AGTC and National Neurovision Research Institute Collaborate, Funding Research in two Genetic Retinal Diseases

* Reuters is not responsible for the content in this press release.

Mon Jun 29, 2009 1:50pm EDT

Combined Funds and Resources to Be Used to Further Demonstrate the AGTC`s AAV
System`s Ability to Deliver Sustainable Treatments for Human Retinal Diseases
GAINESVILLE, Fla.--(Business Wire)--
Applied Genetic Technologies Corporation (AGTC), a privately-held, clinical
stage biotechnology company developing novel systems to deliver human
therapeutics, announces that AGTC has entered into an agreement with the
National Neurovision Research Institute (NNRI), the clinical trial support
organization for the Foundation Fighting Blindness(FFB), to collaborate in
experiments using the AAV delivery system in the treatment of two genetic
retinal diseases known to cause blindness at an early age. The research will be
coordinated by AGTC and will be conducted at The University of Florida, Oregon
Health & Science University, The University of Pennsylvania, and The University
of British Columbia. 

The collaboration will focus on development of treatments for two of the more
common genetic retinal diseases that cause blindness at a very early age:
X-Linked Retinoschisis (XLRS) and Achromatopsia. "We are delighted to expand our
strong relationship with the FFB and the NNRI through this collaboration," said
Sue Washer, President and CEO of AGTC. "We continue to be encouraged by the data
supporting the AAV vector system`s ability to provide sustained delivery and
expression of therapeutic levels of many different biologics in the eye with
minimal observable toxicity to date in either animal or human testing. There are
hundreds of thousands of patients suffering from retinal diseases who currently
have no treatment options and this research collaboration is another step
towards using the AAV delivery system to address this unmet need and improve the
patients` quality of life." 

"This collaboration is a tremendous boost for the development of gene therapy
products for retinal degenerative diseases and NNRI`s partnership with AGTC
accelerates these emerging treatments into and through the clinical trial
process," said Stephen Rose, Ph.D., Chief Research Officer, Foundation Fighting
Blindness. "It affirms the great potential for science guided foundations and
academic researchers to work in partnership with commercial firms like AGTC that
have the commitment and experience to bring these promising treatments directly
to patients." 

XLRS is an inherited form of retinal degeneration affecting young boys. Patients
present with poor vision either in infancy or at school age. Visual acuity
usually worsens during the teenage years and then stabilizes until complicated
by vitreous hemorrhage or retinal detachment during adulthood. There is no
treatment available for the retinal degeneration in XLRS, which affects
approximately 34,000 patients in the US and Europe. Previous research has shown
promising signs of efficacy in mouse models and this collaboration will explore
safety and efficacy in primates. 

Achromatopsiais an inherited condition that presents at birth with impaired
visual acuity, lack of color discrimination and extreme light sensitivity
resulting in daytime blindness. There is no specific treatment for
Achromatopsia, although deep red tinted spectacles or contact lenses can reduce
symptoms of light sensitivity. Approximately 22,000 patients in the US and
Europe suffer from this disease. Previous research has shown promising signs of
efficacy in dog models and this collaboration will enable expanded safety and
efficacy studies. 

AboutAGTC: AGTC is focused on the research and development of novel therapeutics
for patients with unmet medical needs utilizing AGTC`s proprietary,
non-pathogenic adeno-associated virus (AAV) delivery system. AGTC has
demonstrated that this system can be used to deliver a normal form of a gene in
both animals and humans thus allowing their own body to produce sustained
therapeutic levels of important biologics. The Company`s most advanced programs
in development are treatments for Alpha-1 antitrypsin deficiency, a disease
causing a progressive loss of lung function, and Leber`s Congenital Amaurosis,
an inherited condition causing early blindness. Both utilize AGTC`s proprietary
AAV system and production methods. AGTC has licensed a significant portion of
its intellectual property from the University of Florida where researchers
originated this ground-breaking work and has received significant financing from
some of the world`s leading venture capital firms: InterWest Partners,
Intersouth Partners and MedImmune Ventures. For more information see
www.agtc.com. 

About Foundation Fighting Blindness (FFB):FFB is the largest source of
non-governmental funding for retinal degenerative disease research in the world.
The urgent mission of the Foundation Fighting Blindness is to drive the research
that will provide preventions, treatments and cures for people affected by
retinitis pigmentosa, macular degeneration, Usher syndrome, and the entire
spectrum of retinal degenerative diseases. The Foundation has invested over $140
million to provide seed money for scientific research of diseases of the retina,
which cause blindness. Further information is available at
www.FightBlindness.org. 

About National Neurovision Research Institute (NNRI): NNRI is a
recently-established non-profit support organization of the Foundation Fighting
Blindness, the leading non-government funding source for inherited orphan
retinal degeneration research. The mission of NNRI is to accelerate the
translation of laboratory-based research into clinical trials for treatments and
cures of retinal degenerative diseases. It is a medical research institute that
obtains support from government agencies, corporations and private foundations.
It may also receive royalties or licensing fees from the drug discovery
processes and commercialization of new therapies. Further information is
available at www.NNRI.info. 



Applied Genetic Technologies Corporation
Susan B. Washer, CEO, 386-462-2204, Ext. 208
swasher@agtc.com

Copyright Business Wire 2009

Comments (0)
This discussion is now closed. We welcome comments on our articles for a limited period after their publication.