. . . if that sort of self-deception helps you sleep at night, cr -- sure, maybe 10-20 years . . . heck, make it 30 . . . good night . . .
. . . character assassination, cn, is not the way to move this pig of a drug forward . . . just sayin' . . .
. . . and there's one other tidbit I should have included from the Xconomy story: "One of Solid GT’s scientific advisory board members, University of Missouri professor Dongsheng Duan, recently completed a study published in Human Molecular Genetics showing the delivery of a Duchenne gene therapy in dogs. Duan’s technology was licensed to Solid GT, and that work helped the company get its investment from Biogen and Perceptive, according to Ganot.". . .
. . . (By Pallab Ghosh Science correspondent, BBC News, 5/27/16) . . . "Regulators have given one of the world's largest drug companies approval to sell a new gene therapy. The treatment is for an illness called ADA-SCID which prevents babies from fighting off everyday infections. This is the first approval for a genetic therapy granted to a large multinational drug company, GSK. . . . Commentators say the development marks the beginning of many more genetic medicines from so-called "Big Pharma". The condition is extremely rare and affects around two dozen babies each year. Approval of the gene therapy paves the way for the development of treatments for more widespread illnesses such as thalassemia and sickle cell disease. . . . Hundreds of inherited disorders such as cystic fibrosis, muscular dystrophy and many types of blindness are caused by faulty genes.". . .
. . . you really don't have a clue, g3, but that, of course, is not news . . . Ganot, with BIIB's help won't be ushering in any second- or third-generation DMD drug -- like that oliglop flop, etep . . . they'll likely be providing patients with the first-ever single-treatment cure for DMD -- that is, if they're not beaten to the punch by one of the other genetic engineering outfits chasing that same target . . . you don't seem to comprehend that this is a worldwide competition -- the U.S will be fighting Europe and the rest of the world for dominance in this new technology . . . and you can bet the FDA knows it . . . your line: "He'll have the same problems finding enough kids to power his studies" is absolutely ludicrous . . . parents will be lined up to get their kid into one of these trials -- right behind Ganot's son . . . but there likely won't be many slots available -- maybe six or nine patients total in a phase one, with clear dystrophin results available within six months . . . get a clue . . .
. . . (excerpted and paraphrased from an 11/5/15 Xconomy Boston story by Ben Fidler) . . . “The money, a $42.5 million Series B round from Biogen (NASDAQ: BIIB), New York’s Perceptive Advisors, funds managed by Janus Capital Management, and other unnamed private investors” . . . is going to Solid GT, which is advancing a gene therapy treatment that will employ an adeno-associated virus to circulate the new dystrophin-producing genetic material throughout the body . . . “The University of Pennsylvania’s James Wilson, a gene therapy pioneer . . . chairs Solid GT’s scientific advisory board. Wilson played a big part in advancing the use of so-called adeno-associated virus (AAV) vectors, which have become a gene therapy workhorse.” Also worth noting, a DMD cure is of vital, personal interest to the company’s founder, Ilan Ganot, a former J.P Morgan banker, as his son suffers from the disease . . . though Solid Biosciences is comprised of three subsidiaries -- all focused on DMD -- the new capital infusion will be used exclusively by the sub that’s focused on the gene therapy program “that Ganot aims to get into clinical testing in 2017” . . .
. . . "public record" doesn't cut it, l9 -- cite the quote and the source . . . or is this just a another of your fictional facts? . . .
. . . anything is possible, d7, including a buyout of this pig . . . but at what price? . . . not much, I suspect, as the FDA will likely put etep on a pretty short leash . . . that said, I've been wrong before . . . more interesting to watch, I think, will be etep's pricing if it gets the nod . . . wait for the hue and cry, if srpt tries to gouge . . .
. . . "Then lets add the fact that 37 of the last 42 drugs that the decision was delayed on were approved." . . . and your source for that pearl? . . .
. . . I'm mistaken? . . . really? . . . you sound like every other overly emotional retail investor who is in over his head on a long-shot bet -- a position in which you have limited information, a highly active imagination, and zero control over the outcome -- if you want to live in that world -- swell . . . it's no skin off my nose . . . but as I told mw (in more detail, below), I think etep has a small shot at approval -- that's why I hold shares in srpt -- for the chance at long-shot winnings . . . is it likely to pay off -- no . . . does my ownership satisfy any moral imperative -- absolutely not . . . it’s just a bet -- one that I can afford to make and lose -- to feed my need to gamble on occasion . . . understand? . . . verstehen sie? . . . capiche? . . .
. . . you, apparently, want to live in the world of pumper mania, mw . . . no matter how negative the facts, you folks read the tea leaves and concoct the flimsiest of positive spins and -- worse -- conspiracy theories to rebut reality . . . sure, etep has a small shot at approval -- that's why I hold shares in srpt -- for the chance at long-shot winnings . . . is it likely to pay off -- no . . . does my ownership satisfy any moral imperative -- absolutely not . . . it’s just a bet -- that I can afford to make and lose -- to feed my need to gamble on occasion . . . I remember taking a flier years back on Human Genome Sciences (HGSI) at $1.75 . . . they had a lupus drug finishing phase 3 trials, and analyst predictions, as I recall, were universally grim . . . I enjoyed selling the last of that position near $30 . . . long-shots can pay off, mw . . . just not very often . . .
. . . I don't see big pharma as a buyer, because they're already invested in gene therapy outfits that are likely to bury srpt's technology . . . even BMRN's latest successful effort in hemophilia A is a pure gene therapy play . . . I think the industry thinks -- as I do -- that oliglops are flops . . . we'll see . . .
. . . I don't think etep can win on its merits at this point, mw, so its only chance for a near-term aa, imo, may be political pressure; however, I think it's more likely that the FDA throws srpt a CRL softball, like some guarantee of aa in 12 months -- if the data continues to show a trend of clear patient improvement . . . that said, I, personally, don't think the drug works . . . I don't think the FDA thinks it works . . . but I also think that srpt knows that the window of opportunity is rapidly closing on this technology, as gene therapies -- offering the promise of permanent cures -- continue to make dramatic advances with big pharma support. . .
. . . "There are numerous pitfalls attempting to force Sarepta to treat an entire class of DMD sufferers for free via CU" . . . wrong again, g3 -- as usual . . . the FDA does not FORCE any company to provide a drug for free under CU . . . get the facts straight for change directly from the FDA website . . .
. . . the point, d9, is that the FDA's number one consideration is EFFECTIVENESS -- which according to the agency is something etep clearly lacks . . .
. . . agree, dk, that it's all about the money . . . but, personally, I'm lookin' for a four-fold gain before I dump this pig of a placebo . . .
. . . yeah, little marco is an absolute expert on dmd . . . in fact, I'm betting on misguided political pressure to push this pig across the finish line . . . (holdin' my shares, holdin' my nose, and hopin' for $60 and a quick exit) . . .