rna

Thailanstatin A is an ultra-potent inhibitor of eukaryotic RNA splicing (IC50 = 650 nM). It inhibits multiple cancer cell lines via non-covalent binding to the SF3b subunit of the U2 snRNA subcomplex of the spliceosome, displaying low-nM to sub-nM IC50s. Thailanstatin A, as a payload for ADCs, is conjugated to the lysines on trastuzumab, yielding linker-less ADC.

RNA recruiter-linker 1 is part of the RNA ligand-linker in RNAse L RIBOTAC, which is an RNA-degrading chimera designed to bind the four-stranded RNA helix SL5 within the 5' untranslated region of the SARS-CoV-2 RNA genome. This binding inhibits the replication of the virus in lung epithelial carcinoma cells. RNA recruiter-linker 1 is also useful in RIBOTAC synthesis.

RNA recruiter 1 is the RNA ligand segment of RNAse L RIBOTAC, an RNA degradation chimera capable of binding the four-stranded RNA helix SL5 in the 5' untranslated region of the SARS-CoV-2 RNA genome, thereby inhibiting viral replication in lung epithelial carcinoma cells. RNA recruiter 1 is useful for the synthesis of RIBOTAC.

RNAse L RIBOTAC (compound C64) is an RNA-degrading chimera that binds to a four-way RNA helix called SL5 located in the 5’ UTR of the SARS-CoV-2 RNA genome, thereby inhibiting viral replication in pulmonary epithelial cancer cells.

RNase L ligand 1 (Compound F1-COOH) serves as a ligand for RNase L and is utilized in the synthesis of F1-RIBOTAC.

Methotrexate disodium is an tetrahydrofolate dehydrogenase inhibitor

Tri-GalNAc-PEG3-Azide is a specialized compound designed for lysosomal targeting chimera (LYTAC) research, featuring three asialoglycoprotein receptor (ASGPR) ligands linked by a PEG3 spacer and an azide group for easy conjugation. This compound facilitates efficient internalization into hepatocytes through ASGPR-mediated endocytosis, making it a strategic tool for delivering RNA, Cas9 complexes, or other cargo specifically to liver cells. Additionally, it can be utilized to create LYTACs or to be marked with a dye for tissue imaging applications.

MC-VC-PABC-DNA31 is a drug-linker conjugate designed for Antibody-Drug Conjugates (ADC), exhibiting potent antitumor activity. It utilizes DNA31, a powerful inhibitor of RNA polymerase, connected via the MC-VC-PABC ADC linker.

F1-RIBOTAC is a ribonuclease-targeting chimera (RIBOTAC) that reduces QSOX1-amRNA expression through an RNase L-dependent mechanism. It is applicable in cancer research. (Pink: RNA ligand; Black: linker; Blue: RNase L ligand.)

PT-129 is a RPOTAC degrader that targets the G3BP1 2NTF2 domain (protein-RNA interaction site), facilitating the breakdown of intracellular stress granules. It prevents the formation of stress granules in stressed cells and deconstructs existing ones, thereby disrupting ATF4 transmission and inhibiting cancer cell proliferation. Stress granules (SGs) are membraneless cytoplasmic compartments formed under stress, which aid in the transfer of ATF4 from fibroblasts to tumor cells, promoting fibroblast-related tumor growth. G3BP1 2 serve as central proteins in the SG network, and inhibiting them may reduce the stress resilience of cancer cells within the tumor microenvironment. PT-129 consists of a target protein ligand (red part) G3BP1 2-Targeting ligand-1, an E3 ligase ligand (blue part) Thalidomide 4-fluoride, and a PROTAC linker (black part) Amino-PEG3-C2-acid; the E3 ligase ligand and linker form the complex Thalidomide-NH-PEG3-propionic acid.

Mal-C6-α-Amanitin, an ADC (antibody-drug conjugate) linker drug, incorporates the powerful antitumor agent α-Amanitin, an inhibitor of RNA polymerase II, through the Mal-C6 linker, showcasing potent antitumor activity.

Dth is a proteolysis-targeting chimera (PROTAC) composed of the fluorescent RNA probe DFHBI and the immunomodulatory compound thalidomide.

MC-Val-Cit-PAB-rifabutin is a drug-linker conjugate for antibody-drug conjugates (ADC), featuring the potent antitumor agent rifabutin (a DNA-dependent RNA polymerase inhibitor) connected via the MC-Val-Cit-PAB ADC linker, which endows it with significant antitumor activity.

Dovitinib RIBOTAC TFA effectively and selectively cleaves pre-miR-21, functioning as a targeted RNA degrader.

Dovitinib RIBOTAC is a potent, selective targeted RNA degrader that cleaves pre-miR-21.