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.

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 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.

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

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