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.
Epirium Bio Inc. has developed a series of small-molecule 15-PGDH inhibitors, the phase II-ready MF-300 and preclinical candidate MF-1305, as potential therapeutics for inflammatory bowel disease (IBD).
Andzonbio2 has signed agreements with the Alborada Drug Discovery Institute (ADDI) at the University of Cambridge and Cambridge Enterprise to advance a new class of therapeutics targeting neuroinflammation, a central driver of multiple neurodegenerative and neurological conditions.
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.
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.
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.
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.
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.
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.
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