1️⃣ Tenaya put PKP2 gene therapy into the clinic Tenaya said TN-401 cut daily PVC burden by a mean 64% across six adults with PKP2-associated ARVC, with biopsy evidence of cardiac transduction and EMA PRIME status.
💡 Why it matters This is still N=6, but cardiac gene therapy needs human tissue activity and rhythm signal. Tenaya now has both, early.
☕ Coffee talk If six patients can move the room, what does the next cohort need to show before this stops being a curiosity?
2️⃣ Aardvark lost control of the PWS clock FDA placed a full clinical hold on Aardvark’s ARD-101 IND, covering the Phase 3 HERO and OLE trials, and the company will unblind accumulated data before deciding next steps.
💡 Why it matters The program moved from registrational execution to salvage analysis. In Prader-Willi, that changes both timing and optionality.
☕ Coffee talk How much is a Phase 3 asset worth when the next readout is forced by a hold, not designed by the sponsor?
3️⃣ CG Oncology added a bladder-cancer combo signal CG Oncology reported first CORE-008 Cohort CX data: 89.5% high-grade event-free survival at six months and 85.7% complete response in CIS-containing patients on cretostimogene plus gemcitabine.
💡 Why it matters Cretostimogene is already a late-stage bladder-sparing story. A tolerable intravesical combo could widen the commercial frame beyond monotherapy.
☕ Coffee talk If urologists can add gemcitabine without much extra toxicity, how narrow does the monotherapy label really stay?