{"meta":{"source":"The CRISPR Atlas","count":14,"note":"Trial records are compiled from public registry entries and sponsor announcements; confirm current status on ClinicalTrials.gov."},"data":[{"slug":"climb-121","name":"A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease","short_title":"CLIMB SCD-121 — the trial that led to the first CRISPR medicine approval","status":"Completed","nct":"NCT03745287","gene":"BCL11A enhancer","phase":"Phase II/III","start":"2018","design":"Open-label, single-arm, multi-site","primary":"Proportion free of severe vaso-occlusive crises for at least 12 consecutive months","sponsor":"Vertex Pharmaceuticals","partners":"CRISPR Therapeutics","countries":["United States","United Kingdom","Canada","France","Germany","Italy","Belgium"],"enrolment":"Approximately 63 participants","secondary":"Proportion free of inpatient hospitalisation for vaso-occlusive crises; haemoglobin measures; safety","treatment":"Casgevy (exagamglogene autotemcel)","completion":"Long-term follow-up continuing","technology":"CRISPR-Cas9","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/climb-121"},{"slug":"climb-111","name":"A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia","short_title":"CLIMB THAL-111 — the beta thalassemia trial behind the Casgevy approval","status":"Completed","nct":"NCT03655678","gene":"BCL11A enhancer","phase":"Phase II/III","start":"2018","design":"Open-label, single-arm, multi-site","primary":"Transfusion independence for at least 12 consecutive months","sponsor":"Vertex Pharmaceuticals","partners":"CRISPR Therapeutics","countries":["United States","United Kingdom","Canada","France","Germany","Italy"],"enrolment":"Approximately 52 participants","treatment":"Casgevy (exagamglogene autotemcel)","completion":"Long-term follow-up continuing","technology":"CRISPR-Cas9","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/climb-111"},{"slug":"magnitude","name":"A Phase 3 Study of Nexiguran Ziclumeran in Participants With Transthyretin Amyloidosis With Cardiomyopathy","short_title":"MAGNITUDE — Phase 3 in vivo CRISPR for ATTR cardiomyopathy","status":"Active, not recruiting","nct":"NCT06128629","gene":"TTR","phase":"Phase III","start":"2023","design":"Randomised, double-blind, placebo-controlled","primary":"Composite of all-cause mortality and recurrent cardiovascular events","sponsor":"Intellia Therapeutics","countries":["United States","United Kingdom","Germany","Spain","Japan","Brazil","Australia"],"enrolment":"Approximately 765 participants planned","treatment":"Nexiguran ziclumeran (nex-z)","completion":"Estimated 2028","technology":"CRISPR-Cas9 (in vivo)","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/magnitude"},{"slug":"magnitude-2","name":"A Phase 3 Study of Nexiguran Ziclumeran in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy","short_title":"MAGNITUDE-2 — Phase 3 in vivo CRISPR for ATTR polyneuropathy","status":"Paused","nct":"NCT06672237","gene":"TTR","phase":"Phase III","start":"2024","design":"Randomised, controlled","primary":"Change in neuropathy impairment score","sponsor":"Intellia Therapeutics","countries":["United States","Europe","Japan"],"treatment":"Nexiguran ziclumeran (nex-z)","technology":"CRISPR-Cas9 (in vivo)","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/magnitude-2"},{"slug":"haelo","name":"A Phase 3 Study of Lonvoguran Ziclumeran in Adults With Hereditary Angioedema","short_title":"HAELO — the first positive Phase 3 result for in vivo gene editing","status":"Completed","nct":"NCT06634420","gene":"KLKB1","phase":"Phase III","start":"2024","design":"Randomised, double-blind, placebo-controlled","primary":"Number of angioedema attacks per month","sponsor":"Intellia Therapeutics","countries":["United States","United Kingdom","Germany","Israel","Japan"],"enrolment":"Approximately 60 participants","treatment":"Lonvoguran ziclumeran (lonvo-z)","completion":"2026","technology":"CRISPR-Cas9 (in vivo)","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/haelo"},{"slug":"beacon","name":"A Study of BEAM-101 in Patients With Sickle Cell Disease With Severe Vaso-Occlusive Crises","short_title":"BEACON — base editing for sickle cell disease","status":"Active, not recruiting","nct":"NCT05456880","gene":"HBG1/HBG2","phase":"Phase I/II","start":"2022","design":"Open-label, single-arm","primary":"Safety and tolerability; fetal haemoglobin induction","sponsor":"Beam Therapeutics","countries":["United States"],"treatment":"Risto-cel (BEAM-101)","technology":"Base editing","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/beacon"},{"slug":"beam-302-trial","name":"A