tag:blogger.com,1999:blog-7857054149675424609.post9030345535890979336..comments2024-02-24T08:58:08.581+00:00Comments on RNAi Therapeutics: Oligonucleotide Therapeutics Companies Crowding into GalNAc-ConjugationDirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.comBlogger15125tag:blogger.com,1999:blog-7857054149675424609.post-46628833457108473972014-05-07T12:33:22.333+00:002014-05-07T12:33:22.333+00:00Alnylam research and RNAi lead development vice pr...<br />Alnylam research and RNAi lead development vice president Dr Rachel Meyers noted that the company's pre-clinical research efforts have now led to the selection of a DC in its ALN-AAT program, which is part of its genetic medicine pipeline. <br /><br />The <a title="company" href="http://itsoftware.pharmaceutical-business-review.com/news/Anonymoushttps://www.blogger.com/profile/05606128604396120464noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-19459760181011971672014-03-03T03:58:28.455+00:002014-03-03T03:58:28.455+00:00Will all these developments of meaningful science ...Will all these developments of meaningful science and statistics ever translate into meaningful commerce?<br />It's like we're making great progress but the underlying assumption is it all means nothing until a disease is curable or preventable. Only then will someone pay for it.<br />Why haven't we got a model for business where an improved state of well-being or a prevention of Anonymousnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-82368710832732966142014-03-02T15:55:57.652+00:002014-03-02T15:55:57.652+00:00Whether ISIS, Alnylam, Arrowhead, or another compa...Whether ISIS, Alnylam, Arrowhead, or another company...yes, GalNAc may help in achieving oral delivery. Dirk Hausseckerhttps://www.blogger.com/profile/18320439857875629714noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-35307697539535704182014-02-28T16:29:33.613+00:002014-02-28T16:29:33.613+00:00how close is Isis getting to oral delivery? Could...how close is Isis getting to oral delivery? Could galnac aid its efforts to achieve oral delivery?Anonymousnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-64180508013213296942014-02-27T18:40:01.289+00:002014-02-27T18:40:01.289+00:00'if you achieve knockdown of 95% at 0.1mg/kg o...'if you achieve knockdown of 95% at 0.1mg/kg or at 0.5mg/kg?'<br /><br />Agreed. But what about if you can achieve 99% and more extended knockdown with the more potent tech at 0.5mg/kg?<br /><br />In Asia (culture) and when it comes to docs (adherence), i.v. seems to enjoy more acceptance than subQ.<br /><br />And when both methods can achieve similar knockdown, it may actually come downDirk Hausseckerhttps://www.blogger.com/profile/18320439857875629714noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-23540392563361613432014-02-27T15:15:33.889+00:002014-02-27T15:15:33.889+00:00"Well. I like this one today. But I liked a d..."Well. I like this one today. But I liked a different one yesterday. So stay tuned, I may like the old one again tomorrow, like I once favored this one, or I may not like any of the old ones. And pick an altogether new one.<br />Are you all still following me?"tufuliponoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-12517481890548259592014-02-27T14:59:23.914+00:002014-02-27T14:59:23.914+00:00Dirk,
Does it really make any difference if you a...Dirk,<br /><br />Does it really make any difference if you achieve knockdown of 95% at 0.1mg/kg or at 0.5mg/kg? Both of them are very safe and well under 1ml volume of injection. Seems to me Subq will be the preferred mode of delivery going forward.Anonymousnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-52672142341304272332014-02-27T14:16:44.948+00:002014-02-27T14:16:44.948+00:00'Santaris and Isis will be the winner since it...'Santaris and Isis will be the winner since it will eliminate any perceived advantage that RNAi companies had in liver delivery'<br /><br />As I said, knockdown potency will matter for most diseases. The difference of a 85% vs 95% knockdown e.g. is 3x the amount of insulting protein left.<br /><br />Yes, it should improve the toxicity profile of ASOs (Santaris and ISIS), especially Dirk Hausseckerhttps://www.blogger.com/profile/18320439857875629714noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-50239746526529155282014-02-27T13:30:27.062+00:002014-02-27T13:30:27.062+00:00With improving potency by competing delivery techn...With improving potency by competing delivery technologies, I think any advantage that any company had over other company in its delivery is getting marginal and not a significant factor any more. This is good news for patients but it will create fierce competition between all the RNAi drug developing companies. Santaris and Isis will be the winner since it will eliminate any perceived advantage Anonymousnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-56775103884712640152014-02-27T13:16:30.087+00:002014-02-27T13:16:30.087+00:00Dirk, for a Marina or Benitec, how much in $US bal...Dirk, for a Marina or Benitec, how much in $US ball park figures is a decent lab to 1) set up, 2) run p/a (with quality staff who will do the job), in say a typical biotech hub?<br /><br />And if that string is too variable, then seems to me there is a need looking for a solution, and you are quite probably better placed than most to facilitate the answer. <br /><br />As you hinted, Pfizer (and Anonymousnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-74013773489164681222014-02-27T12:52:25.967+00:002014-02-27T12:52:25.967+00:00$mrna can really enhance value from this point on....$mrna can really enhance value from this point on. Very cheap IMHO.Anonymousnoreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-27043676716364800792014-02-27T12:49:28.779+00:002014-02-27T12:49:28.779+00:00Let's not forget Arcturus and Mirna, the latte...Let's not forget Arcturus and Mirna, the latter meaning $7M per selected target that enters development. Their IP is already far more advanced than the rest ,and sorry, SNALP included. Just take a look at Alison Silva's last slideshows that can or could be found somewhere on their site.. Now the only thing they need is a few phase I results. Disclaimer, yes, I've been invested in thisFMShttps://www.blogger.com/profile/02113286551862140535noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-27577054738546168232014-02-27T12:43:21.076+00:002014-02-27T12:43:21.076+00:00It's a shop for over the counter tailored knoc...It's a shop for over the counter tailored knockdown, and remember, it's transkingdom (Monsanto deal) so they can keep advancing the science indefinitely as they partner out. CEQ508 was always Michael's baby and the top priority, even though new clinical programs will start. FMShttps://www.blogger.com/profile/02113286551862140535noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-42527382259391667062014-02-27T12:15:42.171+00:002014-02-27T12:15:42.171+00:00'until we establish a sponsored research and d...'until we establish a sponsored research and development collaboration with a partner.'<br /><br />Dirk Hausseckerhttps://www.blogger.com/profile/18320439857875629714noreply@blogger.comtag:blogger.com,1999:blog-7857054149675424609.post-10253288213754094742014-02-27T12:10:36.170+00:002014-02-27T12:10:36.170+00:00Dirk, from Marina's latest PR I guess French a...Dirk, from Marina's latest PR I guess French answered your question.<br /><br />"Since we are transitioning from a primarily research focused company to a primarily development focused company, we intend to create a small initial footprint in the Boston area and keep our operating costs at a minimum. We will utilize contract research organizations as well as academic centers to advance FMShttps://www.blogger.com/profile/02113286551862140535noreply@blogger.com