Topic Archives: Hemophilia

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    that is a great question, and I suspect fixing bad genes in cancers will be difficult; maybe fixing bad genes in heredit...

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    Can't say which tech is better, but SGMO appears to be much farther ahead in research. "In 2017, Sangamo is conducting ...

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    $SNY $BIIV = crooks to pay fines... why don't they ever go to jail? Regulators at the SEC are sounding an alarm fo...

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    $BioMarin Interesting UK study Hemophilia A success. Small study but quite the patient response....

  • SoGiAm

    #ASH17 NEWS http://e.endpointsnews.com/t/ViewEmail/t/16F7237A4D7B4F15/2F80028A944F69C925D77A725F39070E The BCMA-targ...

  • Dr. KSS MD PhD

    Imagine having hemophilia or von Willebrand disease and not bleeding all over creation when you scrape yourself. I HAVE ...

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    $Rubius Therapeutics Just secured $120M in funding from Flagship. Torben Straight Nissen, president at Rubies and partn...

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    $SGMO np: Receives Fast Track Designation From The FDA For SB-525 Investigational Hemophilia A Gene Therapy https://fi...

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    $SGMO Amazing. Always been great at raising large amounts of capital and having numerous irons in the fire. I ha...

  • Dr. KSS MD PhD

    At-heart coagulationist that I am, I'm pretty amped about Genentech/Roche's phase 3 success today with emicizumab. This ...

  • SoGiAm

    $QURE rpts Q2: Add. data on AMT-060 in Hemophilia B Later this YrData fr Sanfilippo B & Parkinson's Dis. in Q1'17 h...

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    to be more precise, in paragraph 1 "I don't see a gene therapy for Hemophilia B".......

  • Dr. KSS MD PhD

    Hi eyedoc! Thanks for doing some ferreting. In actual fact, Baxalta is recruiting now for its in-house factor IX gene th...

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    Doc, I don't see a gene therapy for either Baxalta (which has a recombinant Factor IX replacement therapy and BAX 888 in...

  • achandra13

    I think the answer is (b) to the best of my knowledge as Biomarin developing a gene therapy for Hemophilia A....

  • Dr. KSS MD PhD

    Question of the Day: Which ONE of the following companies is NOT developing a gene therapy for factor IX deficiency (...

  • SoGiAm

    $ALNY, $BMRN, $PFE, $QURE, $SHPG Gene Therapy and Other New Hemophilia Products https://twitter.com/Liquid_Biopsy_/st...

  • SoGiAm

    $ONCE $PFE Spark Therapeutics and Pfizer Receipt of FDA Breakthrough Therapy Designation for SPK-9001 for the Treatment...

  • SoGiAm

    $QURE Announces Presentation of Updated Data for AMT-060 at the World Federation of Hemophilia World Congress 7/27 http...

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    Spark Therapeutics (ONCE) doing some good things with gene therapy treating hemophilia. https://www.technologyreview....

  • Dr. KSS MD PhD

    Lovely pop today in $ONCE, the firm treating hemophilia B with factor IX gene therapy. ONCE is attaining 30 percent norm...

  • Dr. KSS MD PhD

    Ben: You were asking about Versartis $VSAR. In no particular order: (1) the company share price is in a perpetual dow...

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    A few months old article, but a good article on the gene therapy race and competition to possibly cure Hemophilia. ht...

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    A promising looking gene therapy company Dimension Therapeutics (DMTX) looks interesting trading a bit over 6.00. It is ...

  • sogiam

    TLOG- TetraLogic Pharmaceuticals Announces Data on Lead Clinical Programs: http://globenewswire.com/news-release/2016/...

  • sogiam

    TGTX-TGTX Phase I/Ib Study Evaluating the Efficacy/Safety of TGR-1202 Est Study Completion Dec 2015 BXLT-PDUFA Adnovate...

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    Baxalta (BXLT) and Obizur get Health Canada approval for acquired Hemophilia A. http://finance.yahoo.com/news/baxalta...

  • Dr. KSS MD PhD

    Thanks, bird-dog Ben. The insider selling troubles me in that the stock is well off its highs. I have a long position in...

  • Dr. KSS, MD PhD

    Don, yes, AC5 would definitely have a role in helping the hemophiliac pt. They come in several flavors: A, which confuse...

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    I have no idea of the current prevalence of Hemophilia or how crises are treated, but wonder if AC5 could be a home use ...

  • Dr. KSS, MD PhD

    I have begun drafting a gene therapy primer for you. I think the ophthalmology community may be waking up to Molecular P...

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    Well here's another M&A rumor from SA regarding PFE... -According to Reuters, Pfizer (PFE +0.2%) is negotiating a tak...

  • Dr. KSS, MD PhD

    Hi don: we have pretty vigorously discussed Spark $ONCE in prior threads. You might do Control F searching to find those...

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    Maybe yes, maybe no. This update occurred after Doc's opinion was expressed. Watertown, Mass. – June 17, 2014 – S...

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    In a report released yesterday, Cory Kasimov from J.P. Morgan reiterated an Overweight rating on Spark Therapeutics (NAS...

  • Dr. KSS, MD PhD

    Larry, I don't personally think it competes with ARTH because this product's huge downside is that it makes recipients t...

  • Dr. KSS, MD PhD

    Someone commented recently on $OPKO's recombinant factor VIIa-CTP product, soon to enter phase 2a. I wrote something abo...

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    Thank you Dr. KSS. I have re-reviewed your comments on prior thread at post # 74 and 251; and have also noted your comm...

  • Dr. KSS, MD PhD

    A good synopsis today from John Carroll, the editor at Fierce. $ONCE has competition in hemophilia B, BUT has a heads...

  • Dr. KSS, MD PhD

    Rae: it's a good company in a quite unusual position: it hits the street having completed proof of concept in a set of v...

  • Dr. KSS, MD PhD

    I am keen on Spark. I hope they will push ahead with IPO'ing. I know Kathy High, MD, the CEO. I would describe her, and ...

  • Dr. KSS, MD PhD

    To ScorpioRising and Kris Prasad: sorry for being a latecomer to the discussion about AAV-8. I appreciate the references...

  • Dr. KSS, MD PhD

    Dan: the dose in question here is 4 x 10 to the tenth virus genomes per kg. And that is given by peripheral iv. By co...

  • om sharma

    Dr. KSS, Any thoughts about this - Uniqure - (QURE ) http://www.uniqure.com/ What is the future of the company ? A...

  • Dr. KSS, MD PhD

    Gary: Thanks for posting and I appreciate your comments. Alnylam certainly has a future, though by now it is big. I don'...

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    Dr. KSS, re: # 419..... you said "A number can be x times greater than predicted. It cannot be x times less than predict...

  • karmaswimswami

    Siva and biocqr: As to the objections raised regarding TT-034, AAV8 and liver transfection/transduction efficacy, I have...

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    Thanks Siva, Regardless of how the Nature AAV8 trial was run, the fact remains that Dr. Kay was surprised that in the...

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    Dr. K, I’m still somewhat confused about the viability of using AAV8 for the successful transduction of TT-034. Accor...

  • karmaswimswami

    I meant also to point out that while this is a global/multinational $109 billion company that controls the markets for i...

  • karmaswimswami

    This 2011 NEJM paper should truly lay to rest any concerns that AAV8, as Benitec is using in the TT-034, can succesfully...

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    Below is a piece from http://rnaitherapeutics.blogspot.com. Note that the author is pro Tekmira. Dr KSS - I would like t...

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