The Emergence of Oligonucleotide Building Blocks in the Multispecific Proximity-Inducing Drug Toolbox of Destruction DOI
K. Zhou, Katherine E. Bujold

ACS Chemical Biology, Journal Year: 2024, Volume and Issue: unknown

Published: Dec. 20, 2024

Oligonucleotides are a rapidly emerging class of therapeutics. Their most well-known examples informational drugs that modify gene expression by binding mRNA. Despite inducing proximity between biological machinery and mRNA when applied to modulating expression, oligonucleotides not typically labeled as "proximity-inducing" in literature. Yet, they have recently been explored building blocks for multispecific proximity-inducing (MPIDs). MPIDs unique because can direct endogenous destroy targeted molecules cells, contrast traditional inhibit only their functions. The mechanism action has enabled the targeting previously "undruggable" molecular entities cannot be effectively inhibited. However, development must ensure these will selectively potent, destruction-based toward intended targets over healthy tissues avoid causing life-threatening toxicities. emerged promising design sequence-controlled rationally designed program interactions. In this Review, we examine emergence oligonucleotide-containing induction space, which dominated antibody small molecule MPID modalities. Moreover, developed candidates immunotherapy protein degradation discussed demonstrate utility expanding scope selectivity toolbox. Finally, discuss programming "AND" gates into oligonucleotide scaffolds encode conditional responses potential incorporated MPIDs, further enhance selectivity, thus increasing drug category.

Language: Английский

Evolution of antisense oligonucleotides: navigating nucleic acid chemistry and delivery challenges DOI

Ruchi Ruchi,

Govind Mukesh Raman,

Vikas Kumar

et al.

Expert Opinion on Drug Discovery, Journal Year: 2024, Volume and Issue: unknown

Published: Dec. 9, 2024

Antisense oligonucleotide (ASO) was established as a viable therapeutic option for genetic disorders. ASOs can target RNAs implicated in various diseases, including upregulated mRNA and pre-mRNA undergoing abnormal alternative splicing events. Therapeutic applications of have been proven with the Food Drug Administration approval several drugs recent years. Earlier enzymatic stability delivery remains big challenge ASOs. Introducing new chemical modifications formulations resolving issues related to nuclease Excitingly, ASOs-based bioconjugates that hepatocyte gained much attraction. Efforts are ongoing increase application extrahepatic tissue well.

Language: Английский

Citations

0

The Emergence of Oligonucleotide Building Blocks in the Multispecific Proximity-Inducing Drug Toolbox of Destruction DOI
K. Zhou, Katherine E. Bujold

ACS Chemical Biology, Journal Year: 2024, Volume and Issue: unknown

Published: Dec. 20, 2024

Oligonucleotides are a rapidly emerging class of therapeutics. Their most well-known examples informational drugs that modify gene expression by binding mRNA. Despite inducing proximity between biological machinery and mRNA when applied to modulating expression, oligonucleotides not typically labeled as "proximity-inducing" in literature. Yet, they have recently been explored building blocks for multispecific proximity-inducing (MPIDs). MPIDs unique because can direct endogenous destroy targeted molecules cells, contrast traditional inhibit only their functions. The mechanism action has enabled the targeting previously "undruggable" molecular entities cannot be effectively inhibited. However, development must ensure these will selectively potent, destruction-based toward intended targets over healthy tissues avoid causing life-threatening toxicities. emerged promising design sequence-controlled rationally designed program interactions. In this Review, we examine emergence oligonucleotide-containing induction space, which dominated antibody small molecule MPID modalities. Moreover, developed candidates immunotherapy protein degradation discussed demonstrate utility expanding scope selectivity toolbox. Finally, discuss programming "AND" gates into oligonucleotide scaffolds encode conditional responses potential incorporated MPIDs, further enhance selectivity, thus increasing drug category.

Language: Английский

Citations

0