ProQR’s lead program is indicated for Leber’s congenital amaurosis (LCA) Type 10, caused by mutations in CEP290. This represents approx. 15% of LCA cases and is estimated to impact just 2,000 people in the western world.
Though PYC might have compared its dose level for effective delivery of ASO to the retinal cells to those used by ProQR in its preclinical studies for LCA Type 10, I don’t take that to necessarily mean that PYC intends to target the same indication.
Anyway…it seems we will have the chance to have some questions answered on September 4.
PYC Price at posting:
3.8¢ Sentiment: Hold Disclosure: Held
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(20min delay)