Gene & Cell Therapy

The FDA approved Tregzi on June 30, 2026 — the first precision-engineered regulatory T cell therapy ever authorized. In a randomized Phase 3 trial in blood cancer patients undergoing allogeneic stem cell transplant, Tregzi achieved 78% one-year cGVHD-free survival versus 38% with standard of care, with 94% overall survival versus 83%. The data doesn't just improve on the existing standard — it reframes what the standard should be.

On June 9, 2026, a person received the world's first injection of a partial cellular reprogramming therapy — ER-100, built by Life Biosciences on David Sinclair's Harvard research. Using three Yamanaka factors to reset the epigenetic clock without erasing cell identity, the Phase 1 trial targets age-related vision loss. It is also the opening shot of an entirely new category of medicine.

The FDA expanded Casgevy, the first approved CRISPR/Cas9 gene therapy, to sickle cell and beta thalassemia patients as young as 2 years old. It's a real clinical win for early intervention — but three years post-launch, patient numbers remain in the hundreds, and the real bottleneck is treatment infrastructure, not the science.