Gene therapies continue to advance in AMD
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WAILEA, Hawaii — Gene therapy is a rapidly advancing innovation in the world of retinal diseases, according to a speaker at Retina 2024.
Allen C. Ho, MD, said gene therapies have already taken hold across medicine, with about 20 therapies obtaining FDA approval.
“Gene therapy is happening now, and it’s something to pay attention to,” he said. “I’m not naive or biased enough to think that this is going to replace our therapies or injections.”
The group of approved treatments includes therapies such as Luxturna (voretigene neparvovec, Spark Therapeutics) for Leber congenital amaurosis and CRISPR exa-cel therapy for sickle cell disease.
Ho said the cutting-edge biofactory gene therapies utilize better vectors and transgenes, as well as improved delivery methods.
In retina care, pivotal trials are currently underway for ABBV-RGX-314 (Regenxbio/AbbVie) in patients with neovascular age-related macular degeneration. Ho said previous studies have demonstrated that the therapy is efficient, increasing protein levels, reducing the need for rescue injections and lowering the treatment burden. The therapy can be administered subretinally or suprachoroidally.
The potential to administer the therapy with a suprachoroidal injection presents a unique opportunity because it can be done in the office. Ho said the injections can be more nuanced, but they have been well tolerated by patients.
Innovation in the retina gene therapy space continues. Several other therapies are in development, including ixoberogene soroparovec (Adverum) and 4D-150 (4DMT) for neovascular AMD and AAV-CD59 (Janssen) for atrophic AMD. Development of GT005 (Novartis/Gyroscope) for atrophic AMD was discontinued due to lack of efficacy.
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Ho AC. Gene therapy for neovascular and atrophic AMD: What can we expect? Presented at: Retina 2024; Jan. 13-19, 2024; Wailea, Hawaii.
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