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Drugs in development · updated 30 October 2026

CTX001: clinical trials and development status

Other names and codes: exagamglogene autotemcel

NLM verified identityApproved in the US (FDA)Authorised in the EU (EMA) since 9 February 2024

As of 30 October 2026 CTX001 is in 7 active clinical trials (recruiting or ongoing, phases I–III) for 9 diseases. The most advanced phase is III.

7Active trials
IIIMost advanced phase
9Diseases studied

Summary updated every week · data as of 30 October 2026. In Phuturdex you can follow CTX001 and get an email at every change or safety alert.

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Diseases studied

For each disease: the drug’s most advanced phase and active trials, with the reference trial on ClinicalTrials.gov.

DiseaseMost advanced phaseActive trialsReference trial
Hematologic DiseasesIII4NCT04208529
HemoglobinopathiesIII4NCT04208529
Beta thalassemiaIII3NCT04208529
Genetic diseaseIII3NCT04208529
Sickle-cell diseaseIII3NCT04208529
ThalassemiaIII3NCT04208529
AnemiaI2NCT07901933
Chronic renal insufficiencyI2NCT07901933
Iron DeficienciesEarly I1NCT07577817

Everything on CTX001

In Phuturdex you get every trial with sponsors, countries and dates, development events, FDA and EMA approvals, patents and exclusivities, orphan designations and safety alerts, with PDF and Excel export. Free until 31 March 2027.

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Source: Phuturdex analysis of ClinicalTrials.gov (U.S. National Library of Medicine), FDA and EMA data, updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III. Diseases are those named by each trial (NLM MeSH terms). The estimated launch, when shown, is a Phuturdex calculation described on the Method page, not official data.

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