
by severe impairment of vision from birth and total blindness by
the third decade of life. There is no treatment currently available
for LCA.
"We are excited to have demonstrated proof-of-principle and
early clinical benefit, and will be enrolling additional patients
in this study. This approach provides promise for treating LCA
caused by RPE65, and eventually, other forms of inherited
blindness," stated Professor Robin Ali, University College of
London Institute of Ophthalmology and Moorfields Eye Hospital, who
led the study. "These findings support further clinical studies of
this innovative approach for the treatment of LCA and many
different eye conditions."
In the Phase 1/2 study there were no serious adverse events, and
in one subject there was a consistent improvement in visual
function as measured by visual field tests and improvement in
subjective tests of visual mobility.
"The fact that this approach helped one of the patients to have
better visual function leading to increased visual mobility is very
exciting. Of course, additional studies are needed in order to
assess this approach fully, including the expansion of the study to
include younger children, but these initial results suggest that
AAV-based delivery of genes in the eye can be accomplished," said
Barrie J. Carter, Ph.D., executive vice president and chief
scientific officer of Targeted Genetics and a co-author of the
publication. "We believe gene therapy holds great promise in the
creation of a broad new class of innovative medicines and Targeted
Genetics is well positioned to participate in this
opportunity."
Extensive preclinical testing demonstrated that this
AAV-mediated delivery of RPE65 can improve and preserve vision. In
the open label, single center, Phase 1/2 clinical study, three
young adults between the ages of 17 and 23 years of age with
early-onset severe retinal dystrophy, were administered
subretinally a single injection of the AAV vector expressing RPE65.
In each subject, the eye with the worse acuity was selected as the
study eye and the other was used as a control. The subject who
showed the objective response probably had less advanced retinal
disease at baseline than the other two subjects. The purpose of
this study was to determine whether gene therapy for retinal
dystrophy caused by RPE 5 mutations was associated with immediately
obvious adverse events (primary endpoint) and whether efficacy
could be demonstrated in humans (secondary endpoint). Targeted
Genetics, a leader in the development and manufacture of AAV-based
product candidates, manufactured the vector that is being used in
this trial.
These data also were presented today at ARVO by James W.
Bainbridge, Wellcome Trust Advanced Fellow at UCL Institute of
Ophthalmology and Consultant Ophthalmologist at Moorfields Eye
Hospital, at the Association for Research in Vision and
Ophthalmology annual meeting in Ft. Lauderdale, Florida.
About Targeted Genetics Corporation
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.
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Company's liquidity and financial resources, its ability to fund
ongoing and future operations, its business strategy and product
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the LCA study, the potential impact of the LCA study on our results
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Investor and Media Contact: Stacie D. Byars WeissComm Partners
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