A clinical-stage, fully integrated, next generation, systemic AAV based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases. We aim to deliver one-time gene therapy treatments that provide functional cures through permanently sustained physiological protein levels, leveraging the high expression enabled by our proprietary gene therapy platform. Our initial focus is on developing treatments for monogenic diseases with high unmet need.
Lead Underwriter
Evercore Group L.L.C, J.P. Morgan Securities LLC, Morgan Stanley & Co. LLC
