Work at Blueprint Medicines Corp. to identify a selective and potent CDK4 degrader led to the identification of BLU-448, with minimal activity against CDK6 for treating HR+/HER2- breast cancer.

Resistance to microtubule-targeting agents such as taxanes and vinca alkaloids is often driven by drug efflux and changes in tubulin behavior. Targeting the colchicine-binding site provides an alternative strategy that may circumvent these resistance mechanisms.

Shanghai Henlius Biotech Inc.’s HLX-48 for injection has received approval from the Human Research Ethics Committee (HREC) in Australia and been acknowledged by the Therapeutic Goods Administration (TGA). The first-in-human phase I study in Australia will evaluate HLX-48 in patients with advanced or metastatic solid tumors.

A few months after winning its first regulatory clearance in China, Beone Medicines Inc.’s next-generation BCL2 inhibitor, sonrotoclax, gained the U.S. FDA’s accelerated approval for use in patients with relapsed or refractory mantle cell lymphoma (MCL) following treatment with a BTK inhibitor. The therapy, branded Beqalzi, marks the first BCL2-targeting drug for MCL in the U.S., but the company has pointed to a bigger opportunity in the potential combination with blockbuster BTK drug Brukinsa (zanubrutinib).

Circular RNA (circRNA) is not a new concept, but it is a novel strategy in the field of gene and cell therapy. While mRNA vaccines have revolutionized medicine, this RNA fragment without free ends surpasses their performance in both efficacy and durability, bringing it to the attention of several pioneering companies. The latest advances in circRNA presented at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) clearly surpass the performance achieved with linear mRNA.