Competitive RiskA rival company has advanced a regulatory submission for a competing gene therapy while certain kidney function data remain undisclosed, increasing competitive pressure and regulatory vulnerability for the company's programs.
Financing PressureA requirement to run a large randomized, sham‑controlled study would significantly increase trial size and duration, creating material additional capital needs and the potential for shareholder dilution.
Safety ConcernsDosing in higher groups was paused after patients experienced dose‑limiting toxicities, including notable liver enzyme elevations, creating uncertainty around optimal dosing and safety management.