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PYC Therapeutics Highlights Continued Progress of Ocular and CNS Pipeline Programs and U.S. Expansion in First Quarter Update

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NEW YORK and PERTH, Australia, April 30, 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 a first quarter update highlighting the progress of its development pipeline, growth of its U.S. operations and upcoming milestones.
PYC has had an exciting quarter of progress against our Company objectives. We have achieved continued validation of our PPMO technology in our lead candidate VP-001 for retinitis pigmentosa type 11, further development of our ocular pipeline as well as PYC s first set of preclinical data from our CNS discovery efforts demonstrating superior delivery of RNA therapeutic throughout the brain and spinal cord. The potential of our pioneering PPMO technology is vast and we look forward to advancing this technology to provide solutions for patients with inherited o ....

Western Australia , Michael Rosenblatt , Phosphorodiamidate Morpholino Oligomer , Kaggen Ausma , Sahm Nasseri , Leo Vartorella , Deborah Elson Matthew Deyoung , Glenn Noronha , Drug Administration , Executive Officer , Alan Tribe , Ocular Diseases , Investigational New Drug , Nervous System , Chief Development Officer , Chief Business Officer , Board Directors , Jason Haddock , West Coast , Adeno Associated Virus , Mol Ther Methods Clin , Global Burden , Disease Study , Pyc Therapeutics , மேற்கு ஆஸ்திரேலியா , மைக்கேல் ரோசன்ப்ளாட் ,

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 replicate ....

Deborah Elson Matthew Deyoung , Sahm Nasseri , Leo Vartorella , Drug Administration , Retinal Pigment Epithelium , Blood Retina Barrier , Adeno Associated Virus , Investigational New Drug , Cell Penetrating Peptides , Mol Ther Methods Clin , Matthew Deyoung , Pyc Therapeutics , டெபோரா எல்சன் மேத்யூ டியூன் , இரத்தம் விழித்திரை தடை , மோள் தெர் முறைகள் கிளினிக் , மேத்யூ டியூன் , பய்க் சிகிச்சை ,