tag:blogger.com,1999:blog-7857054149675424609.post1002982971659837273..comments2024-02-24T08:58:08.581+00:00Comments on RNAi Therapeutics: An RNAi Therapeutic Could Be Last Man Standing in PCSK9Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.comBlogger14125tag:blogger.com,1999:blog-7857054149675424609.post-89312811950807267382014-03-12T17:19:59.956+00:002014-03-12T17:19:59.956+00:00Right. I have full faith in Crook's unbiased c...Right. I have full faith in Crook's unbiased conclusions such when he stated during the keynote address at the 2012 OTS meeting in Boston that 'mipomersen has no side effects'. Dirk Hausseckerhttps://www.blogger.com/profile/18320439857875629714noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-22188059585153289562014-03-11T19:59:53.768+00:002014-03-11T19:59:53.768+00:00The paper you cite, J Lipid Res. 2013 Feb;54(2):56...The paper you cite, J Lipid Res. 2013 Feb;54(2):561-6, shows that normal levels of a mutant protein, missense SNP R46L, does not appear to cause neurocognitive defects.<br />The paper to which you link, N Engl J Med 2006; 354:1264-1272, discusses 3 variants, R46L, Y142X, and C679X, only one of which (Y142X) is likely to cause nonsense-mediated decay. But the phenotypic effects of Y142X haven'bion4maticistnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-91872107996283361662014-03-11T03:05:51.403+00:002014-03-11T03:05:51.403+00:00http://in.reuters.com/article/2014/03/07/regeneron...http://in.reuters.com/article/2014/03/07/regeneron-cholesterol-idINL1N0M41TH20140307Anonymousnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-71824222362216785272014-03-10T07:38:39.094+00:002014-03-10T07:38:39.094+00:00Here is a recent study that argues against PCSK9 d...Here is a recent study that argues against PCSK9 deficiency per se and lower cognitive impairment:<br /><br />J Lipid Res. 2013 Feb;54(2):561-6. <br /><br />PCSK9 SNP rs11591147 is associated with low cholesterol levels but not with cognitive performance or noncardiovascular clinical events in an elderly population.<br />Postmus I1 et al.<br /> <br />Department of Gerontology and Geriatrics, Dirk Hausseckerhttps://www.blogger.com/profile/18320439857875629714noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-85526531966570228822014-03-10T07:34:39.185+00:002014-03-10T07:34:39.185+00:00RE Stan's comments on PCSK9: don't you thi...RE Stan's comments on PCSK9: don't you think it's ironic that it comes from somebody that has essentially killed ApoB as an approvable target?<br /><br />RE Steve and whether too low LDLc could be bad for the brain: it would be great if you could find such evidence from studies on the PCSK9-deficient population. It is important that we don't jump to conclusions about PCSK9 as a Dirk Hausseckerhttps://www.blogger.com/profile/18320439857875629714noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-84875972202860152392014-03-10T00:46:55.403+00:002014-03-10T00:46:55.403+00:00Benitec is a failure. However, some power brokers ...Benitec is a failure. However, some power brokers in Wall St have tipped some money in to it. They must know something. I suspect what they know is the necessary IP for BLT to actually carry some worth has been inlicenced. Or its life expectancy is short.<br />BLT is a faux biotech being managed on PR's and video puff pieces.Anonymousnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-66445474537714543592014-03-09T09:48:45.993+00:002014-03-09T09:48:45.993+00:00You shouldn't ask about Benitec on this blog....You shouldn't ask about Benitec on this blog. It would be nice to get objective insights regarding ddRNAi- anyone?<br />(Read the Voyager essay on this blog)Anonymousnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-12492277065510445112014-03-09T03:52:22.797+00:002014-03-09T03:52:22.797+00:00Dirk what are your thoughts on Benitec and their T...Dirk what are your thoughts on Benitec and their TT-304 Hepatitis C trials going on currently.Looks very promising.Anonymoushttps://www.blogger.com/profile/12555309976991494742noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-64376675059326298312014-03-08T15:23:20.991+00:002014-03-08T15:23:20.991+00:00"There are lots of issues with PCSK9 as a tar..."There are lots of issues with PCSK9 as a target and in fact the FDA has recently written a letter to IND holders of PCSK9 drugs expressing a great deal of concern about PCSK9 as a target, independent of modality, whether its monoclonal antibody or an siRNA or an antisense or a small molecule."<br />Stanley T. Crook 1/11/2012bion4maticistnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-88636667050990623562014-03-08T07:43:40.378+00:002014-03-08T07:43:40.378+00:00We still have berberine. Not sure if it is active ...We still have berberine. Not sure if it is active through PCSK9 but it has been noticed by the Norwegians in this play. I don't know if anyone has put down roots in IP for it here.Richard Lundnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-43843056218689966472014-03-07T23:58:00.022+00:002014-03-07T23:58:00.022+00:00I think PCSK9 is dead for now. Monoclonal antibodi...I think PCSK9 is dead for now. Monoclonal antibodies have killed the market for PCSK9. Even if RNAi doesn't cause this problem, it won't be easy to get FDA approval for this type of drugs. Most likely RNAi companies will be required to prove that they don't have this problem. This could mean further long and expensive clinical study. Not only that, but the RNAi companies developing Anonymousnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-78635095171769464202014-03-07T22:51:48.435+00:002014-03-07T22:51:48.435+00:00Is there a chance that people with naturally occur...Is there a chance that people with naturally occurring low levels of LDL have more neurocognitive issues than people with higher levels of LDL? This could open a lot of questions about what are the best levels of LDL. Maybe what's best for cardio is not what's best for neuro.<br />SteveSteve_382noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-54254431437372199452014-03-07T22:39:49.771+00:002014-03-07T22:39:49.771+00:00...and you didn't mention Alnylam's partne......and you didn't mention Alnylam's partner, even once! LOLexperiencedmentornoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-87292252559880752482014-03-07T21:31:50.836+00:002014-03-07T21:31:50.836+00:00So true Dirk, that is the beauty of the paradigm -...So true Dirk, that is the beauty of the paradigm - already differentiating itself!Fontainebleauhttps://www.blogger.com/profile/03326892408103837271noreply@blogger.com