El CRISPR Atlas

Treatments

Named gene-editing and gene-therapy medicines, from approved products to programmes that have been discontinued.

15 treatments in the Atlas. Regulatory status is stated on every record. “Phase III” or “in trials” never means a treatment is proven.

15 mostrados

Tratamiento	Enfermedad	Tecnología	Administración	Fase
Casgevy (exagamglogene autotemcel) Vertex Pharmaceuticals / CRISPR Therapeutics	Sickle cell disease; beta thalassemia	CRISPR-Cas9	Ex vivo, single IV infusion	Approved
Lyfgenia (lovotibeglogene autotemcel) bluebird bio	Sickle cell disease	Lentiviral gene addition	Ex vivo, single IV infusion	Approved
Zynteglo (betibeglogene autotemcel) bluebird bio	Beta thalassemia	Lentiviral gene addition	—	Approved
Nexiguran ziclumeran (nex-z) Intellia Therapeutics	ATTR amyloidosis	CRISPR-Cas9	In vivo, lipid nanoparticle IV	Phase III
Lonvoguran ziclumeran (lonvo-z) Intellia Therapeutics	Hereditary angioedema	CRISPR-Cas9	In vivo, lipid nanoparticle IV	Phase III
Risto-cel (BEAM-101) Beam Therapeutics	Sickle cell disease	Base editing	Ex vivo	Phase III
BEAM-302 Beam Therapeutics	Alpha-1 antitrypsin deficiency	Base editing	In vivo, lipid nanoparticle IV	Phase III
VERVE-102 Eli Lilly	Familial hypercholesterolemia	Base editing	In vivo, lipid nanoparticle IV	Phase II
PM359 Prime Medicine	Chronic granulomatous disease	Prime editing	Ex vivo	Phase I/II
EDIT-401 Editas Medicine	Familial hypercholesterolemia	CRISPR-Cas9	In vivo	Preclinical
CTX112 CRISPR Therapeutics	Cancer	CRISPR-Cas9	Ex vivo, allogeneic	Phase I/II
CB-010 Caribou Biosciences	Cancer	CRISPR-Cas12a	Ex vivo, allogeneic	Phase I
EDIT-101 (Brilliance) Editas Medicine	Inherited blindness	CRISPR-Cas9	In vivo, subretinal AAV	Discontinued
Bespoke base editor for CPS1 deficiency (the KJ Muldoon case) Academic — CHOP and Penn	Rare genetic disease	Base editing	In vivo, lipid nanoparticle IV	Completed
Hypoimmune islet cell therapy Sana Biotechnology	Diabetes	CRISPR-Cas9	Ex vivo, allogeneic	Phase I

La fase indicada es la etapa de desarrollo más avanzada reportada públicamente para ese programa, a la fecha de cada registro. Un programa en Fase I, II o III no ha demostrado ser seguro ni eficaz.