There is significant opportunity to expand the applications of cell therapy further into rare diseases as well as to tackle widespread conditions.
Tuesday, January 27, 2026
Sponsors with a licensed gene therapy can use the same vector platform to reference manufacturing, analytical and safety data for other candidates.
Tuesday, January 20, 2026
A powerful shift is underway within the rare disease space. It started as a handful of inspiring stories of parents stopping at nothing to save the lives of their children. But while this patient advocacy-driven movement is compelling — it shouldn’t have to exist.
Tuesday, January 13, 2026
Characterizing 2025 from a regulatory perspective is uniquely challenging. Perhaps David Bowie said it best when he said, “I don’t know where I’m going from here, but I promise it won’t be boring.”
Viral clearance studies, long a cornerstone of biologics production, are now being reevaluated and adapted for genetically engineered viral vectors and vector-derived products.
Wednesday, October 15, 2025
Because CGTs are designed to have lasting and lifelong effects, regulatory bodies often mandate 15-year, long-term follow-up. Fortunately, collaborative groups are creating resources and recommendations for these emerging studies.
Wednesday, September 17, 2025
A robust CMC strategy cannot be retrofitted; it should guide decisions from the very beginning. For developers aiming to bring iPSC-based therapies to the commercial market, the starting point is not the laboratory bench, it is the strategic plan.
Wednesday, September 10, 2025
Biopharma companies advancing cell therapies must confront a range of key considerations and risks. A strategic, collaborative approach can accelerate development and ensure patients reliably access safe and effective cell therapy products.
Wednesday, September 03, 2025
The emergence of nonviral vectors represents an opportunity to propel in vivo gene therapy further into commercial adoption and expand its impact globally. LNPs are poised to become the first commercially approved nonviral vectors for in vivo transgene delivery and genome editing approaches.
Wednesday, August 27, 2025
Given the undeniably novel aspect of this new paradigm, however, conducting CAR-T clinical trials presents a unique set of obstacles for sponsors, sites and regulators to carefully navigate. Moreover, the complexity of manufacturing, delivering and managing genetically modified cells has made CAR-T ...
Wednesday, August 20, 2025