Over the past month, updates in the cell and gene therapy sector were defined by the release of longer-term efficacy data alongside continued regulatory scrutiny of safety. Sarepta reported 3-year controlled Phase 3 functional outcomes for ELEVIDYS, while Ultragenyx extended UX111 follow-up to 8. 5 years, reinforcing the industry’s pivot toward demonstrating durability with clinically meaningful endpoints rather than short-term biomarker wins alone. Regulatory actions underscored that durability must be matched by confidence in safety surveillance: the US FDA lifted the hold on Intellia’s MAGNITUDE-2 with enhanced liver monitoring but placed Regenxbio’s RGX-111 and RGX-121 on hold after a tumor finding in a long-dosed patient. In parallel, ‘getting therapies into systems’ showed up in tangible examples; Zemcelpro® secured Germany’s NUB Status 1 for interim reimbursement, and the financing and public-market moves by Orca Bio and Innovacell highlighted how late-stage developers are positioning for launch execution. Innovacell targeted 92M in Tokyo IPO to advance Phase 3 regenerative program; Market Trends.
Abigail Pinchbeck (Thu,) studied this question.