While the target cells for VP-001 in RP11 are in the Retinal Pigment Epithelium (RPE), the target cells for PYC-001 in ADOA are in the inner retina, within the neural retina.
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Tested in rabbit retina >55% target engagement was seen in the neural retina following a single 10mcg dose of PYC’s PPMO.
Mention was also made in yesterday’s presentation to how the PYC PPMO delivery profile differs from that of a naked antisense approach to ADOA. For evidence of this, review of PYC’s ASX announcement of 3 October 2022 was suggested.
That announcement related to a video poster presentation, “Enhancement of Antisense Oligomer Cell Penetration in Retinal Layers Using a Modular Cell Penetrating Peptide Platform”, which was delivered at the Oligonucleotide Therapeutics Society (OTS) 2022 Annual Meeting in Arizona last October.
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The superior delivery and target engagement achieved using PYC’s PPMO approach in the neural retina compared with using naked antisense is quite outstanding and bodes well for the coming clinical trial in ADOA. The company has also stated that the improved outcomes conferred by PYC’s approach in the inner retina may also offer advantage in the treatment of glaucoma.
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While the target cells for VP-001 in RP11 are in the Retinal...
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