A raft of potentially high-value drug development collaborations, most for gene and RNA therapies, led the first day of the 40th Annual J.P. Morgan Healthcare Conference Monday. Pfizer Inc. enlisted Beam Therapeutic Inc. to advance in vivo base editing programs for up to $1.35 billion, while Bayer AG tapped Mammoth Biosciences Inc.’s in vivo CRISPR systems expertise in a potential $1 billion-plus deal. Selecta Biosciences Inc. inked a new $1.1 billion partnership with Ginkgo Bioworks Inc. to develop next-generation gene therapy capsids, while Acadia Pharmaceuticals Inc. agreed to pay Stoke Therapeutics Inc. as much as $967 million to develop RNA-based medicines. Work on new mRNA vaccines and therapies is also proceeding, with both Pfizer and Biontech SE announcing new collaborations in the space.
Avrobio Inc., stung by variable outcomes in a phase II test of its investigational Fabry disease therapy, is quitting further enrollment in the program, one of the first from its gene therapy platform, Plato. The team's attention will shift instead to other clinical-stage lysosomal disorder programs amid "an increasingly challenging market and regulatory environment for Fabry disease," the company said.
DUBLIN – Novartis AG is deepening its commitment to ocular gene therapy by picking up Gyroscope Therapeutics Ltd. for $800 million up front and up to $700 million more in potential milestone payments.
Wall Street didn’t much like Uniqure NV’s decision to hold off reporting efficacy measures in the phase I/II trial with one-time gene therapy AMT-130 for the treatment of Huntington’s disease (HD), but safety findings proved encouraging and analysts held out hope.
LONDON – Aviadobio Ltd. has raised $80 million in a series A round to take a precision microdosed gene therapy for treating familial frontotemporal dementia into a phase I/II clinical trial.
Japan’s Astellas Pharma Inc. is continuing its investment in gene therapies, following up its $3 billion acquisition of Audentes Therapeutics Inc. with a technology licensing deal with Dyno Therapeutics Inc. potentially worth more than $1.6 billion. Central to the deal is Cambridge, Mass.-based Dyno’s adeno-associated virus (AAV) vector technology, which can be used to direct gene therapies to skeletal and cardiac muscle.
Sangamo Therapeutics Inc. rolled out pleasing preliminary data from the first four patients treated in the phase I/II study known as Staar, evaluating isaralgagene civaparvovec, or ST-920, a gene therapy for Fabry disease. Results as of the Sept. 17, 2021, cutoff date from the four patients in the first two dose cohorts showed that the drug was generally well-tolerated, and all four patients exhibited above normal alpha-galactosidase A activity.
Targeting the thousands of rare inherited diseases that have no treatments in the U.S., a newly launched public-private group plans to pursue efforts to optimize and streamline the gene therapy development process.
Sio Gene Therapies Inc. CEO Pavan Cheruvu said the company sees no “significant rate limiters to moving forward” with FDA fast-tracked AXO-AAV-GM1 for the treatment of GM1 gangliosidosis. Shares of the New York-based firm (NASDAQ:SIOX) closed at $2.36, up 21 cents, as Wall Street welcomed positive interim data from the ongoing phase I/II study with the adeno-associated viral vector 9-based gene therapy for GM1 gangliosidosis.
PTC Therapeutics Inc. is already established as a player in rare diseases, working with Roche Holding AG to develop and market Evrysdi (risdiplam) to treat certain patients with spinal muscular atrophy. With Evrysdi now approved in the U.S. and Europe, and Translarna (ataluren) approved in Europe for Duchenne muscular dystrophy, South Plainfield, N.J.-based PTC is approaching a crucial juncture with its first gene therapy product.
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