/Compositions And Methods Comprising A Ttr Guide Rna And A Polynucleotide Encoding An Rna-guided Dna Binding Agent
Abstract
Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.
Full Text
What is claimed is:
Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.
Timeline
Filed
03/26/2026Published
07/16/2026Granted
Not AvailableIPC Codes(4)
C12N 15/90:Stable introduction of foreign DNA into chromosome
A61K 38/00:Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K 31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K 31/00; ergoline-based peptides A61K 31/48; containing macromolecular compounds having statistically distributed amino acid units A61K 31/74; medicinal preparations containing antigens or antibodies A61K 39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K 47/00)
C12N 9/22:Ribonucleases