Clinical TrialsEnrollment continues in the pivotal Phase 2 study of RP-A501 in Danon disease, which is viewed as the key value driver.
Financial StabilityRocket reported cash, cash equivalents, and marketable securities of about $278.8 million, which is expected to be sufficient to fund operations into 2026.
Regulatory ApprovalThere is a path forward in which Kresladi could be approved without a new BLA being submitted, potentially resulting in a 'surprise' announcement by year-end.