Current holders will remain holders of the platform which it seems currently is only effective in the eye.
Current focus is on ASO delivery into the eye, so that’s where validation of the technology is most advanced.
But I think your statement fails to reflect the fact that IV animal experiments (in mouse models) showed evidence of both efficacy and low toxicity in delivering PYC CPP-ASOs in models of both Duchenne Muscular Dystrophy (for which Sarepta has had eteplirsen approved) and Spinal Muscular Atrophy (for which Ionis has had nusinersen approved).
Effect was demonstrated in muscles in a range of locations (including cardiac, diaphragm and tibia).
PYC Price at posting:
4.6¢ Sentiment: Hold Disclosure: Held
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