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Promising Research Presented at 2009 ARVO Annual Meeting
Every year, thousands of eye researchers from around the world convene at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting to share the latest advances and breakthroughs in eye and vision research. ARVO’s Annual Meeting is regarded as the premier annual research conference for the ophthalmological community.
Approximately 225 retinal research projects funded by the Foundation Fighting Blindness were presented at this year’s meeting, held May 3-7 in Ft. Lauderdale. Many of the Foundation’s commercial partners made press announcements and presented their findings at the meeting, as well.
Highlights in retinal degenerative disease research from the 2009 ARVO meeting include:
Leading Retinal Disease Gene Therapy Developer Receives $11.8 Million Investment
Applied Genetic Technologies Corporation (AGTC), a clinical-stage gene therapy development company, announced that it has raised $11.8 million in venture capital funding to advance treatments for the retinal degenerative diseases Leber congenital amaurosis (LCA) and achromatopsia. The investment will also be used to fund research for an early onset form of emphysema.
AGTC is extending the Phase I clinical study of gene therapy for LCA underway at the University of Pennsylvania and the University of Florida. Young adults in the study who were virtually blind have had some vision restored. The company is preparing for a Phase I/II study of gene therapy for LCA, which will include younger participants and higher dose treatments. LCA is a severe form of retinitis pigmentosa that causes children to be born blind or with severe vision loss.
Neurotech’s ECT Stabilizes Vision in People with Dry AMD
As reported by the Foundation Fighting Blindness in March 2009, Neurotech has announced that its innovative Encapsulated Cell Technology (ECT) preserved vision in a majority of the 51 people with dry age-related macular degeneration who participated in a Phase II clinical trial. Neurotech reported that 96.3 percent of participants receiving the high dose treatment had stable vision over a 12-month period. People with better visual acuity at the start of the treatment — 20/63 or better — appeared to benefit most.
The ECT is a tiny device — the size of a grain of rice — that is implanted into the eye, where it provides long-term, sustained release of a vision-preserving protein. The Foundation Fighting Blindness funded preclinical studies of ECT, and is currently funding two Phase II/III clinical studies of the treatment for retinitis pigmentosa, Usher syndrome, and choroideremia. Results from Phase II/III studies of the treatment for these conditions are forthcoming.
New Investment to Boost Gene Therapy Development
Biopharmaceutical company Oxford BioMedica has established a collaboration with Sanofi-aventis, a major international pharmaceutical company, to develop and commercialize gene therapy treatments for vision-robbing retinal degenerative diseases that affect tens of millions of people around the world.
Oxford BioMedica is a key partner of the National Neurovision Research Institute, the Foundation’s clinical trial support organization.
Based on the agreement, Oxford BioMedica will receive an upfront payment of $26 million and a further $24 million from Sanofi-aventis over a three-year period.
The collaboration will significantly bolster the development of the following gene therapy products: StarGen™ for the treatment of Stargardt disease, a form of early-onset macular degeneration; UshStat™ for the treatment of Usher syndrome, the leading cause of deaf-blindness; and RetinoStat® for the treatment of age-related macular degeneration, the leading cause of vision loss in people 55 and older in developed countries.
The treatments will utilize Oxford BioMedica’s LentiVector® gene delivery technology to deliver healthy vision-saving genes to the retina.
Fenretinide Slows Progression of Dry AMD by 45 Percent
Fenretinide, a drug being developed by Sirion Therapeutics, slowed the progression of advanced dry age-related macular degeneration by 45 percent in people receiving a higher dose of the treatment in a Phase II clinical trial.
Sirion has been granted a Fast Track designation for the treatment by the FDA. The designation provides a quicker review of a New Drug Application for fenretinide — six months instead of ten months — enabling the treatment, if approved, to get to the market more quickly.
The Foundation Fighting Blindness funded earlier preclinical studies of fenretinide, which demonstrated the drug’s potential for slowing vision loss in people affected by dry AMD, Stargardt disease, Best disease, and other conditions characterized by the buildup of toxic waste products in the retina.
Pfizer to Fund Development of Stem Cell Treatment for AMD
Embryonic stem cells are taking an important step toward becoming a treatment for age-related macular degeneration thanks to Pfizer’s investment in the development of this promising therapeutic approach at University College London (UCL).
Researchers at UCL hope to begin a clinical study of the stem cell treatment in two years. If it is eventually proven to be safe and effective in restoring sight in humans, the treatment could be available seven years from now.
The investigators will use embryonic stem cells to produce new retinal cells, which can replace retinal tissue lost to AMD. If successful, the treatment could restore the central vision typically lost to both the dry and wet forms of AMD.
VEGF-Trap-Eye Improves Vision in People with Wet AMD
Regeneron, a biopharmaceutical company in Tarrytown, New York, reported that their emerging treatment VEGF-Trap-Eye significantly improved visual acuity for 157 people with wet age-related macular degeneration participating in a Phase II clinical study at Harvard University Medical School.
On average, participants gained five letters of visual acuity after three monthly treatments, and their improved visual acuity was maintained for 18 months with three additional “as needed” treatments.
Participants with wet AMD in two other VEGF-Trap-Eye Phase II clinical studies — at Wilmer Eye Institute, Johns Hopkins University School of Medicine and New York University School of Medicine and Manhattan Eye, Ear and Throat Hospital — also demonstrated improved visual acuity.
Regeneron is currently recruiting 1,200 people for a Phase III clinical study of VEGF-Trap-Eye.
The treatment is administered through an intravitreal injection.
Recent Retinitis Pigmentosa Articles
- Bone Marrow Stem Cells in Clinical Trial for Retinal Diseases
- GenSight Receives $41.3 Million for Ocular Gene Therapy Development
- Stem Cell Pioneers Creating Retinal Patch to Restore Vision
- XLRS Natural History Study Beginning in Portland, Oregon
- Six Emerging Retinal Therapies Receive $3.1 Million in Foundation Funding