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

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.

HHS is asking the court to affirm its Office of Inspector General's finding that paying for fertility services would run ...

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

The company anticipates launching three CRISPR products by 2030, including therapies for hereditary angioedema and ...

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

Despite Leqembi's initial slow sales, the firm's CEO cited areas for the drug's growth and ambitions for an antisense ...

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

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

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

The firm will pay $45 million upfront for several new targeted agents that it plans to pair with isotopes to develop both ...