The firm said it has received positive feedback from the FDA on the design of a Phase III trial of ELI-002 in KRAS-mutant pancreatic cancer.

The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.

BH-30643 is an OMNI-EGFR inhibitor that targets classical, atypical, and compound EGFR mutations and acquired resistance mutations in HER2.

The companies will focus on advancing AXV-101, with which they hope to treat retinal dystrophy by targeting mutations in the BBS1 gene.

Doctors and patient advocates supporting pretreatment testing at times challenged FDA and other experts who aren't swayed by available evidence.

The deal advances Galapagos' plan to develop a decentralized CAR T-cell therapy for non-Hodgkin lymphoma with a one-week vein-to-vein time.

The company's multiple myeloma franchise, which includes Carvykti, stands to contribute significantly to its growth in 2025.

The resolution aims to provide Illinois patients with equitable access to emerging treatments, including precision and genomic medicines.

The company expects to submit a biologics license application for NTLA-2002 in 2026 and potentially launch it in the US in 2027.

NEW YORK – A first-in-human clinical trial is underway for an experimental treatment for X-linked chronic granulomatous disease (X-CGD) using a new approach to base editing, a type of gene editing.