EP4551701A1 — Methods for in vivo editing of klkb1
Assigned to Intellia Therapeutics Inc · Expires 2025-05-14 · 1y expired
What this patent protects
The first systemic administration of a CRISPR/Cas9-based therapeutic for in vivo editing of a liver KLKB1 gene in a clinical trial is described. Described herein are methods for in vivo editing of a liver KLKB1 gene by systemically administering a lipid nanoparticle compo…
USPTO Abstract
The first systemic administration of a CRISPR/Cas9-based therapeutic for in vivo editing of a liver KLKB1 gene in a clinical trial is described. Described herein are methods for in vivo editing of a liver KLKB1 gene by systemically administering a lipid nanoparticle composition comprising an mRNA encoding a Cas9 nuclease and a guide RNA that targets the gene. Assessment of biosafety metrics and clinical efficacy metrics, as well as methods of treatment, are also described herein.
Drugs covered by this patent
Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.
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