Simos Simeonis of RBS quoted by Barron's approvingly, I suppose...
"However, we also believe that he deserves credit for having raised awareness for the company, the drug and the disease, by being a passionate and charismatic advocate for all three. And even though he is leaving Sarepta unceremoniously today, his work in the past four and a half years helped bring then-AVI Biopharma out of obscurity and very close to an NDA, with a large contingent of the Duchenne muscular dystrophy patient community behind the company."
Chris will be missed..
Hillary's dopey reset button actually read "overpriced"...Ours reads "underpriced".
For "Rico" as for Piney, it's always all about "her money". And resentment about Chris's money, of course...What tripe indeed.
Ed Kaye should be perfect right now .... and for the next 2 or 3 years. To bring this drug and it's cousins thru the FDA...as well as to get all the new trials we'll be needing going.....As well as bringing on partners for our potentially vast pipeline. His old company Genzyme is right down the block. Maybe he'll start with them..
A bogus takedown. Pre-planned for this positive conference. Pretty obvious to me. That big buy in the morning must have spooked them too. The usual games. Shorts want to exit on their schedule - whatever it may be.
They conclude suggesting this heart focused PipPMO chemistry could be used for other heart conditions:
"In addition, this work demonstrates ‘proof of principle' for the utilisation of Pip6-conjugated AOs for the correction of cardiac structural protein defects. Three genes encoding cardiac sarcomeric proteins, MYH7, MYBPC3 and TNNT2 have been implicated in over 70% of all familial hypertrophic cardiomyopathies52 and indeed exon skipping technology has already been demonstrated as a possible therapy for Mbpc3 mutations53. Thus Pip-PMO compounds could have wider application in the treatment of many familial cardiac defects."
From the current article. (compliments of our British collaborators of happy memory Woods, & Gait) --....
"A powerful approach to improve AO potency is the conjugation of cell penetrating peptides to uncharged AOs, such as PMO24, 25, 26, 27. While early generation peptide-PMOs were weakly active in heart, they were shown to have an influence on heart function in mdx mice displaying a mild cardiac phenotype27, 28. Recently we have developed a novel series of highly active peptides known as Pip's (PMO internalising peptides) for PMO delivery29. This series of peptides comprise a hydrophobic central core with 2 flanking arginine-rich domains. This combines the improved skeletal muscle delivery capacity observed with the arginine rich B-peptide, with a hydrophobic sequence that dramatically enhances efficacy to create a new innovative series of peptides30. Previous studies showed that the hydrophobic region is critical for the delivery to the heart tissue as other arginine-rich peptides with a shorter hydrophobic region failed to achieve this. Progressive evolution of this peptide series through structure-activity studies has identified novel peptides with dramatically enhanced cardiac dystrophin restoration30, 31, 32. Pip6-PMO is currently the most potent class of peptide-PMO, demonstrating high levels of dystrophin protein restoration following a single low intravenous dose, most notably in cardiac muscle."
Click on this headline and check out tis old PipPMO thread for background.
Other than that, the title Gary-Piney is keeping from you is "Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice'
Manipulators have a field day with any Punit talk. it's a golden opp. and they know it. They must love the guy. His speaking ability is a sure loser and of course they manipulate accordingly. who can blame them? Still a great buy, of course. That's the whole point.
And I gave you a thumbs UP for your info about the Cambridge branch. I'm sure they want to sell that off asap. It's a loser.
See Jan.15th JPMorgan (2nd pdf) - pages 34-38. Reseach Pipeline. No mention of an anti-cholesterol in those 5 pages of research pipeline....and I'm happy to see it's not there. We've got much more interesting stuff planned than that.
Funny how Winter, Simp, (and Piney on I.V.) are all foaming at their inarticulate mouths about this obscurity. What is ruffling their feathers this time? It's kind of hard to see the exact tiny spark that has set "them" off today... but generally it's just what it usually is: someone is making money and it's not ... her. Boo hoo hoo. I must say whenever it happens to her, I do enjoy it. That God for these small pleasures that investing in Sarepta allows me. It's all I've got!!
Each drug will be tested in up to 100 patients. It ends December 2016.
"The trial will enroll adults and children admitted to Ebola treatment units in Liberia, health care workers infected with Ebola virus in West Africa who have returned to the United States for treatment, and adults and children who may have acquired Ebola in the United States through secondary transmission"
He wants wants nailed down. Nailed down. Nailed down. Nailed down. Screwed down. Glued down. Sat upon by a sumo wrestler with arms crossed.....Then we submit.
Nicely put, md:
"It's just a shame that DMD boys have been denied a drug that is safe, and given early enough, can likely prevent muscle fibrosis, and all the eventual physical / medical complications that result from that."