. . . since you're obviously not the sharpest knife in the drawer, g3, I'll say it once more --slowly: "watch . . . for . . . the . . . first . . . dmd . . . patient . . . to . . . be . . . dosed . . . with . . . a . . . potential . . . genetic . . . cure . . . in . . . 2017" . . . if you still don't understand, maybe you can find someone else to read it and explain it to you . . .
FDA website: "Wherever possible, use of an investigational medical product by a patient as part of a clinical trial is preferable because clinical trials can generate data that may lead to the approval of products and, consequently, to wider availability. However, when patient enrollment in a clinical trial is not possible (e.g., a patient is not eligible for any ongoing clinical trials, or there are no ongoing clinical trials), patients may be able to receive the product, when appropriate, through expanded access." . . . individual patients need only apply; however, srpt is not required to play ball, and I think they've already said as much . . . and, of course, insurers are likely not obliged to cover any associated costs . . .
. . . "they logically have to incorporate into their thinking what a CRL would mean for future investment in DMD" . . . more likely, mh, the FDA is fully aware of new genetic technologies that are rapidly coming on stream that very likely will obsolete sprt's antisense nonsense approach . . . why do you suppose big pharma partnering in this technology has all but dried up? . . . watch for the first dmd patient to be dosed with a potential genetic cure in 2017. . . just sayin' . . . (still holdin' srpt, holdin' my nose, and hopin' for $60 and a quick exit) . . .
. . . au contraire, f0, I lost money -- though mostly on srpt . . . but I prefer -- as a longtime biotech investor -- to take the long view . . . yes, I sold some BMRN at $140 to buy more srpt as a hedge and both got killed by the FDA . . . that said, my considerable stake in BMRN was originally purchased at $17 per share, and I had been selling it off since it broke $100 . . . and, as I posted here some months back, I sold the last of it to buy more BLUE, which is where I think the future of medicine is headed . . . just confessin' . . .
. . . now, now, ci, this drug, imo, actually has some remote chance of winning approval, or maybe just a softball CRL, if enough anti-science, right-wing dopes in congress -- who hate "gummit" -- put enough pressure on the FDA . . . truth be told, I have material positions in both PFE and Spark (ONCE) and a number of other genetic therapy outfits, as well -- but I like taking a flier now and then on a real long shot, and this pig appears to have the potential upside to make the bet worthwhile. . . that said, I don't recommend you follow suit, unless you can also afford to lose big . . . just sayin' . . .
. . . I wonder why PFE or some other big pharma never partnered with srpt? . . . you think maybe because they prefer drugs and technologies that actually work -- I mean beyond a placebo effect, of course? . . . just speculatin' . . . (still holdin' my srpt shares, holdin’ my nose, hopin’ for $60, and lookin’ to make a quick exit) . . .
. . . Reuters, May 19, 2016 . . . “Spark therapeutics and Pfizer announce data from initial subjects in hemophilia b trial demonstrating consistent therapeutic levels of factor ix expression” . . . “Subjects received one-time administration of a highly optimized gene therapy at initial low dose without need for immunosuppression” . . . “Over combined 28 weeks of observation, none of three subjects received regular infusions of factor ix concentrates to prevent bleeding events” . . . clock is tickin' on DMD . . . (still holdin' my srpt shares, holdin’ my nose, hopin’ for $60, and lookin’ to make a quick exit) . . .
. . . great firsthand reporting, cm . . . I'll have to look into that BIIB connection and Solid Biosciences . . . I wonder if Duan -- now three years later -- would have the same assessment on etep? . . . regarding the cost of gene therapies, that's obviously still evolving, but my expectation remains some type of pay-for-performance scheme, i.e., some agreed-upon max paid out over so many years, so long as the cure holds -- in any case, very likely cheaper than etep over the long term . . . and the translation of gene therapies from animals to humans -- from everything I've read -- seems to be going exceedingly well . . . overall, I'm very optimistic at this point -- though, as a longtime biotech investor, I know stuff can go terribly wrong in a hurry . . . that said, I'm reminded of a comment by BLUE's CEO Nick Leschly after a recent quarterly Q&A (I'm paraphrasing here): you can bet against us, but I don't advise it . . . thanks, again, cm, for that insider's perspective . . .
