Thanks SoT, I have listened to the Canaccord presentation a number of times. There is an observation around the 23 minute mark in relation to an exchange between Michelle Gilson and Sahm Nasseri that gives me a high level of confidence that the program will successfully translate to the clinic.
The VP-001 program was selected because of an observation that patients who have the mutation but are asymptomatic naturally express higher levels of the PRPF31 protein compared to patients who have both the mutation and the disease. PYC target the negative regulator gene of PRPF31 by toggling down the negative regulator so that the protein can be upregulated to levels consistent with asymptomatic patients. Just brilliant.
The scientific team at PYC realised that if they could replicate what happens in asymptomatic patients they could arrest the cause of blindness in Retinitis Pigmentosa Type 11. In essence, as remarked by Gilson, they let nature run the experiment.
PYC Price at posting:
16.0¢ Sentiment: Buy Disclosure: Held
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