Tolerability is a key factor in the appeal of Enliven Therapeutics Inc.’s ELVN-001 for previously treated chronic myeloid leukemia (CML), which yielded positive updated phase I data from the Enable trial. Dennis Kim, hematologist from Princess Margaret Cancer Center in Toronto, said he would use the drug for a patient “who doesn’t have any other options.”

China’s National Medical Products Administration (NMPA) on June 4 granted conditional approvals to two oncology drugs from Lupeng Pharmaceutical Co. Ltd. and Vcare Pharmatech Co. Ltd., as well as one hemophilia drug developed by Staidson Biopharmaceutical Co. Ltd.

Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional Crispr-Cas9 editing.

In its largest acquisition to date, Incyte Inc. snagged rights to phase III-stage VGA-039, a monoclonal antibody the firm said could offer a new standard of care in von Willebrand disease (VWD), in a buyout of Vega Therapeutics Inc. that includes a $1.25 billion up-front payment and up to $750 million tied to the achievement of sales milestones. The announcement was followed shortly by news of another buyout, Johnson & Johnson’s deal to acquire Firefly Bio Inc. for $1 billion in cash, both moves continuing this year’s trend of larger, more strategic M&A transactions.

Agios Pharmaceuticals Inc.’s potential $665 million deal for Oscotec Inc.’s cevidoplenib, a phase III-ready oral SYK inhibitor to treat immune thrombocytopenia, marks a new chapter of development focused on cancer therapy resistance for Oscotec, CEO Yoon Tae-young said.

Shares of Fulcrum Therapeutics Inc. (NASDAQ:FULC) fell 54% after the company said it is discontinuing work on sickle cell disease (SCD) candidate pociredir, its only clinical-stage candidate, and reviewing strategic alternatives in the wake of the U.S. FDA’s safety concerns regarding the drug target.

Payoff for the November 2023 buyout of Immunogen Inc. came for Abbvie Inc. in the form of U.S. FDA clearance for the CD123-targeting antibody-drug conjugate Decnupaz (pivekimab sunirine) to treat adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an ultra-rare, aggressive and quick-killing hematologic malignancy.

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.

In a deal potentially worth up to $15.2 billion, Jiangsu Hengrui Pharmaceuticals Co. Ltd. is joining efforts with Bristol Myers Squibb Co. to advance 13 early development programs in the fields of oncology, hematology and immunology. Shanghai-based Hengrui will hold exclusive rights in mainland China, Hong Kong and Macau, while Princeton, N.J.-based BMS will hold exclusive rights in the rest of the world. The deal includes four oncology/hematology assets from Hengrui, four immunology assets from BMS, and five assets that the two companies will jointly discover and develop.