. . . morpholinos, I suppose, "can target almost anything" . . . but it's results that count, cs, and that's the difference between gene editing and the the morpholino antisense nonsense: gene editing is producing stunning results -- right now -- and oliglops continue to produce flops . . . look no further than where big pharma is partnering today . . . it's in gene editing, because they know -- PFE, BMY, GSK and all the rest -- that it's the future and that there's a global race on to be first to market . . .
. . . “Today, several hundred gene therapies are in development, and many aspire to be out-and-out cures for one of about 5,000 rare diseases caused by errors in a single gene.” . . . I wonder how many big pharmas are still pursuing the oliglop-flop approach? . . . hmm . . . (holdin' my shares, holdin' my nose, and hopin' for $60) . . .
. . . agree completely, cs . . . the cocktail should easily double the placebo effect -- synergistically speaking, of course . . . you folks are a hoot . . . (holdin' my shares, holdin' my nose, and hopin' for $60) . . .
. . . you misunderstand as usual, cs . . . I'm simply hoping for an absurd overreaction in the market on any good news from the FDA, so I can cut this pig loose and move on . . . that said, I do think news of a legitimate DMD cure will be announced in 18 to 24 months . . . that, I think would news worth hearing -- about a cure . . . not just an over-hyped placebo effect . . .
. . . in fact, check out the latest DMD breakthrough developments at Duke University and the University of Missouri . . . amazing stuff . . . (still holdin' my srpt shares, holdin' my nose, and hopin' for $60 -- and a quick exit) . . .
. . . you need to get current, cn, and quit makin' stuff up . . . just sayin' . . . (holdin' my shares, holdin' my nose, and hopin' for $60) . . .
. . . you clearly have not been keeping up, s2 . . . watch for more big new later this year . . .
. . . by Antonio Regalado (excerpted from MIT Technology Review, 5/6/16) . . . “A treatment now pending approval in Europe will be the first commercial gene therapy to provide an outright cure for a deadly disease. . . . Called Strimvelis, and owned by drug giant GlaxoSmithKline, the treatment is for severe combined immune deficiency, a rare disease that leaves newborns with almost no defense against viruses, bacteria, or fungi and is sometimes called “bubble boy” disease" . . . "The treatment is different than any that’s come before because it appears to be an outright cure carried out through a genetic repair. The therapy was tested on 18 children, the first of them 15 years ago. All are still alive.” . . . “The British drug giant licensed the treatment in 2010 from the San Raffaele Telethon Institute for Gene Therapy, in Milan, Italy, where it was developed and first tested on children." . . . On April 1, European advisers recommended that Strimvelis be allowed on the market and if, as expected, GSK wins formal authorization it can start selling the drug in 27 European countries. GSK plans to seek U.S. marketing approval next year. . . . GSK is the first large drug company to seek to market a gene therapy to treat any genetic disease. If successful the therapeutic could signal a disruptive new phase in medicine in which one-time gene fixes replace trips to the pharmacy or lifelong dependence on medication.” . . . “Today, several hundred gene therapies are in development, and many aspire to be out-and-out cures for one of about 5,000 rare diseases caused by errors in a single gene.” . . . and DMD is already in the cross hairs . . . just sayin’ . . .
. . . show some class, cn, and give the mindless character assassination a rest . . . thanks . . .
. . . I meant exactly what I wrote, mr . . . etep likely garnered a number of sympathy votes that were undeserved in light of its stinkin' performance . . . just sayin' . . . (still holdin' my shares, holdin' my nose, and hopin' for $60) . . . Less