By delivering the protein follistatin via gene therapy, researchers at Washington University in St. Louis were able to increase skeletal muscle mass, decrease fat, and reverse obesity-related arthritis in mice who developed osteoarthritis as a result of a high-fat diet.
Chinese scientists have shown for the first time that the down-regulation of a single RNA-binding protein, polypyrimidine tract-binding protein 1 (Ptbp1), locally converted glial cells to neurons and showed promise for treating the symptoms of neurodegenerative diseases in mice.
An experimental gene editing therapy for an inherited form of blindness has become the first in vivo CRISPR medicine to be administered to patients, according to Editas Medicine Inc. and its partner, Allergan plc, which licensed the candidate in 2018.
New gene therapy treatments could add $45 billion to the cost of health care over the next five years, according to a new report from CVS Health Corp., of Woonsocket, R.I. While the number is staggering, without knowing the price of the currently unapproved therapies, how many patients will seek treatment and the likelihood of approval, the pharmacy benefit manager's estimate is basically an educated guess.
BEIJING – Chinese scientist He Jiankui has been sentenced to three years in prison and fined ¥3 million (US$429,421) for illegally carrying out the human embryo gene editing that led to the birth of twin girls and another baby with heritable changes to their genomes.
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.
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.