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

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

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.