One of the world's leading gene therapy companies recently entered a global collaboration with Roche that could see Australian patients gain timely access to an emerging curative treatment for Duchenne muscular dystrophy.
US-based Sarepta Therapeutics signed the deal with Roche that means the Swiss company acquired non-US global rights to SRP-9001.
Under the deal, which is considered the largest gene therapy licensing agreement, Sarepta received an upfront payment of US$750 million in cash and US$400 million in equity. It will also receive regulatory and sales milestones as well as royalties on net sales. Sarepta will manufacture SRP-9001 in the US.
Duchenne muscular dystrophy is a rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death.
SRP-9001 is designed to deliver the microdystrophin-encoding gene directly to the muscle tissue for the targeted production of the microdystrophin protein.
The therapy is currently in late-stage development with trial results anticipated in 2021.
Small emerging biopharmaceutical companies are responsible for many of the promising gene therapies currently in development. However, few of these companies have any presence in Australia, meaning global licensing deals like Sarepta's with Roche will be pivotal in ensuring patient access to the new generation of treatments.
According to Doug Ingram, president and CEO of Sarepta, “As a mission-driven organization, we are inspired to partner with Roche with the goal of bringing SRP-9001 to patients outside the United States. This collaboration will not only increase the speed with which SRP-9001 could benefit patients outside the United States, but will also greatly expand the scope of territories within which we could potentially launch SRP-9001 to improve and save lives.”
Pfizer is also developing a gene therapy for the treatment of Duchenne muscular dystrophy. It gained rights to PF-06939926 through its 2016 acquisition of Bamboo Therapeutics.