Financial PerformanceThe public offering results in gross proceeds of $165 million with an underwriter’s option for additional shares.
Gene Therapy SuccessThere is a high 65-75% chance of success for the pivotal Phase II study for Danon heart disease, which supports regulatory filings thereafter.
Positive Clinical DataAll evaluable patients from the Phase I study of RP-A501 in Danon disease remain alive and transplant-free, surpassing the indication's typical natural history.