For biopharma, 2019 can be described as a terrific year – with a few asterisks. The financial markets were flourishing, with venture capital dollars, in particular, flowing to the sector, while dealmaking reached historic proportions. Meanwhile, scientific breakthroughs led the way as cell and gene therapies gained ground, the first signs of success emerged with new technologies like CRISPR and the long-awaited promise of genomics found its way to the front lines of health care.
A single injection of SOD1-targeting RNA into the subpial space, which is below the innermost meningeal layer, was able to spread throughout the spinal cord and, via retrograde delivery, into brain centers that project to the spinal cord in several animal models, including primates.
A single injection of SOD1-targeting RNA into the subpial space, which is below the innermost meningeal layer, was able to spread throughout the spinal cord and, via retrograde delivery, into brain centers that project to the spinal cord in several animal models, including primates.
Startup firm Dyno Therapeutics Inc. is attempting to engineer a new generation of adeno-associated virus (AAV) capsids by navigating its way across what it calls the “capsid fitness landscape,” in order to optimize the key parameters that affect capsid performance: production, delivery efficiency, biodistribution, immunogenicity and thermostability.
Audentes Therapeutics Inc. CEO Matthew Patterson early last month characterized results with lead compound AT-132 in X-linked myotubular myopathy (XLMTM) as “unprecedented in neuromuscular disease,” and the value apparently wasn’t lost on Tokyo-based Astellas Pharma Inc., which signed a deal worth about $3 billion to take over the company. Shares of Audentes (NASDAQ:BOLD) closed at $58.93, up $30.32, or 106%, on word of the buyout – which pairs the two firms’ gene therapy expertise and is slated to close in the first quarter of next year – at a cost of $60 per share in cash.
BOSTON, Mass. – KRAS used to be the poster child for undruggability. No more. Amgen Inc. and Mirati Therapeutics Inc. are in the clinic with KRAS inhibitors. Boehringer Ingelheim GmbH is planning to join them by the end of the year. And multiple other companies are moving programs forward.
The mantra for a while has been that it's not good enough to get your drug approved, you've got to get it payed for, too. For gene therapy treatments, the payment is more complex, and you can add manufacturing and supply chain logistics to the list of challenges.
The leucine-repeat rich kinase 2 (Lrrk2) gets most of its attention in the context of Parkinson's disease (PD). Variants in Lrrk2 are a major cause of familial PD (though familial PD makes up only a small fraction of overall PD cases).
TEL AVIV, Israel – With shares now listed in the U.S. and data from a pivotal trial for its key bladder cancer therapy coming soon, Anchiano Therapeutics Ltd. is poised to ride the wave of interest in gene therapy.
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