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Celgene Corp. (CELG) Message Board

usagary1 388 posts  |  Last Activity: 11 hours ago Member since: Jul 29, 2003
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  • Reply to

    Alert ,,,,found Sarepta on a WHO doc

    by usagary1 Sep 10, 2014 11:34 AM
    usagary1 usagary1 Sep 10, 2014 12:09 PM Flag

    you must remember this...the marburg human trial is on going with the same drug just a different sequence...per say....see the recent safety on that as well

  • Reply to

    Alert ,,,,found Sarepta on a WHO doc

    by usagary1 Sep 10, 2014 11:34 AM
    usagary1 usagary1 Sep 10, 2014 12:04 PM Flag

    This was before the meeting,so i think they will order 1000 doses ''a guess''
    I just want the validation man''
    AVI 7537 (Sarepta)
    In monkey studies, doses of 14 to
    40 mg/kg for 14 days showed
    typical survival ranging from 60% to
    80% when given at the time of
    Human tolerability has been
    demonstrated in early studies.
    The active pharmaceutical
    ingredient is available for 20 to
    25 courses by mid-October.
    Potential production of
    approximately 100 treatment
    courses by early 2015


  • Reply to

    Is today a news day?

    by usagary1 Sep 10, 2014 6:54 AM
    usagary1 usagary1 Sep 10, 2014 8:50 AM Flag

    Thanks for that ..don't care

  • you time is coming..

  • if not it should be, much going on with Sarepta...bullish

    Sentiment: Strong Buy

  • shorts love to talk trash thinking they help drive the price down more..haaa ..what a joke

  • usagary1 usagary1 Sep 9, 2014 2:36 PM Flag

    From what i understand early approval for exon 51 drug is a done deal.The other exons in the new trial will follow with AA with the proof of production.

  • usagary1 usagary1 Sep 9, 2014 2:35 PM Flag

    From what i understand early approval for exon 51 drug is a done deal.The other exons in the new trial will follow with AA with the proof of production.

  • greatness is a coming

  • Huntington's disease (HD) is a neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the huntingtin (HTT) gene. Disease pathogenesis derives, at least in part, from the long polyglutamine tract encoded by mutant HTT. Therefore, considerable effort has been dedicated to the development of therapeutic strategies that significantly reduce the expression of the mutant HTT protein. Antisense oligonucleotides (ASOs) targeted to the CAG repeat region of HTT transcripts have been of particular interest due to their potential capacity to discriminate between normal and mutant HTT transcripts. Here, we focus on phosphorodiamidate morpholino oligomers (PMOs), ASOs that are especially stable, highly soluble and non-toxic. We designed three PMOs to selectively target expanded CAG repeat tracts (CTG22, CTG25 and CTG28), and two PMOs to selectively target sequences flanking the HTT CAG repeat (HTTex1a and HTTex1b). In HD patient–derived fibroblasts with expanded alleles containing 44, 77 or 109 CAG repeats, HTTex1a and HTTex1b were effective in suppressing the expression of mutant and non-mutant transcripts. CTGn PMOs also suppressed HTT expression, with the extent of suppression and the specificity for mutant transcripts dependent on the length of the targeted CAG repeat and on the CTG repeat length and concentration of the PMO. PMO CTG25 reduced HTT-induced cytotoxicity in vitro and suppressed mutant HTT expression in vivo in the N171-82Q transgenic mouse model. Finally, CTG28 reduced mutant HTT expression and improved the phenotype of HdhQ7/Q150 knock-in HD mice. These data demonstrate the potential of PMOs as an approach to suppressing the expression of mutant HTT.

