CEO Brian Goff brings deep rare‑disease commercialization experience from Alexion, supported by a CMO/R&D head who led pivotal mitapivat programs and a seasoned CFO with large‑cap biotech finance experience. Execution in 2025–2026 shows progress: U.S. thalassemia launch, EU approval, GCC distribution, and an accepted sNDA for SCD.
Key tests ahead are maintaining disciplined spend, navigating REMS and payer logistics, and delivering outcomes that justify broader adoption. Track record and bench strength merit a positive view.







