The Readout Loud 391: Breaching the IBD efficacy ceiling, and sham surgeries
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Mar 5, 2026 Cameron Turtle, CEO of Spire Therapeutics and builder of long-acting biologics and combination IBD therapies, discusses strategies to surpass the IBD efficacy ceiling. He explains why co-formulated combinations and replacing older mechanisms could boost remission rates. The conversation also touches on regulatory debates around rare-disease approvals and sham-surgery ethics.
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UniQure Conflict Over Sham Surgeries In Huntington Study
- UniQure's Huntington's gene therapy dispute highlights tension over sham surgeries and control design.
- Adam explains UniQure used historical controls; FDA demands a new sham‑surgery controlled trial despite risks and community pushback.
Regulatory Philosophy Shifts Are Reshaping Rare Disease Approvals
- FDA leadership swings change regulatory philosophy and patient expectations.
- Adam contrasts Peter Marks' permissive stance with Vinay Prasad's conservative approach, leaving rare‑disease communities whipsawed between flexibility and rigor.
Plausible Mechanism Pathway Enables Small‑N Gene Edits
- FDA's plausible mechanism pathway enables novel gene editing approvals even with very small patient data sets.
- Prime Medicine plans to seek approval after treating two patients using prime editing for CGD under this pathway.