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Posts tagged with "Clinical Trials"

  • Clinical Trials

    Sangamo’s Gene Therapy Results

    Let the arguing commence! That’s my response to the announcement this morning by Sangamo, a company that’s been trying to do gene therapy in the clinic. They’re not a CRISPR outfit, though – they’re using an alternate technique for gene silencing, using zinc finger regions instead. Even at that level, there’s mor… Read More
  • Cancer

    Real-Time Approval

    Here’s an example of the current regulatory framework – you may like it, you may not, but if you’re doing drug research you should know that it’s going on. The way it’s traditionally worked – for decades – has been that a company develops a drug, runs clinical trials, etc., puts together a (huge) data… Read More
  • Alzheimer's Disease

    Biogen and Eisai: Let the Alzheimer’s Arguing Commence

    Well, the tease is over: Biogen and Eisai have released data on their Alzheimer’s antibody, BAN2401. And the situation is messy, as many had feared. The top-line results are mostly positive, but there are several confounding factors that make them a matter of argument (see below). The companies were scheduled to go public with the… Read More
  • Alzheimer's Disease

    The Case of Verge Genomics

    A number of people have passed along the recent press stories about Verge Genomics, a new company out of YCombinator that has just raised $32 million for neuroscience drug discovery. Now that, as literally anyone who’s ever done it can tell you, is a hard field of a hard field, and I wish Verge good… Read More
  • Alzheimer's Disease

    Biogen And Eisai Tease an Alzheimer’s Result

    OK, we have kind of a complicated situation today. Biogen and Eisai have press-released what appear to be positive results for an anti-amyloid antibody therapy for Alzheimer’s. Since every single other attempt in this area has failed, often at great expense, what are we to make of this? Well, the first thing to say is… Read More
  • Business and Markets

    Welcome to Right to Try

    Update: Brainstorm has now abandoned their Right-to-Try approach. Who’s next? That didn’t take long. That didn’t take long at all. The federal “Right to Try” bill was just signed the other week, and we already have a company that’s willing – no, eager – to try it out. I will now cruelly caricature som… Read More
  • Clinical Trials

    Gene Therapy for Duchenne

    I have said unkind things about Sarepta’s drug for Duchenne muscular dystrophy, Exondys (eteplirsen). That’s because I did not think that there was enough information to approve it at the FDA, and I had trouble believing that its biochemical effects were enough to be meaningful in general. I have no reason to modify those opinions… Read More
  • Clinical Trials

    Finger-Pointing at Celgene

    Just what is going on at Celgene? They’ve had some odd recent setbacks (such as as the failure of Mongersen), and another such acquisition, Ozanimod, had its filing recently rejected by the FDA. Several things about that incident were eyebrow-raisers – for one, it’s rare for a large, established company to get an outright refusal- Read More
  • Alzheimer's Disease

    An Alzheimer’s Statement

    “Despite this latest setback, Eli Lilly remains committed to plunging through this concrete wall headfirst. This is a sad day for our WallBreaker 2020 program, and some of our longtime head bashers will recall similar periods during SkullButt 2012 and ConcreteCracker 2016. But we continue to believe that the only way past this wall is… Read More
  • Cancer

    More Thoughts on AbbVie’s Rova-T Implosion

    I wrote a couple of months ago about the disappointing results that AbbVie had obtained with their cancer stem cell therapy “Rova-T” (rovalpituzumab tesirine) in small-cell lung cancer. This was the antibody-drug conjugate that they’d purchased from Stemcentryx – OK, let’s clarify that, they bought Stemcentryx out comp… Read More
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