While regulatory uncertainty has dominated biopharma headlines this year, long-term data suggest that companies' own organizational deficiencies may be just as damaging. Consider that, on the same day in July 2025, three advanced therapy programs each received a Complete Response Letter (CRL) from the FDA.
All three — Capricor's CAP-1002 for Duchenne muscular dystrophy, Ultragenyx's UX111 for Sanfilippo syndrome type A, and Rocket Pharmaceuticals' Kresladi for leukocyte adhesion deficiency — were rejected for CMC deficiencies. These structural issues had been building for some time; analysis of FDA CRLs from 2020 to 2024 showed that 74% cited manufacturing or quality issues.
And that pattern may also be a harbinger of future struggles, if the industry, which has been slow to confront the issue, neglects the following reality about drug development: The primary constraint is no longer scientific or regulatory but organizational. And most biopharma companies are not built for what’s coming.
Rohit Gupta, Partner at Beghou, breaks down why the real constraint has moved inside the building, and the shifts in capital timing, cross-functional planning, and leadership needed to close the gap.