Opportunities and Challenges in Developing Stem Cell Therapies

FDA approves Sun Pharma’s Ilumya for active psoriatic arthritis, BioMarin licenses a subcutaneous Fabry enzyme replacement therapy from GC Biopharma and Hanmi, and Lonza expands bioconjugation capacity in Visp to meet rising ADC demand.

Genmab’s petosemtamab plus pembrolizumab cuts the risk of death by 50% in phase 3 head and neck cancer, AbbVie submits a BLA for etentamig in relapsed or refractory multiple myeloma, and Benitec reports interim data for BB-301 gene therapy in OPMD-related dysphagia.

Direct-to-Biology workflows move crude reaction mixtures straight into assays, giving hit-to-lead teams earlier data to guide series selection.

BMS and SystImmune’s bispecific ADC iza-bren nearly doubles progression-free survival in EGFR-mutated lung cancer, GSK and Hansoh’s B7-H3 ADC Ris-Rez extends progression-free survival in relapsed osteosarcoma, and Immatics’ TCR bispecific IMA402 posts a 32% response rate in PRAME-high tumors.

Merck and Kelun’s sac-TMT extends overall survival in pretreated endometrial cancer, Bristol Myers Squibb reports interim data and a patient death in its phase 1 CAR-T trial in multiple sclerosis, and BioNTech and DualityBio’s HER2 ADC meets its phase 3 goal but with limited expected benefit.