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.
Dublin – Shares in Combigene AB, a little-known Swedish gene therapy firm, surged upward by 172% Oct. 12 on news of a preclinical licensing deal in epilepsy with Spark Therapeutics, which is potentially worth $328.5 million.
Poseida Therapeutics Inc.’s R&D Day in February – where much of its technology was made public for the first time – created “a flood of interest” in deals and officials were “pretty selective,” said CEO Eric Ostertag, whose remarks came as the company nailed down a whopping research collaboration and exclusive license agreement with Takeda Pharmaceutical Co. Ltd. The arrangement will deploy Poseida’s Piggybac and Cas-CLOVER, as well as biodegradable DNA and RNA nanoparticle delivery technology and other genetic engineering platforms to come up with as many as eight gene therapies.
Intergalactic Therapeutics Inc. is aiming for the stars with a nonviral gene therapy platform, backed with $75 million in series A financing from life sciences venture capital firm Apple Tree Partners. While management are tongue in cheek about the name of the company, they are serious about their mission to produce a next generation of gene therapies that overcome the issues associated with marketed adeno-associated virus-based products.
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