Study of BEAM-302 in Patients With Alpha-1 Antitrypsin Deficiency","short_title":"BEAM-302 — first in vivo correction of a disease-causing point mutation in humans","status":"Recruiting","nct":"NCT06389474","gene":"SERPINA1","phase":"Phase I/II","start":"2024","design":"Open-label, dose escalation, then pivotal cohort","primary":"Safety and tolerability; change in total and functional alpha-1 antitrypsin","sponsor":"Beam Therapeutics","countries":["United Kingdom","United States","Australia","Netherlands"],"treatment":"BEAM-302","technology":"Base editing (in vivo)","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/beam-302-trial"},{"slug":"pm359-trial","name":"A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease","short_title":"The first prime-editing therapy in humans","status":"Recruiting","nct":"NCT06559176","gene":"NCF1","phase":"Phase I/II","start":"2024","design":"Open-label, single-arm","primary":"Safety; proportion of dihydrorhodamine-positive neutrophils","sponsor":"Prime Medicine","countries":["United States","Australia"],"treatment":"PM359","technology":"Prime editing","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/pm359-trial"},{"slug":"brilliance","name":"Single Ascending Dose Study of EDIT-101 in Participants With Leber Congenital Amaurosis Type 10","short_title":"BRILLIANCE — the first in vivo CRISPR administration to a human organ","status":"Completed","nct":"NCT03872479","gene":"CEP290","phase":"Phase I/II","start":"2019","design":"Open-label, single ascending dose","primary":"Safety and tolerability","sponsor":"Editas Medicine","countries":["United States"],"enrolment":"14 participants","treatment":"EDIT-101","completion":"2023","technology":"CRISPR-Cas9 (AAV, subretinal)","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/brilliance"},{"slug":"ntla-2002-ph1","name":"A Study of NTLA-2002 in Adults With Hereditary Angioedema","short_title":"The Phase 1/2 study that took lonvo-z into late-stage development","status":"Completed","nct":"NCT05120830","gene":"KLKB1","phase":"Phase I/II","start":"2021","design":"Open-label then randomised","primary":"Safety; plasma kallikrein reduction; attack rate","sponsor":"Intellia Therapeutics","countries":["United Kingdom","New Zealand","Netherlands"],"treatment":"Lonvoguran ziclumeran (NTLA-2002)","technology":"CRISPR-Cas9 (in vivo)","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/ntla-2002-ph1"},{"slug":"antler","name":"A Study of CB-010 in Subjects With Relapsed or Refractory B Cell Non-Hodgkin Lymphoma","short_title":"ANTLER — allogeneic CAR-T built with hybrid RNA-DNA guides","status":"Recruiting","nct":"NCT04637763","phase":"Phase I","start":"2021","design":"Open-label, dose escalation and expansion","primary":"Safety and tolerability; overall response rate","sponsor":"Caribou Biosciences","countries":["United States"],"treatment":"CB-010","technology":"CRISPR-Cas12a (chRDNA)","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/antler"},{"slug":"ctx112-trial","name":"A Study of CTX112 in Subjects With Relapsed or Refractory B-Cell Malignancies","short_title":"Allogeneic CAR-T with next-generation potency edits","status":"Recruiting","nct":"NCT05643742","phase":"Phase I/II","start":"2023","design":"Open-label, dose escalation","primary":"Safety; overall response rate","sponsor":"CRISPR Therapeutics","countries":["United States","Australia"],"treatment":"CTX112","technology":"CRISPR-Cas9 multiplex","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/ctx112-trial"},{"slug":"upenn-nyces","name":"NY-ESO-1-Redirected CRISPR-Edited T Cells in Patients With Multiple Myeloma and Sarcoma","short_title":"The first US trial of CRISPR-edited T cells in cancer patients","status":"Completed","nct":"NCT03399448","phase":"Phase I","start":"2018","design":"Open-label, single-arm","primary":"Safety and feasibility","sponsor":"University of Pennsylvania","countries":["United States"],"enrolment":"3 participants treated","completion":"2020","technology":"CRISPR-Cas9 multiplex","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/upenn-nyces"},{"slug":"sangamo-sb728","name":"Zinc Finger Nuclease Modification of CCR5 in Autologous CD4 T Cells in HIV","short_title":"The first human gene-editing clinical trial","status":"Completed","nct":"NCT00842634","gene":"CCR5","phase":"Phase I","start":"2009","design":"Open-label","primary":"Safety and tolerability","sponsor":"Sangamo Therapeutics","countries":["United States"],"completion":"2014","technology":"Zinc finger nucleases","url":"https://crispr2.2.25.209.181.nip.io/clinical-trials/sangamo-sb728"}]}