CASGEVYexa-cel / CASGEVY / BCL11A erythroid-specific enhancer / Non-viral ex vivo CRISPR/Cas9 gene-edited autologous cell therapy / Hemoglobinopathies Sickle cell disease, ages 5-11Regulatory submissions expected H1 2026Active pediatric expansionGlobalTransfusion-dependent beta thalassemia, ages 5-11Regulatory submissions expected H1 2026Active pediatric expansionGlobal
CTX310CTX310 / ANGPTL3 / In vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular/metabolic Severe hypertriglyceridemiaPhase 1bActive; update expected H2 2026Refractory hypercholesterolemiaPhase 1bActive; update expected H2 2026Heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias or severe hypertriglyceridemiaPhase 1Developed/being evaluated
CTX320CTX320 / LPA / In vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular Elevated lipoprotein(a)Clinical trial / dose escalationActive; Lp(a) program update expected in 2026
CTX321CTX321 / LPA / Next-generation in vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular Elevated lipoprotein(a)IND/CTA-enablingActive
CTX460CTX460 / SERPINA1 / In vivo SyNTase gene correction; LNP delivery / Rare disease Alpha-1 antitrypsin deficiencyPreclinical; clinical trial expected mid-2026Active
CTX340CTX340 / AGT / In vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular Refractory hypertensionIND/CTA-enabling; clinical trial expected H1 2026Active
CTX611SRSD107 / Factor XI / FXI mRNA / Long-acting double-stranded siRNA / Thromboembolic/cardiovascular diseases Venous thromboembolism prevention after total knee arthroplastyPhase 2Ongoing; top-line data expected H2 2026EuropeAtrial fibrillation, venous thromboembolism, ischemic stroke, cancer-associated thrombosis, chronic kidney disease, peripheral vascular disease, chronic coronary artery diseaseTarget indications disclosed
Zugocabtagene geleucelzugo-cel; formerly CTX112 / CD19 / Allogeneic CAR-T cell therapy; CRISPR Cas9 engineered / Autoimmune disease; oncology Systemic lupus erythematosusPhase 1Ongoing; update expected H2 2026Systemic sclerosisPhase 1Ongoing; update expected H2 2026Inflammatory myositisPhase 1Ongoing; update expected H2 2026
CTX213CTX213 / iPSC-derived allogeneic gene-edited beta islet cell precursor therapy / Regenerative medicine / diabetes Type 1 diabetesPreclinical; progressing toward clinicActive