PYC's Lead Investigational Drug, VP-001, Demonstrates Another Key Functional Improvement in Patient-Derived Models

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NEW YORK and PERTH, Australia, March 2, 2021 /PRNewswire/ -- PYC Therapeutics (ASX: PYC), a biotechnology company developing a new generation of precision RNA therapeutics to change the lives of patients with inherited diseases, today announced that the Company's lead investigational drug, VP-001, for the treatment of RP11 has restored function of the Retinal Pigment Epithelium, the target cells for the therapy, in patient-derived models of the disease.
Figure 1. Structure of the retina and changes in RP-11 patients
Image created using Biorender.com
Figure 2. VP-001 treatment improves barrier function in penetrant patient iPSC-RPE Barrier function is assessed by trans-epithelial electrical resistance, T.E.E.R. (measured in Ω.cm2), with increasing Ω.cm2 representing increasing barrier function. Statistical significance calculated as one-way ANOVA *= p ≤ 0.05 in cells from a single patient across two technical replicates

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