The future biotech landscape is not US vs. China, but a "multipolar" world where savvy companies operate as "hybrid biotechs." They will selectively build bridges, cherry-picking talent, capabilities, and operational models across the US, Europe, and China to accelerate development.
A new wave of therapies for Stargardt disease is moving beyond simply slowing progression. Approaches like optogenetics aim to restore vision even in advanced patients by creating new light-sensing capabilities in retinal cells, bypassing the photoreceptors already lost to the disease.
Europe, despite excellent science, lost its co-equal status in drug development to the U.S. due to restrictive pricing and lack of growth capital. These same challenges are now emerging in the U.S., threatening its innovation leadership as China accelerates its efforts.
Commissioner Marty McCary's unprecedented public discussion of a pending therapy and a director's political affiliations reveal a highly politicized FDA. Describing CBER Director Vinay Prasad as being "on loan" suggests his tenure is fragile and agency leadership is unstable.
Large multinational pharma companies publicly express concern about the threat from China's biopharma sector. Simultaneously, these same companies are investing billions, actively integrating China into the global ecosystem and contradicting their own zero-sum game narrative.
While the FDA's new "plausible mechanism framework" is officially for bespoke, N-of-one therapies, experts at its rollout expressed an expectation that its principles could be applied more broadly. This suggests a potential new pathway for other rare diseases, moving beyond an ultra-rare scope.