drugset
Drugs / Exa-cel
last change Nov 2023 re-read 3 minutes ago

Exa-cel

CRISPR / gene editing

Developed for
sickle cell disease
Investigated by
Vertex Pharmaceuticals Incorporated

Trials 1

today NCT05951205 · Ph 3
2028202920302031203220332034
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 3 NCT05951205 Jul 2027 → Dec 2033 sickle cell disease Vertex Pharmaceuticals Incorporated Withdrawn No outcome recorded

Press releases naming this drug 6 releases

DateIssuerRelease
2023-11-02 Vertex Pharmaceuticals Incorporated Vertex to Present New Data on Exagamglogene Autotemcel (exa-cel) at the American Society of Hematology (ASH) Annual Meeting and Exposition vrtx.com ↗
2023-06-09 Vertex Pharmaceuticals Incorporated Positive Results From Pivotal Trials of exa-cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease Presented at the 2023 Annual European Hematology Association (EHA) Congress vrtx.com ↗
2023-06-08 Vertex Pharmaceuticals Incorporated FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia vrtx.com ↗
2023-04-03 Vertex Pharmaceuticals Incorporated Vertex and CRISPR Therapeutics Complete Submission of Rolling Biologics License Applications (BLAs) to the US FDA for exa-cel for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia vrtx.com ↗
2022-09-27 Vertex Pharmaceuticals Incorporated Vertex and CRISPR Therapeutics Announce Global exa-cel Regulatory Submissions for Sickle Cell Disease and Beta Thalassemia in 2022 vrtx.com ↗
2022-06-11 Vertex Pharmaceuticals Incorporated Vertex and CRISPR Therapeutics Present New Data on More Patients With Longer Follow-Up Treated With exagamglogene autotemcel (exa-cel) at the 2022 European Hematology Association (EHA) Congress vrtx.com ↗

Evidence & citations 3 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as Exa-cel ClinicalTrials.gov intervention name — accepted as the source's own label NCT05951205 ↗
Modality CRISPR / gene editing “modified with CRISPR-Cas9” NCT05951205 ↗
Route Intravenous “Administered by intravenous (IV) infusion” NCT05951205 ↗