May 8th 2024
Cell-selective, adeno-associated virus gene therapy DB-OTO restored hearing to normal levels within 24 weeks in a child born with profound genetic deafness due to variants of the otoferlin gene.
Phase III Trial Data for Scemblix Show Clinically Meaningful Response in Chronic Myeloid Leukemia
January 8th 2024Phase III ASC4FIRST show Scemblix (asciminib) plus investigators’ choice of tyrosine kinase inhibitor produced a statistically significant response in patients newly diagnosed with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase.
Sharon Hesterlee of the Muscular Dystrophy Association Speaks on New Therapy, Vamorolone
December 18th 2023In an interview with ACT editor, Andy Studna, Hesterlee, Chief Research Officer at the Muscular Dystrophy Association discusses a new therapy for Duchenne Muscular Dystrophy, Vamorolone, and what sets it apart from other therapies in the market.
Novel Cancer Vaccine Plus Keytruda Cuts Risk of Death, Recurrence by Nearly Half in Phase IIb Trial
December 15th 2023A planned analysis of the KEYNOTE-942/mRNA-4157-P201 clinical trial found that at a median follow-up of approximately three years, adjuvant treatment with Moderna’s mRNA-4157 (V940) in combination with Keytruda continued to show a clinically meaningful improvement in recurrence-free survival in patients with resected high-risk melanoma.