Trump talked big since he was a kid. He talked of being the king of NYC real estate......And then he did it....and beyond. Some guys that talk a good game also play a good game. It happens.
CRISPER seems to be TOO good. I.e. off target phenomena. Isn't that the latest data on it? Also it's permanent. If it can overcome the off target problem it may someday supplant us in inherited genetic rare diseases. But by then we'll probably be moving our stuff into "regular" diseases or "conditions". For example, skin problems, rheumatoid arthritis, that soluble TNFa thing from a few years back, cancer - all that fun stuff. Not permanently changing someone's DNA like CRISPER. Have a little imagination.
If worse comes to worst, I could see - or would like to see - a big name philanthropist taking his eyes off poor old Africa and turning it towards helping kids who need it even if their only lowly Americans. Like Gates for instance. Or Bloomberg. Is that too much to ask for? Is DMD sexy enough?
Yeah. I suppose the next best thing would be a Sanofi/Genzyme type arrangement. So we're still - sort of - independent.
That's how I see it too, Spec. And by the time we reach that $140 level the talk will be of Europe and rest of world, exons 44 and 53 etc etc .....so the froth above $140 won't REALLY be froth....!
Insurance. Call Sarepta - or go to that "skip-ahead" site they have. They supposedly are working with parents to work out that side of the equation.
Ten more years as an independent company becoming a great company has always been our dream, Rats.
Whether the present board and company leadership shares it, or even if it's possible these days, is the only remaining big question in my mind.
I now know our science is great - it works - and will be a benefit to DMD patients and probably a slew of other rare diseases victims. That question is answered.
The next question we have is going to be: Can Sarepta become a giant like we also pictured? .... That's the next big question we can worry ourselves sick over ... NEXT year!
Great research. "Let's start at the very beginning. It's a very good place to start."
But, Biomarin paid some $500 million for Prosensa knowing the FDA would approve (not accelerated approval, straight out approval) drisaperson ... because they approve everything these days. So what if they never sell one vial of the drug! They still get their voucher which is worth probably $400 million to them. The other $100 million is what Prosensa's science was worth. That's why Biomarin is going through with this whole thing. Now they've stepped into the exon-skipping arena and everybody knows it. After the voucher they'll get it didn't really cost them that much and look at all the publicity!. Maybe they'll get the Prosensa scientists to do something with a PMO variation next time around and maybe next time they'll come up with something they can get people to actually use.
Thanks for continuing to quote the FDA's advice to Biomarin and Biomarin's failure to take any of it. It will be very educational to now see how the FDA responds to such inaction. Giving them an okay after this would kind of screw their credibility and make one wonder how Biuomarin could have such "pull". I don't really care what they decide but if they do approve the drug after Biomarin's "non-performance" I'm sure a lot of heads will be shaking, wondering, and being scratched.....Strange goings on.
"VII. Advantages of the Method
 The present invention demonstrates that inhibition or alteration of protein expression can be achieved by antisense targeting of a region downstream of a splice acceptor, in the coding region of a pre-mRNA, using a non-RNAse competent oligomer. The present compositions and methods have several advantages over prior art methods of antisense inhibition, in which the targeting antisense compound spans and hybridizes to a splice acceptor sequence (e.g. Giles, cited above; Kole and Dominski, U.S. Pat. No. 5,665,593).
 One such advantage is that exon sequences tend to much more highly conserved among species than intron sequences. This allows for greater predictability in testing such methods on animal models.
 In addition, greater flexibility is allowed in selecting a sequence for targeting, as the oligomer is not required to actually bind to a precise site such as a splice acceptor junction or an AUG start codon. The present invention thus increases the range of sequences which may be successfully targeted in an antisense application. Such flexibility can be advantageous in avoiding undesirable side reactions, such as caused by inadvertent targeting of non-target proteins in a subject, or targeting of host proteins when attacking an infectious agent such as a virus or bacterium. This is demonstrated in Example F, above.
 In the present strategy, in contrast to methods which target the splice junction directly, various regions of the exon downstream of the SA site may be blocked. This opens the possibility of producing different variant proteins, by directing splicing to different cryptic splice sites downstream of the normal SA. As described above, certain variant proteins, such as dominant negative proteins, can have unique advantages. Accordingly, the antisense could be designed to promote splicing at a particular cryptic splice site, which would give rise to the desired variant protein, over others. For example, a less desirable cryptic splice site close to the normal SA site could be hindered in favor of a site further downstream.
