Krystal Biotech presents preclinical ocular gene therapy data at ARVO 2024

Krystal Biotech, Inc.

Subretinal and suprachoroidal injections achieved widespread mCherry expression across the retina in mice.
Vector transduced both photoreceptors (RHO+) and RPE cells (RPE65+) with minimal inflammation.
Intravitreal injection resulted in mCherry signal in cornea, iris, and ciliary body, not retina.
Low systemic dissemination detected; platform shows potential for inherited retinal diseases (IRDs).

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