C12N2310/3511

CONJUGATED ANTISENSE COMPOUNDS AND THEIR USE

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.

Compositions and methods for modulating apolipoprotein C-III expression

Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein C-III (ApoCIII). In certain embodiments, the ApoCIII targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting ApoCIII for use in decreasing ApoCIII to treat, prevent, or ameliorate diseases, disorders or conditions related to ApoCIII. Certain diseases, disorders or conditions related to ApoCIII include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.

Compositions and methods for modulating TTR expression

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.

EXTRACELLULAR VESICLE COMPRISING A BIOLOGICALLY ACTIVE MOLECULE AND A DUAL CLEAVABLE LINKER
20260055411 · 2026-02-26 ·

The present disclosure relates to extracellular vesicles (e.g., exosomes) comprising a biologically active molecule covalently linked to the extracellular vesicle via a dual cleavable linker and an anchoring moiety. The extracellular vesicles can be useful as an agent for the prophylaxis or treatment of cancer or other diseases. Also provided herein are methods for producing the extracellular vesicles and methods for using the extracellular vesicles to treat diseases or disorders.

COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRESSION

Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein C-III (ApoCIII). In certain embodiments, the ApoCIII targeting oligomeric compounds are conjugated to N-Aceteylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting ApoCIII for use in decreasing ApoCIII to treat, prevent, or ameliorate diseases, disorders or conditions related to ApoCIII. Certain diseases, disorders or conditions related to ApoCIII include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat disease, disorders or conditions in an individual in need thereof.

Light-inducible CRISPR/Cas9 system for genome editing

A very fast and efficient CRISPR/Cas9 system is provided. Compositions include light-sensitive caged nucleotides at the PAM distal region of guide RNAs (gRNA.sup.caged) to create artificial mismatches as a roadblock. Upon light stimulation, the caging group (roadblock) is removed and the gRNA fully hybridizes with the target DNA. Thus, the pre-bound inactive Cas9/gRNA.sup.caged is rapidly converted to active Cas9.