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Some takeaways...ARWR showed some 'muscle' today!November 12, 4 presentations (2 @ AASLD, 2 at AHA) and Nov 13-16th in Dublin (EORTC/AACR/NCI Symposium) late-breaker on ARO-HIF2. Muscle as a new target (good KD of mRNA shown)-lots of muscle types. ARO-ANG3 is not just for CVD anymore...(also NASH)...the fact it will help CVD at same time as NASH may enamor it to FDA, since most NASH patients die of CVD...so if you have benefits to both, all the better. Must say, enjoyed the condemnation with faint praise of antisense: A neat scientific concept, but hard to compete with RNAi...good for proof of conecpt, but not for the clinical drug... (in the context of triglyceride KD). APOC3: Phase 2/3 pivotals will be pursued for pancreatitis orphans, saving 6 months to a year. ENaC: looking to get closer to the Amiloride mucus clearance level in sheep model (22% vs 12%), so working on improvements general observations: safety not an issue so far,TRiM allows good therapeutic index, ARWR approach gives them a lot of confidence. In sum, worth a listen! |
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Msg # | Subject | Author | Recs | Date Posted |
9834 | Re: Some takeaways...ARWR showed some 'muscle' today! | evernewbliss | 0 | 10/16/2018 7:09:26 PM |