 Further benefits of flexibility of design may include convenience of synthesis or enhanced binding affinity. In addition, by using the present strategy of targeting fully within the exon, the target RNA can be identified by the antisense oligomer in either the nucleus or the cytoplasm, which can be advantageous for purposes of analysis.
Barry now being on our board is good. Naturally, he'd be hip to this strangulation strategy of Big Pharma if it's for real. So we shouldn't get taken. If it's a given we'll get approved (the general consensus) I imagine there is movement afoot now amongst the players involved Time for everyone to start showing their cards ... In strictest privacy, of course!.......
The short positions are financed by Big Pharma. Look, if they can buy us out for only 6 billion as opposed to 10 billion they can afford to lose a lousy1 billion financing the Sarepta strangulation effort they have had a part of their hedge fund "investments" focused on. They are loaded with cash, they invest that cash, and one of those investments is the Sarepta short.....I'm sure they're doing it. Why wouldn't they?. They are sitting on mountains of cash and they know we've got the goods. It's been going on ever since they saw our stuff was working 3 years ago. I suppose it's legal.......Probably the smart money is hip to this operation and have been taking their sweet time accumulating shares for themselves at opportune moments. Sarepta just forges ahead against this onslaught - raising cash at two surprise moments but otherwise just resigned to relative poverty until drug is sold and cash comes in. The voucher on approval will help.....At some point smart money will jump in as we approach the end game --- shortly. And that will slowly be raising our stock price...One thing's for sure - this is a great opportunity for individual investors to hop on board .....for as long as this Big Pharma financed short death grip operation proceeds..... I'm sure this three year head-lock is financed by parties interested in our science who want to buy us out as cheaply as possible -- if not smother this new science that scares the bijeezus out of them .... especially if they can't get their own hands on it !!!
I would imagine a lot of our new customers will experiment with no steroids on their own. Especially the younger ones, I'd think. Maybe it won't need a formal study...?
Has there ever been a case of shorts being in cahoots with Big Pharma? I.e. keeping the price of a juicy potential takeover target down and off the radar? Especially a scary company like Sarepta - a company with breakthrough technology that will lead the next revolution in medicine? If I were a big pharma without access to our technology I would be VERY nervous about the future. I can easily see them all teaming up (formally or informally) to keep our profile down - way down. Science like we've got is disruptive and I'm quite sure they wish we just weren't here. And they're eyeing us nervously ... and watching their competitors eyeing us nervously .... and they probably don't like it a bit.
That would be funny if Johnson&Johnson was interested in us. I think my second posting here - 10 years ago! - suggested just that scenario might be afoot!! Things really do take time, I guess!
(...."J&J was reported recently(six months ago) to be interested in RNA-targeted therapeutics for the treatment of rare, infectious and other life threatening diseases."----)
Happened at 5pm yesterday. It's probably a key milestone that might just now (for some reason) be hard to identify as the turning point it really was. ....But, at long last ..... we finally won one !!
As intellectually certain as I was that we'd achieve this milestone, I have to admit I had butterflies - thanks to the last two or three key "turning points" that suddenly opened up an abyss under this company, rather than the expected happy path forward! We seemed to be jinxed. Now at last it's looking like that jinx is finally gone. The shorts will try to maintain the jinxed aura for a while, of course....but I think the jinx is truly gone.
But the stink was so bad, it'll probably take the adcom and a fast approval to banish it for good - in the mind of the everyday investor. That's probably why it's hard to appreciate fully just now even for us what's actually just begun : - a sea change in Sarepta's psychology...... But it really DID just happen.
Here's to many more such happy milestones!
Well, I figure if the FDA had one day to respond it would have been June 27th....since they have 59 days more than that it would be August 26th.
But they can either phone Ed Kaye tomorrow or send him a letter. If they go the letter route it will be an additional 2 weeks. They'll probably phone. And then Kaye has 4 days to tell the world.
So ....... it's silly to do anything but throw up your hands and think about something else!
Today marks 60 days since June 26th.
"...today announced the completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for eteplirsen on June 26, 2015"