1️⃣ Regenxbio put DMD back in play Regenxbio said RGX-202 hit the pivotal AFFINITY DUCHENNE primary endpoint, with 93% of patients above 10% microdystrophin expression and a potential 2027 accelerated approval path.
💡 Why it matters After the Elevidys safety overhang, DMD gene therapy needed a cleaner competitive signal. Regenxbio now has one, with function correlation attached.
☕ Coffee talk If FDA accepts this surrogate package, how much of Sarepta’s DMD moat is still real?
2️⃣ Biogen kept tau alive after a miss Biogen and Ionis’ diranersen missed the Phase 2 CELIA primary dose-response endpoint, but tau lowering and cognitive signals were enough for Biogen to move toward registrational development.
💡 Why it matters Alzheimer’s tau remains investable, but the bar is awkward: biology moved, the primary endpoint did not. Phase 3 will carry a lot of mechanism risk.
☕ Coffee talk How many investors pay for tau conviction when the cleanest line in the study is still not the primary one?
3️⃣ CREATE raised for in vivo CAR CREATE Medicines raised $122 million from Newpath, ARCH, Hatteras and others to push RNA-based in vivo CAR-T and multi-immune programming candidates into human studies.
💡 Why it matters The in vivo CAR field is moving from clever platform story to financed clinical race. Fresh money is starting to sort real programs from slideware.
☕ Coffee talk After Kyverna, does every in vivo CAR deck now need an autoimmune plan as well as an oncology one?