Treatments
Gene-Editing Treatments in Phase III
Programmes in registrational trials — the closest thing to a preview of what may be approved next.
How this list is built
Phase 3 is the last stage before a company can apply for approval. A treatment reaching it has already cleared meaningful hurdles — and Phase 3 trials still fail, sometimes for safety and sometimes because the benefit does not hold up at scale.
| # | Entry | Category | Status |
|---|---|---|---|
| 1 | Nexiguran ziclumeran (nex-z) The first CRISPR therapy ever administered into a human bloodstream — a single infusion… | ATTR amyloidosis | Phase III |
| 2 | Lonvoguran ziclumeran (lonvo-z) The first in vivo gene-editing therapy to report a positive Phase 3 result — a single… | Hereditary angioedema | Phase III |
| 3 | Risto-cel (BEAM-101) A base-editing therapy for sickle cell disease that mimics a naturally protective genetic… | Sickle cell disease | Phase III |
| 4 | BEAM-302 The first therapy to correct a disease-causing point mutation inside a living person — a… | Alpha-1 antitrypsin deficiency | Phase III |
What this list does not tell youBeing in Phase 3 is not evidence that a treatment works. It means the trial that will answer that question is under way.