In Vivo CAR-T for CNS Autoimmune Disease Looks Promising. The Trial Design Problem Is Hiding in Plain Sight.
NEJM's lentiviral in vivo CAR-T data in neurologic autoimmune disease is compelling, but the trial infrastructure to validate it...
CAR-T Cell Therapy
iPSC Cardiomyocytes Reach the Beating Heart: What This Trial Design Tells Sponsors About the Next Frontier of Cell Therapy Protocols
A Nature Medicine RCT of allogeneic iPSC-derived cardiomyocytes in advanced ischemic heart failure just set the protocol bar for...
RiboX’s Circular RNA CAR-T IND Clearance Forces the Industry to Rethink Everything It Knows About Cell Therapy Trial Design
FDA cleared RiboX's RXIM002 IND for the first circular RNA-based in vivo CAR-T therapy — here's what it means...
The 2-Week Window: What a Phase 1 iPSC Trial Teaches Us About Designing Cell Therapy Studies That Can Actually Win Approval
A Nature Medicine phase 1 iPSC neural progenitor trial in subacute spinal cord injury shows how to build the...
The Pediatric CNS Immunotherapy Signal That Sponsors Are Underreading
ReMIND's multi-antigen T cell data in 25 pediatric CNS patients points to a regulatory inflection point sponsors haven't mapped...