Gene Therapy in Ophthalmology 2022

Gene Therapy in Ophthalmology 2022
---Presented at Medicine 2042
---Thomas Ciulla MD MBA

In ophthalmology and retina, we are so extraordinarily fortunate to be the epicenter of so much gene therapy innovation.

Gene therapy research has focused on the retina, given:
---the presence of post mitotic cells,
---relative immune-privilege that limits inflammatory response,
both small and large animal models,
---direct accessibility via intraocular delivery, and
---the noninvasive ability to monitor for disease progression or therapeutic response.

In addition, there are a large number of monogenic inherited retinal disease, as well as several multibillion dollar markets for non-inherited disease, such as AMD, diabetic retinopathy and diabetic macular edema.

This presentation covers a conceptual framework to organize, better understand and differentiate these therapies.

Very broadly, these differentiating features include an increasing number of strategic categories, novel delivery techniques, and evolving administration procedures.

These innovations can address current challenges such as exploring gene independent approaches for 100’s of rare inherited diseases, transferring genes too large for AAV vectors, producing proteins in mitochondrial disorders, knocking down and replacing genes in autosomal dominant disorders, minimizing ocular inflammatory response, and investigating novel methods of ocular administration