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multiQure is a life sciences company developing therapies for rare genetic neurodegenerative disorders. The company's primary focus is RNAi gene therapy targeting Huntington's disease and other polyglutamine diseases, including SCA3 and DRPLA. Its therapeutic portfolio also includes immuno-oncology projects. The company was formerly known as Pure Biologics, a name it adopted after integrating the multiQure RNAi platform in 2026. Key drug candidates in development include PB003G and PB004, which are advancing toward Phase I clinical trials. The technology involves the selective silencing of mutated genes responsible for these conditions, with a specific molecule, amiR136-A2, designed to improve safety and efficacy. The company operates on a biotech model aimed at confirming the efficacy and safety of its RNAi therapy in patients. Summarised from the company's own disclosures, fiscal year ended Oct 2026 |
Industry Biotechnology | Homepage multiqure.com |
| Sector Healthcare | IR page multiqure.com | |
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Tags
gene therapy, rnai, neurodegenerative diseases, huntington's disease, polyglutamine diseases, immuno-oncology, rare diseases, PB003G, PB004, amiR136-A2, Pure Biologics
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