Jan. 2 at 6:33 PM
$RNAC Part 2 - They are also unique in that they have already built an FDA approved clinical‑grade cGMP facility at their corporate headquarters in Maryland. The science is solid and their efficacy results in the MG space exceed other biotechs playing in the same space. Also, the stock ownership amongst the founders, the operating/executive team and their clinical and scientific advisory committees is aligned with the success of the company - management, directors and insiders own about 1/3 of the FD shares (12M of 35M out). Institutions own a majority of the float. This company has a world-class team of Harvard and H-affiliated scientists on their boards. Finally, they already agreed to pivotal Phase 3 trial design with the FDA (ongoing) and have RMAT (rare disease), orphan drug for MG, and Rare Pediatric Disease designations with the FDA. They could file a 2027 BLA for MG - market potential (assuming mid-teens penetration) for this indication alone is +
$1B and implies a
$100 stock.
