Monday, April 25, 2016

Gene Therapy Offers Hope for Patients with Eye Diseases


Ophthalmic research in gene therapy could lead to treatments for a number of diseases that have hitherto been untreatable, Avalanche Biotechnologies CEO Thomas Chalberg recently told Retina Today. The field is seeing a great deal of interest and innovation, said Chalberg, which could usher in a new generation of medical therapy and a new class of biologics.

Avalanche's current trials focus on treatment for wet age-related macular degeneration. So far, participants in the trials have not exhibited any serious adverse events related to the medication. Avalanche Biotechnologies, a leading developer of ophthalmic gene therapy, also is investigating the potential for gene therapy to treat vascular diseases such as diabetic macular edema and retinal vein occlusion.

“The idea of using DNA as a pharmaceutical is a sort of marvelous and wondrous proposition,” Chalberg said. “But why now? Because there are positive clinical data emerging that demonstrates that gene therapy is working.”

Gene therapy may not be available in clinics for a few years, but research increasingly shows that it could significantly change how retina specialists treat certain ocular diseases.