Model Background: Today's post anti-VEGF therapy era for macular degeneration, calls for potential cures with less adverse effects or risk of vision loss. Pentagrit's zebrafish xenotransplanted with human retinal pigmented epithelial cells is an ideal model to study delivery of target genes for gene therapy. Human retinal epithelial cells lacking ABCA4 or STGD1 are transplanted and successful carrier fish of mutant human retinal epithelial cells lines are administered with varying dose of vectors.
Endpoint Measure for Therapeutic Efficacy on Comparison with Emixustat
1. Target protein concentration in aqueous humor
2. Mean Change in Visual Activity
3. Central Retinal Thickness
4. Presence of pigment epithelium detachment
5. Presence of Serous-vascularized or fibrovascular condition
Key Barrier in Candidate Identification: Translational barriers have been the largest in the ophthalmic gene therapy domain, owing to lack of established organoids and phenotyping standards. Pentagrit's humanized zebrafish model offers the versatility of assessing drug activity in a human ophthalmic environment providing data that can be translated to clinical trial dosing and outcome. We therefore enable a quicker reach to the market for macular degeneration candidates.