RNA-based vaccines and therapeutics may have faced a bumpy road in the U.S. recently, but a major new federal investment proves the government is still betting big on its future.
The Advanced Research Projects Agency for Health (ARPA-H) has narrowed down to five teams—a mix of biotechs and academic institutions—that will now receive up to $125 million in aggregate to help automate the production process for potential on-demand, personalized RNA-based medicines.
The new project falls under ARPA-H’s Genetic Medicines and Individualized Manufacturing for Everyone (GIVE) program, which it says aims to build out automated production and real-time quality control through a distributed network. The idea, according to the agency, is to enable manufacturing of treatments near the patients they serve, rather than keeping genetic medicines sequestered around “major biomanufacturing hubs.”
Manufacturing expenses and the logistical headaches associated with the actual administration of genetic medicines—such as gene therapies—have proven to be a limiting factor for the field’s uptake in the real world.
Four of the groups tapped for the project will focus on developing automated platforms for RNA-based drugs that can also perform quality control testing either at or near the patient point of care.
These include Centillion Biosciences, which will focus on a system combining cartridge-based tech for DNA and RNA manufacturing with integrated fill, finish, QC and digital process management.
HDT Bio, for its part, will work on a platform combining automated DNA synthesis, chip-based RNA production, closed fill-finish and robotics-enabled quality control testing; while Waterfall Scientific aims to create an automated, benchtop continuous-flow system that combines proprietary tech “from a consortium of expert organizations” to produce RNA drug products.
Massachusetts General Hospital has also enlisted in the cause and will develop a single-use, all-fluidics chip system and control unit hardware for continuous manufacturing and QC testing.
And lastly, the University of Utah will focus exclusively on quality control and has been tapped by ARPA-H to create an “all-in-one genetic medicine testing platform” that can perform full QC testing and lot release within as little as a day.
Should any of those technologies hold promise, ARPA-H said that the GIVE program will work with the FDA to co-develop regulatory guidelines necessary to bring those manufacturing innovations to scale.
In contextualizing the funding, ARPA-H pointed to the groundbreaking treatment of an infant named KJ last year, who became the first patient treated with an in vivo CRISPR gene-editing therapy designed just for him.
But therapies like the one that helped baby KJ thrive currently take “months to manufacture, cost hundreds of thousands of dollars, and require years of clinical trials to prove safe and effective,” ARPA-H said in its project announcement, stressing the need for a faster and more efficient manufacturing system.
ARPA-H was created during Joe Biden’s presidency and survived into President Trump’s second term, despite other substantial cuts to federal science funding by the government since last year.
In setting out her goals for the agency going forward, Alicia Jackson, Ph.D., ARPA-H’s new leader, told Fierce earlier this year that she wants the U.S. to be “winning the biotech race” by supporting bold new technologies that the industry might find too risky to fund.