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.
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.
Something of a duel may be shaping up between Menlo Park, Calif.-based Adverum Biotechnologies Inc. with ADVM-022, the phase I gene therapy candidate for wet age-related macular degeneration (AMD) and Regenxbio Inc., of Rockville, Md., with a similar candidate.
Tenaya Therapeutics Inc. CEO Faraz Ali told BioWorld that the company, which raised $92 million in a series B round, has programs from three cardiac platforms "stacked on top of each other" and wants to enter the clinic by the end of 2021 "with at least one of the multiple projects we're advancing." The South San Francisco-based firm wanted not to "let resources be the barrier" as to which goes first, he said. "We wanted the science to dictate that."
SEOUL – South Korea's investors have become very interested in the global cell and gene therapy market. Licensing and M&A deals in the field have been active – a good sign for Korean biopharma firms eager to tap in.
