I found this interesting
The intra-vitreal injections resulted in exon skipping in the anterior and posterior segment of the eye….
A more detailed data pack on delivery within the eye as a target tissue will be provided in the future.
There are a number of eye diseases being targeted with ASO therapies, including by German biotech Isarna (glaucoma), Wave Life Sciences (rare, inherited eye diseases) and Ionis Pharmaceuticals/Roche (AMD).
Last week, as discussed on the other current thread, Biogen acquired the ophthalmology pipeline of gene therapy biotech, Nightstar Therapeutics, for $677 miillion. Nightstar’s lead therapies use AAV vectors and are administered by subretinal injection.
https://labiotech.eu/medical/isarna-glaucoma-isth0036-phase-i/
https://wavelifesciences.gcs-web.co...es-develop-programs-rare-genetic-eye-diseases
https://wavelifesciences.gcs-web.com/static-files/38c3e333-a7cc-4557-8be4-40ce9b361c93
Ann: Flagship Program - Successful Animal Models, page-11
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