Discovery of STC15, an oally bivilble, highly setive METTL3 inibior for the treatment of AML and solid tumours: The fist molecule sifily trgeting an RNA mtytasferase enzyme to enter dinical development

Discovery of TNG462, a highly potent and selective MTA-cooperative PRMT5 inhibitor that is synthetic lethal for MTAP deleted cancers

TNG908 is a MTAP-cooperative PRMT5 inhibitor that can pass through the blood-brain barrier. TNG908 is 15 times more selective for MTAP null cell lines than MTAPWT cell lines, and can be used in cancer research.

PTC-258 2HCl is a specific and orally active splicing modulator of Elongator complex protein 1 gene (ELP1). PTC258 increases the expression of ELP1 in vitro and in vivo. PTC258 is well tolerated in mouse model.

JNT-517 is a small-molecule inhibitor of the phenylalanine transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe levels. The FDA has granted JNT-517 Rare Pediatric Disease Designation.

CHK-336 (Example 1) is an orally active LDHA inhibitor (IC50<1 nM) that inhibits lactate production in mouse hepatocytes. CHK-336 can be used in the study of hyperoxaluria.

Activity of KIN-3248, a next-generation pan-FGFR inhibitor, against acquired FGFR-gatekeeper and molecular-brake drug resistance mutations

KER-047 is a selective ALK2 inhibitor that has the potential to normalize ALK2 signaling and the downstream sequalae that results in anemia as a result of elevated hepcidin.

BCPA is a Pin1 regulator without cytotoxicity. BCPA attenuates the reduction of Pin1 protein to inhibit receptor activator of RANKL-induced osteoclastogenesis. BCPA regulates osteoclast activation, used to osteoporosis research.

CHBO4 is a potent, reversible, competitive, and selective hMAO-B inhibitor with an IC50 value of 0.031 μM in CHBO subseries and an Ki value of 0.010 ± 0.005 μM. CHBO4 reduce cell damage by scavenging intracellular reactive oxygen species (ROS). CHBO4 can be used for Parkinson's Disease (PD) research.

IK930 (compound I-32) is a potent and orally active TEAD inhibitor with an EC50 value of <0.1 µM.

Methopterin has the activation and bone resorption function of murine osteoclasts.

Nicotinamide ascorbate is a bioactive chemical.

IAJD93(IAJD-93) is a pentaerythritol-based one-component ionizable amphiphilic Janus Dendrimer (IAJD), delivery systems for mRNA delivery.

IAJD 288(IAJD-288) is a pentaerythritol-based one-component ionizable amphiphilic Janus Dendrimer (IAJD), delivery systems for mRNA delivery.

IAJD249(IAJD-249) is a pentaerythritol-based one-component ionizable amphiphilic Janus Dendrimer (IAJD), delivery systems for mRNA delivery.

Anticancer agent 118, a N‑acylated ciprofloxacin derivative, has anti-bacterial and anticancer activities.

MtUng-IN-1 (Compound 18a) is a Uracil DNA glycosylase of Mycobacterium (MtUng) inhibitor (IC50: 300 μM). MtUng-IN-1 can be used for research of cancers and infectious diseases.

CL1H6 is an ionizable lipid and CL1H6-LNP is capable of efficiently introducing both siRNA and mRNA into NK-92 cells.

Lipid 331 is a biodegradable cyclic ionizable lipid. LNPs containing Lipid 331 result in robust transfection in the nasal and lung tissues of mice and efficient transfection of lung epithelial cells and lung-resident APCs. Lipid 331 is a promising candidate for mRNA vaccine delivery, offering the potential for further enhancing the potency of mRNA vaccines.

GalNAc-L96 free base, the G-rich oligonucleotides carrying the longer GalNAc linker that can be used for delivery of nucleic acid drugs.