Bijgaande Seeking Alpha citaten (van mijn favoriete schrijver Culleraa, een aandelen analist in ruste) zijn volgens mij nog steeds van toepassing:
Who said Editas can cure anything? In LCA 10 a one-time, hugely risky surgery which affects 20% of retina, with guaranteed viral side-effects.. for those who can take the AAV. The logistics are a nightmare... if the effects stop, treated retinal cells start to die very soon - so how do you do monitor for that?
RNA treatment is logistically and AE-wise the same as treating wet-AMD, except maybe twice a year.
Sep 11, 2018. 05:30 PMLink
Sangamo: It Worked, Didn't It? - Marty Chilberg
If you mean the IND for LCA10, I expect Editas to delete that program asap. It would be embarrassing to use crispr there when Proqr just showed (almost proved) that anti-sense RNA is safe and highly effective in LCA10 and got FDA approval to go straight into phase 3 registration trial from the phase 1. In fact, the RNA approach will likely take over all retinal disease states where applicable.
Sep 11, 2018. 04:56 PMLink
Sangamo: It Worked, Didn't It? - Marty Chilberg