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Sustained Therapeutic delivery for the eye

Current Treatments

Vascular endothelial growth factor (VEGF) is a protein known to play a central role in the growth of new blood vessels. These new blood vessels tend to leak fluid and blood under the macula, leading to the loss of central vision. A number of therapies have been developed to block the effects of VEGF by binding to and sequestering the protein. The most common anti-VEGF treatments are Lucentis, a recombinant humanized monoclonal antibody fragment that binds to and inhibits VEGF proteins in the eye, EYLEA, a recombinant fusion protein containing portions of the human VEGF receptor that binds to soluble VEGF, and Avastin, a recombinant human monoclonal antibody that binds to VEGF which is also approved as an anti-cancer agent.

While these therapies have proven to be effective, they require injections into the patient’s eye every four to eight weeks. The required frequency of administration for these therapies is burdensome for patients, leading many to terminate treatment or not comply with the prescribed regimen, resulting in patients experiencing vision loss. According to the European Journal of Ophthalmology (2011), 36% of patients who received anti-VEGF treatment for at least six months had stopped receiving therapy within a year. Of these patients, 59% had lost visual acuity.

More great reads

The results of a Phase 1, 52-week trial of Avalanche Biotechnologies’ rAAV.sFLT-1 (AVA-101) gene therapy for the treatment of wet age-related macular degeneration (wAMD) have been published online by The Lancet. The study sought to evaluate the safety of rAAV.sFLT-1 via a single subretinal injection. Results of the study demonstrate that rAAV.sFLT-1 was safe and well tolerated, and support ocular gene therapy as a potential long-term treatment option for wAMD.

“The use of gene therapy to reprogramme genetically normal cells of the retina to take on additional functions opens a new chapter in potential AAV applications.”
– Dr. Robert MacLaren, Moorsfield Eye Hospital

The complete article and editorial commentary by Dr. MacLaren, are available from The Lancet.

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