New Gene Therapy on Horizon for Macular Degeneration

Macular degeneration is devastating. Imagine looking at this posting and being unable to see most of it except maybe the periphery part of the page or cell phone or computer. 

Gene therapy is starting to show promise in many areas. While it will take years to prove the below therapy for macular degernation is better than what is available, it is a step in the right direction as surgeons need to step outside of the box to find better treatment options for these devastating conditions. 

The alternatives currently are very grim so I do hope this proves to be a better alternative. 

Sandra Lora Cremers, MD, FACS 

Surgeons perform first gene therapy procedure aimed at stopping AMD-related blindness 

Researchers have completed the first gene therapy procedure aimed at stopping age-related macular degeneration.

The operation was performed at John Radcliffe Hospital, Oxford, England, by Robert MacLaren, FRCOphth, a professor of ophthalmology at the University of Oxford.

The procedure involved detaching the retina and injecting a virus-containing solution underneath. The modified DNA sequence contained in the virus infects the retinal pigment epithelium cells and corrects the genetic defect that causes AMD, according to a press release from Oxford University Hospitals.

“We’re harnessing the power of the virus, a naturally occurring organism, to deliver the DNA into the patient’s cells. When the virus opens up inside the retinal cell it releases the DNA of the gene we have cloned, and the cell starts making a protein that we think can modify the disease, correcting the imbalance of the inflammation caused by the complement system,” MacLaren said in the release.

“It is an interesting application and approach to this very common cause of vision loss,” Allen C. Ho, MD, Director of the Retina Research Program at Wills Eye Hospital and OSN Retina/Vitreous Board Member, told 

The operation is the first in the phase 1/2 FOCUS trial, an open-label, dose escalation, multicenter study accessing the biological activity of the treatment, which is developed by Gyroscope Therapeutics, a company founded by Syncona. 

“What’s unique about this particular clinical trial is that they are trying gene therapy surgically delivered to the subretinal space to try to modify the disease process of dry macular degeneration,” Ho said. While some clinical trials have looked at injection therapies to treat dry AMD and geographic atrophy, there are currently no proven treatments.

“Using gene therapy for this form of the disease to modify disease progression is interesting and exciting science, but not proven yet,” Ho said. – by Rebecca L. Forand

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