Treatments
Most Advanced CRISPR Treatments
Every treatment in the Atlas ordered by how far it has progressed.
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The full treatment database sorted by development stage, from approved medicines down to programmes that have been discontinued.
| # | Voce | Categoria | Stato |
|---|---|---|---|
| 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 |
Cosa questa lista non ti diceStage is the most advanced publicly reported stage as of each record's last-updated date.