MY favourite bit of understatement ever comes not from a Brit or a Spartan but from the Japanese Emperor Hirohito. In August 1945, following Japan’s defeats […]

Small molecules recruit cyclophilin A to the active state of mutant KRAS to disrupt oncogenic signaling and tumor growth.

Behind the scenes, companies including Novartis, Takeda, and Gilead are collaborating in a loose alliance. Their work might not be done in time to stop COVID-19, but they hope it can prevent the next...

Regulators in the U.K. on Thursday approved a CRISPR-based medicine to treat both sickle cell disease and beta thalassemia, making it the world’s first therapy based on the revolutionary gene-editing...

Brousseau et al. have identified macrocyclic peptides that bind PCSK9 with low-nanomolar affinity, disrupt PCSK9 function, and utilize an induced-fit pocket. 13PCSK9i functions by a distinct,...

Nature - HRO761 is a potent, selective, allosteric WRN inhibitor that binds at the interface of the D1 and D2 helicase domains, locking WRN in an inactive conformation.

Nature - Compounds that enable selective autophagy of mutant Huntingtin protein.

The prize was awarded to William G. Kaelin Jr., Peter J. Ratcliffe and Gregg L. Semenza for discoveries about how cells sense and adapt to oxygen availability.

Bonazzi et al. describe the discovery of clinical candidate NVP-DKY709, a selective degrader of the IKZF2 transcription factor that was identified on the basis of recruitment of IKZF2 to CRBN....

The open source library features a diverse, large array of small molecules for scientists to screen against drug targets

Nature Chemical Biology - A targeted protein stabilization platform termed deubiquitinase-targeting chimera (DUBTAC) was developed based on heterobifunctional small molecules consisting of a...

Designing molecules that make bonds with their biological targets is in vogue

Since joining the Novartis Institutes for BioMedical Research (NIBR) two years ago, I have faced this question many times and in many settings. I must admit responding almost reflexively and dismis...

YTB323, a CD19-directed chimeric antigen receptor (CAR) T-cell therapy, retains T-cell stemness after a manufacturing process time of less than 2 days and demonstrates clinical antitumor activity at...

HSCs are particularly dependent on maintaining proteostasis, but how they eliminate misfolded proteins is unknown. Chua et al. demonstrate that HSCs traffic misfolded proteins to aggresomes in a...