  • usagary1 by usagary1 Sep 8, 2014 3:50 PM Flag

    Background: Peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) are synthetic, DNA analogues that are able to withstand enzymatic degradation and successfully silence genes in a sequence-specific manner by selectively binding mRNA. We screened PPMOs against essential and antibiotic resistance gene targets in the medically important pathogens Escherichia coli (E. coli) and Acinetobacter spp. Methods: PPMO efficacy was evaluated in vitro using minimum inhibitory concentrations (MIC), synergy MIC assays with PPMOs and classic antibiotics (tobramycin, meropenem and colistin) and minimal bactericidal (MBC) viability assays. PPMO toxicity was tested in human A549 and THP-1 cells. Results: In E. coli, PPMOs targeted to acpP and murA were found to be potent in rich media with a larger number of potential leads in minimal media (acpP, rpmB, adkA, murA and infA) with MIC IC75 values ranging from 0.5 to 4µM depending on the gene target. In Acinetobacter, acpP was found to be the most effective target with an IC75 of 2µM in rich media. There was enhanced potency in minimal media, with acpP, ftsZ and accA having an IC75 ranging from 2 to 4µM. PPMOs were bactericidal in MDR strains for both pathogens with 4 log reduction in CFU. The acpP PPMO displayed synergy with multiple classic antibiotics ( 1 log further reduction in CFU than either PPMO or antibiotic alone) in a MDR Acinetobacter strain. Targeting of certain antibiotic resistance genes restored activity to classic drugs. 8 µM PPMO targeting the efflux pump AdeA in Acinetobacter reduced the MIC of tobramycin from 64 µg/ml to 4 µg/ml. Human THP-1 and A549 cells had viability 90% in the presence of 10 µM PPMO. Conclusions: PPMOs targeted to an array of essential genes of Acinetobacter spp. and E. coli, including MDR strains, are bactericidal in clinically relevant concentrations (IC75 of 4µM or less). PPMOs targeting essential genes showed synergy with classic antibiotics. PPMOs targeting nonessentia

  • usagary1 by usagary1 Sep 8, 2014 3:44 PM Flag

    Peptide phosphorodiamidate morpholino oligomers (PPMOs) are DNA analogs that silence expression of specific genes by selectively binding mRNA. PPMOs targeted to antibiotic resistance genes can restore susceptibility to standard antibiotics. We now show that PPMOs targeted to the gene for New Dehli metallo-beta lactamase (NDM-1) reduced the MIC of meropenem and reduced the expression of NDM-1 in various multidrug-resistant, pathogenic bacteria. Methods: PMOs 11 bases in length with sequences complementary to regions near the start codon of NDM-1 were synthesized and conjugated to the peptide (RXR)4, where R is arginine and X is 6-aminohexanoic acid. The resulting PPMOs were tested by measuring the minimal inhibitory concentration (MIC) of meropenem in the presence of various concentrations of PPMO, using NDM-1-expressing strains of Klebsiella pneumoniae, Escherichia coli and Acinetobacter baumannii. Meropenemase activity was measured in periplasm extracts from strains treated with NDM-1 PPMO. Results: PPMOs targeted to various positions of NDM-1 mRNA reduced the MIC of meropenem 8 to 32-fold, depending on the bacterium. At a concentration of 8 μM, the most effective PPMO reduced the MIC of meropenem from 64 μM to 4 uM in K. pneumoniae 2146, from 32 μM to 4 μM in E. coli CVB-1, and from 256 μM to 8 μM in A. baumannii BCT-B-026. Moreover, at the MIC concentration of meropenem in the presence of 8 μM NDM-1 PPMO, the viability of each bacterium decreased by over 3 orders of magnitude. Meropenemase enzymatic activity in the periplasm of PPMO-treated cells was inversely proportional to the amount of PPMO added. Conclusions: PPMOs silenced expression of NDM-1 and reduced the MIC of meropenem to susceptible concentrations in 3 multidrug-resistant pathogens.

  • Discovery and Development of Filovirus Therapeutics
    Solicitation Number: HDTRA1-14-R-0031
    Agency: Other Defense Agencies
    Office: Defense Threat Reduction Agency
    Location: Defense Threat Reduction Agency (Headquarters)

  • usagary1 usagary1 Sep 8, 2014 1:04 PM Flag

    You should watch what you say do not know how hard it was to change a research company into a drug making biotech..

  • usagary1 usagary1 Sep 8, 2014 11:41 AM Flag

    but it is coming

  • usagary1 usagary1 Sep 8, 2014 11:25 AM Flag

    The antiviral platform of Sarepta's could cure many of the single strand viruses ''same drug just a different sequence''
    worth billions.....

  • until patents get older or sarepta getting to much of the market share,

  • usagary1 by usagary1 Sep 8, 2014 8:45 AM Flag


  • usagary1 by usagary1 Sep 8, 2014 8:09 AM Flag

    SKIP-NMD Webinar for DMD families on 8th September
    5 September 2014

    A webinar has been organised by the patient and family representatives in SKIP-NMD to inform the patient community about exon-skipping trials in general and SKIP-NMD in particular. This will take place in the UK on 8th September at 17.00 BST and in Italy at 17.30 CEST.

    Instruction for registering in the UK are below:

    Webinar link:
    Webinar ID: 136-029-587
    Toll: +44 (0) 20 7151 1875
    Access Code: 841-837-690
    Audio PIN: Shown after joining the webinar
    For Italy, please contact Mariateresa Moscato

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