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"The future promise of advanced therapies in ophthalmology"

Six years after the first approved gene therapy in ophthalmology, the field is seeing a flurry of activity in innovative cell and gene therapy (CGT) studies addressing high unmet needs. Here’s what could be in store for the future.

The challenge now is to further develop the field with additional CGTs to address a range of diseases with burdensome or ineffective treatment options. Some of these diseases are extremely rare — an estimated 0.1% of people over 40 develop macular telangiectasia type 21, for example —while others, like age-related macular degeneration, are very common. With their curative intent and some with regenerative potential, such therapies could be strong candidates for these indications. 

The growth of novel CGTs in development for indications in ophthalmology  may also be explained due to the eye’s physiology. Therapies may be administered locally and in small volumes since the organ is relatively compartmentalized. By using targeted delivery of gene therapy vectors, strong inflammatory responses may be avoided by targeting regions of the eye that have immune privilege.  

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