Throughout the year we have published the views of company executives, government regulators, industry analysts and scientists on a variety of topics and, in our popular annual feature, we include a selection of these that paints a picture of the significant events that shaped 2019. The major talking point was on the capital markets front where investors turned their backs on the biopharmaceutical sector for most of the year returned big time in the final quarter.
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.
BEIJING – Gene therapy startup Hui-Gene Therapeutics Ltd. Co., of Shanghai, said it secured more than ¥100 million (US$14 million) in a series A financing round to develop a safer gene therapy to treat genetic diseases caused by single-base mutations.
ORLANDO, Fla. – At the 61st ASH annual meeting late-breaking abstracts session, researchers from Boston Children’s Hospital reported that three adult patients who had received an autologous transplant of gene-edited hematopoietic stem cells lacking BCL11A produced high levels of functional hemoglobin and had reduced disease symptoms for at least eight months after transplantation.
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.
After a two-month 32% swoon in the value of the BioWorld Gene Therapy index, it has managed to recover 6% of that loss since the end of September. Contributing to the recovery was Menlo Park, Calif.-based Adverum Biotechnologies Inc., whose shares (NASDAQ:ADVM) jumped almost 80% in the period.
It has been a productive year for cell and gene therapy companies, and every week the sector never fails to generate exciting news flow to keep investors engaged. Given these ongoing developments, it is not surprising that companies involved in the area continue to attract financing and strategic partners.
LONDON – After a long haul to market, recent approvals and initial commercial successes of advanced cell and gene therapies are shifting the balance of power between biotech and pharma in dealmaking.
DUBLIN – Amarna Therapeutics BV raised €10 million (US$11.1 million) in new financing to move its SV40-based gene therapy platform, SVac, toward the clinic.
