20250170260. Nucleic Acid-polypep (Avidity Biosciences, .)
NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING
Abstract: disclosed herein are molecules and pharmaceutical compositions that induce an insertion, deletion, duplication, or alteration in an incorrectly spliced mrna transcript to induce exon skipping or exon inclusion. also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that induces an insertion, deletion, duplication, or alteration in an incorrectly spliced mrna transcript to induce exon skipping or exon inclusion.
Inventor(s): Arthur A. LEVIN, Andrew John GEALL, Beatrice Diana DARIMONT, Rob BURKE, Yunyu SHI, Michael Caramian COCHRAN, Hanhua HUANG, Venkata Ramana DOPPALAPUDI, Rachel JOHNS
CPC Classification: A61K47/6807 (PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms ; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles ; soap compositions ))
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- Patent Applications
- Avidity Biosciences, Inc.
- CPC A61K47/6807
- Arthur A. LEVIN of Del Mar CA US
- Andrew John GEALL of Carlsbad CA US
- Beatrice Diana DARIMONT of San Diego CA US
- Rob BURKE of Encinitas CA US
- Yunyu SHI of La Jolla CA US
- Michael Caramian COCHRAN of La Jolla CA US
- Hanhua HUANG of San Diego CA US
- Venkata Ramana DOPPALAPUDI of San Diego CA US
- Rachel JOHNS of La Jolla CA US