This is what I would call an "edge-of-the-seat announcement" we are awaiting.
By now, at least 2 of the 3 patients in the high-dose SAD Cohort 4 (75mcg), who received second doses in early August, have passed the 8 week re-dosing mark and should have been assessed prior to a third dose being administered.
So I am hoping that we will find out how efficacy is faring over a 4 month period after 2 high doses.
We might also get an efficacy update on the patients in the SAD Cohort 3 (30mcg), for whom promising efficacy data following a single dose has already been reported. Two of these patients were being scheduled for a second dose in August Week 2 so furher efficacy data might also be available for them.
An update on safety will also be crucial as in previous antisense trials in retinal disease, glaucoma was seen in some patients from the 6 month mark.
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This is what I would call an "edge-of-the-seat announcement" we...
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