If the agency approves its application, the firm will begin a Phase I clinical trial in c-MYC-driven cancers in 2026.

Two family members with a germline ALK-mutant neuroblastoma both experienced complete response to ALK inhibitors in a case study.

The funds will support a Phase I/II trial of its gene therapy candidate in an aggressive form of dilated cardiomyopathy due to LMNA gene mutations.

Makers of organoids and cell models cheered the decision and said it will bolster the development of precision medicines, ...