In a Phase I/II trial, patients with X-linked chronic granulomatous disease will receive a treatment created using a new ...

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

Their study showed that DCIS patients with pathogenic variants in BRCA1/2 and PALB2 had a higher risk of developing invasive breast cancer than those without.

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.

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

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

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

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

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

The company also continues to study Descartes-08 in other autoimmune diseases and has plans for a basket trial in pediatric patients.