Clinical TrialsRP-A501 for Danon Disease is RCKT's leading in vivo asset and major valuation driver, with a high probability of success in its ongoing pivotal trial.
Financial PositionRCKT ended 4Q24 with $372.3mm in cash and equivalents, which is expected to fund operations effectively.
Market PotentialRP-A501 could have a significant impact on the estimated 18,000 Danon patients in the US and EU, who currently face assured heart transplant or death.