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Gene therapy progresses through efforts of small companies, attracts big investments
BARCELONA — Gene therapy has greatly progressed in rare disorders, such as Leber’s congenital amaurosis, but several applications are under evaluation and show promise, according to Jose-Alain Sahel, MD, who chaired a symposium on the topic at the Ophthalmology Futures European Forum. “Gene therapy has also been tested as a novel form of drug delivery to the retina for AMD with anti-VEGFs. Results are so far not perfectly convincing, but there is a rationale to continue research in this direction,” he said.