Treatments
Most Advanced CRISPR Treatments
Every treatment in the Atlas ordered by how far it has progressed.
Comment cette liste est construite
The full treatment database sorted by development stage, from approved medicines down to programmes that have been discontinued.
| # | Entrée | Catégorie | Statut |
|---|---|---|---|
| 1 | Casgevy (exagamglogene autotemcel) The first CRISPR-based medicine approved anywhere in the world — a one-time therapy made… | Sickle cell disease; beta thalassemia | Approved |
| 2 | Lyfgenia (lovotibeglogene autotemcel) Approved on the same day as Casgevy for sickle cell disease — but a gene-addition… | Sickle cell disease | Approved |
| 3 | Zynteglo (betibeglogene autotemcel) An approved gene-addition therapy for transfusion-dependent beta thalassemia — the… | Beta thalassemia | Approved |
| 4 | Nexiguran ziclumeran (nex-z) The first CRISPR therapy ever administered into a human bloodstream — a single infusion… | ATTR amyloidosis | Phase III |
| 5 | Lonvoguran ziclumeran (lonvo-z) The first in vivo gene-editing therapy to report a positive Phase 3 result — a single… | Hereditary angioedema | Phase III |
| 6 | Risto-cel (BEAM-101) A base-editing therapy for sickle cell disease that mimics a naturally protective genetic… | Sickle cell disease | Phase III |
| 7 | BEAM-302 The first therapy to correct a disease-causing point mutation inside a living person — a… | Alpha-1 antitrypsin deficiency | Phase III |
| 8 | VERVE-102 A one-time base-editing infusion intended to lower LDL cholesterol permanently by… | Familial hypercholesterolemia | Phase II |
| 9 | PM359 The first prime-editing therapy given to human patients, with published evidence that it… | Chronic granulomatous disease | Phase I/II |
| 10 | EDIT-401 Editas Medicine's lead in vivo candidate after abandoning its cell-therapy programmes —… | Familial hypercholesterolemia | Preclinical |
| 11 | CTX112 An allogeneic CD19 CAR-T candidate with multiple CRISPR edits intended to make one… | Cancer | Phase I/II |
| 12 | CB-010 An allogeneic CAR-T candidate built with hybrid RNA-DNA guides rather than conventional… | Cancer | Phase I |
| 13 | EDIT-101 (Brilliance) The first CRISPR therapy injected directly into a human organ — scientifically… | Inherited blindness | Discontinued |
| 14 | Bespoke base editor for CPS1 deficiency (the KJ Muldoon case) A gene-editing medicine designed, manufactured and administered for a single infant in… | Rare genetic disease | Completed |
| 15 | Hypoimmune islet cell therapy Transplanted insulin-producing cells edited to be invisible to the immune system, so they… | Diabetes | Phase I |
Ce que cette liste ne vous dit pasStage is the most advanced publicly reported stage as of each record's last-updated date.