Gene therapies rely on vectors to reach the target tissue where they act, such as adeno-associated viruses (AAVs) or lipid nanoparticles (LNPs), among other delivery strategies. Each combination is optimized for a specific cell type and indication, aiming to overcome challenges such as efficacy, specificity and toxicity. On May 13, 2026, two sessions included in the scientific symposia of the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), being held in Boston this week, addressed AAV-related toxicities, which have led to fatal cases in clinical trials and remain an area for improvement in approved therapies.

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.

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.

Chengdu Origen Biotechnology Co. Ltd. and Vanotech Ltd. have announced IND clearance by the FDA for KHN-921 for the treatment of hypertrophic cardiomyopathy (HCM) associated with MYBPC3 mutations.

RuvB-like 1 (RUVBL1) and its paralogue RuvB-like 2 (RUVBL2) are evolutionarily conserved members of the ATPases associated with diverse cellular activities (AAA) superfamily. In a recent study, researchers from Eikon Therapeutics Inc. aimed to leverage the protein dynamics of RUVBL1/2 to facilitate the development of novel inhibitors, instead of using ADP-based biochemical assays, to explore the therapeutic potential of this target.

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.