Argonaut Manufacturing Services Selected to Manufacture siRNA Medicines for Potential Prion Disease Trial

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CARLSBAD, Calif.--(BUSINESS WIRE)--May 15, 2025--

The Vallabh/Minikel lab at the Broad Institute has selected Argonaut Manufacturing Services, Inc., “Argonaut,” as the drug product manufacturer for a therapeutic compound for a potential clinical trial for prion disease.

The Vallabh/Minikel lab has received FDA clearance for the compound, a divalent short interfering RNA (siRNA) intended for delivery via intrathecal injection, as an Investigational New Drug for symptomatic patients with prion disease. The team is now pursuing efforts to launch a clinical trial using this compound.

Prion disease, a rare but devastating neurodegenerative condition, is caused by the misfolding of the prion protein (PrP) and leads to rapid cognitive decline and death. Approximately 500 new cases are diagnosed each year in the United States. The investigational siRNA therapeutic is designed to hybridize with the mRNA encoding PrP, thereby reducing PrP expression levels and potentially altering the course of the disease. The Vallabh/Minikel lab is committed to advancing therapeutic options for patients with prion disease.

“We are proud to support the Broad Institute’s groundbreaking clinical trial efforts by providing critical manufacturing services for this important therapeutic candidate,” stated Argonaut CEO Rick Hancock. “Our collaboration underscores Argonaut’s commitment to advancing innovative treatments for rare and life-threatening diseases. While Argonaut provides clinical to commercial manufacturing of drug products, this partnership highlights the flexibility of our services, where it is critical to achieve maximum fill efficiency as every drop of this product counts.”

The project highlights Argonaut’s specialized capabilities in the manufacture of complex RNA therapeutics with near zero line losses, reinforcing its role as a trusted CMO partner for organizations pursuing novel genetic medicines.

Learn more about prion disease and the upcoming work from the Vallabh/Minikel lab to potentially launch a clinical trial.

About Argonaut

Argonaut Manufacturing Services, Inc. is an FDA-registered cGMP contract manufacturing organization (CMO) dedicated to providing custom manufacturing and supply chain solutions for biopharmaceutical and diagnostic companies. Aseptic drug fill/finish features state-of-the-art automated equipment for high-yield filling of sterile injectable drugs including biologics, peptides, small molecule, and vaccines. Diagnostic manufacturing includes proprietary lyophilization technology and a spectrum of kitting capabilities. Projects are supported with full analytical quality control services including warehousing and global shipping logistics. Serving innovators in the life science, molecular diagnostics, and biopharma industries, Argonaut provides a wide range of flexible solutions for diverse outsourcing needs.

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CONTACT: Media Contact

Scott Goldstein

Argonaut Manufacturing Services

[email protected]

KEYWORD: UNITED STATES NORTH AMERICA CALIFORNIA

INDUSTRY KEYWORD: BIOTECHNOLOGY PHARMACEUTICAL OTHER MANUFACTURING HEALTH FDA GENETICS MANUFACTURING CLINICAL TRIALS

SOURCE: Argonaut Manufacturing Services

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PUB: 05/15/2025 06:00 AM/DISC: 05/15/2025 06:02 AM

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