Ultragenyx, Arcturus sign messenger RNA therapeutics deal

The companies have signed collaboration and licensing agreement, which is expected to generate $1.56bn for Arcturus.

In the first phase of the collaboration, Arcturus will design and optimize mRNA therapeutics for two selected rare disease targets, using its unlocked nucleomonomer agent (UNA) Oligomer chemistry and LUNAR nanoparticle delivery platform.

Ultragenyx has the option to add up to eight additional rare disease targets. The company will also oversee the development and commercialization of all products developed with Arcturus.

In return, Arcturus will receive an upfront payment of $10m, milestones of up to $156m for each target.

Arcturus will also be reimbursed by Ultragenyx for all research expenses, as well as mid-single to low double-digit royalties on commercial sales in the mid-single to low double digits.

Ultragenyx Pharmaceutical CEO Emil Kakkis said: "This collaboration with Arcturus will help us address a wider range of rare diseases than is possible with currently available approaches.

"The joint program combines Arcturus’ technology platform, which we believe solves some of the key issues associated with mRNA therapeutics, with Ultragenyx’s expertise in rare disease drug development."

Arcturus’ RNA therapeutics platform is designed to be applied towards small interfering RNA, messenger RNA, antisense RNA, microRNA, and gene-editing therapeutics.

The company’s UNA technology can target any gene in the human genome, as well as viral genes, and other species for therapeutic purposes.