US puts $125m into personalised RNA therapy production
The US Advanced Research Projects Agency for Health (ARPA-H) has awarded $125 million in funding, split across five groups, to build a network for producing 'made-to-order' RNA-based genetic medicines.
The Genetic Medicines and Individualised Manufacturing for Everyone (GIVE) programme will replace a "slow, costly, centralised model with one created for speed, scale, and reach," according to ARPA-H, which expects it to increase access to new therapies for cancer, rare genetic conditions, and chronic diseases.
The project ties in with ongoing efforts by the FDA to develop new, accelerated routes to market for personalised therapies using the concept of a 'plausible mechanism'. That would allow new technologies, like genome editing and RNA-based therapies, to be approved based on a "single adequate and well-controlled clinical investigation with confirmatory evidence."
The FDA will work with GIVE on the regulatory framework needed to bring personalised therapies to patients at scale.
GIVE's programme manager, John Schiel, said that ramping up capacity for domestic biomanufacturing of RNA therapies will give the US a competitive advantage on the global stage, and "ensure that all patients have access to the latest therapeutic capabilities, no matter where they live."
The award has gone to four groups focusing on creating automated, distributed manufacturing platforms and quality control processes at or near the point of care; namely, Massachusetts General Hospital, Centillion Biosciences, HDT Bio, and Waterfall Scientific.
A fifth group at the University of Utah is tasked with building the production quality control systems – based on digital microfluidics, advanced optics, and AI – to allow the release of medicines as quickly as possible.
ARPA-H said that personalised treatments like Baby KJ's CRISPR cure for metabolic disorder CPS1 deficiency currently take months to manufacture, cost hundreds of thousands of dollars, and require years of clinical trials to prove safe and effective.
If GIVE succeeds, it contends, these therapies could be made locally and on demand, in a fraction of the time and cost, and also make it quicker and easier to run clinical trials.
"The proposed rule to modernise drug manufacturing reflects FDA's recognition that the future of drug manufacturing is distributed, and our regulatory framework needs to evolve alongside it," said Karim Mikhail, acting director of the FDA's Center for Biologics Evaluation and Research (CBER).
Harvard gets funds for emerging RNA class
Also this week, ARPA-H has said it will provide up to $4.4 million in funding to a project at Harvard Medical School, MIT's Whitehead Institute, and RNAV8 Bio that aims to accelerate the development of RNA medications that would use genetic switches – called riboswitches – that can regulate genes within the human body.
The project will use AI models, rapid RNA structural analysis, and large-scale screening to identify and engineer riboswitches that detect chemical signals associated with a broad range of diseases. RNAV8 Bio will focus on engineering and testing mechanisms for the delivery of potential therapies.
