Drugs / EXAGAMGLOGENE AUTOTEMCEL
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed beta-thalassemia intermedia 1 milestone | |||||
| Filed | US (FDA) | — | beta-thalassemia intermedia | — | “Regulatory submissions complete for exagamglogene autotemcel (exa-cel), formerly known as CTX001™, in the U.S. for transfusion-dependent beta thalassemia (TDT) and severe...” crisprtx.com ↗ |
| Filed sickle cell S-Lepore disease 2 milestones | |||||
| Clinical hold | US (FDA) | — | sickle cell S-Lepore disease | 2018-05-30 | “the U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Investigational New Drug Application (IND) for CTX001 for the treatment of sickle cell disease” crisprtx.com ↗ “clinical hold on the Investigational New Drug Application (IND) for CTX001 for the treatment of sickle cell disease” vrtx.com ↗ |
| Filed | US (FDA) | — | sickle cell S-Lepore disease | — | “Regulatory submissions complete for exagamglogene autotemcel (exa-cel), formerly known as CTX001™, in the U.S. for transfusion-dependent beta thalassemia (TDT) and severe...” crisprtx.com ↗ |
| Filed Indication not stated 2 milestones | |||||
| Filed | EU (EMA) | — | — | — | “completed regulatory submissions for exa-cel, formerly known as CTX001™ , with the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA)” crisprtx.com ↗ |
| Filed | UK (MHRA) | — | — | — | “completed regulatory submissions for exa-cel, formerly known as CTX001™ , with the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA)” crisprtx.com ↗ |
Trials 7
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT07577817 | Jun → Oct 2026 expected | anemia, chronic kidney disease, disorder of iron metabolism and transport | Cajal Therapeutics Inc. | Recruiting | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT07392255 | Dec 2025 → May 2028 expected | age-related macular degeneration | Complement Therapeutics | Recruiting | No outcome recorded |
| Phase 2/31 trial · 1 met primary | ||||||
| Phase 2/3 | NCT03655678 | Sep 2018 → Nov 2025 | beta thalassemia | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 34 trials | ||||||
| Phase 3 | NCT05477563 | Aug 2022 → Jun 2027 expected | beta thalassemia, sickle cell disease | Vertex Pharmaceuticals Incorporated | Recruiting | No outcome recorded |
| Phase 3 | NCT05356195 | May 2022 → Nov 2027 expected | beta thalassemia | Vertex Pharmaceuticals Incorporated | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05329649 | May 2022 → Jun 2027 expected | sickle cell disease | Vertex Pharmaceuticals Incorporated | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04208529 | Jan 2021 → Sep 2039 expected | beta thalassemia, sickle cell disease | Vertex Pharmaceuticals Incorporated | Enrolling by invitation | No outcome recorded |
News releases announcing trial results or a regulatory action · 47
| Date | Issuer | Release |
|---|---|---|
| 2024-06-14 | Vertex Pharmaceuticals Incorporated | Results Vertex Presents Positive Long-Term Data On CASGEVY™ (exagamglogene autotemcel) at the 2024 Annual European Hematology Association (EHA) Congress vrtx.com ↗
The results, presented at the annual European Hematology Association (EHA) Congress, confirm the transformative, consistent and durable clinical benefits of CASGEVY over time. |
| 2024-02-13 | Vertex Pharmaceuticals Incorporated | Regulatory European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy. |
| 2024-02-13 | CRISPR Therapeutics | Regulatory European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) crisprtx.com ↗
CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene edited therapy. |
| 2024-01-16 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia vrtx.com ↗ |
| 2024-01-16 | CRISPR Therapeutics | Regulatory CRISPR Therapeutics Announces U.S. Food and Drug Administration (FDA) Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia crisprtx.com ↗
CRISPR Therapeutics Announces U.S. Food and Drug Administration (FDA) Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia |
| 2024-01-09 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces Approval of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™, for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) in Kingdom of Saudi Arabia vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the Saudi Food and Drug Authority (SFDA) granted Marketing Authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). |
| 2023-12-15 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives CHMP Positive Opinion for the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia vrtx.com ↗ |
| 2023-12-11 | Vertex Pharmaceuticals Incorporated | Results Positive Results from Pivotal Trials of CASGEVY™ (exagamglogene autotemcel) Highlighted in Oral Presentations at the American Society of Hematology (ASH) Annual Meeting and Exposition vrtx.com ↗
Data from 96 patients (44 sickle cell disease [SCD], 52 transfusion-dependent beta thalassemia [TDT]) treated with CASGEVY in pivotal studies, with the longest follow-up of more than four years, continue to reinforce the consistent and durable response to treatment. |
| 2023-12-08 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex and CRISPR Therapeutics Announce US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). |
| 2023-12-08 | CRISPR Therapeutics | Regulatory Vertex and CRISPR Therapeutics Announce US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease crisprtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). |
| 2023-11-16 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia vrtx.com ↗
CASGEVY has been authorized for the treatment of eligible patients 12 years of age and older with SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available. |
| 2023-11-16 | CRISPR Therapeutics | Regulatory Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia crisprtx.com ↗
CASGEVY has been authorized for the treatment of eligible patients 12 years of age and older with SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available. |
| 2023-10-31 | CRISPR Therapeutics | Regulatory CRISPR Therapeutics Trading Halted Today; FDA Advisory Committee to Review Biologics License Application (BLA) for Exagamglogene Autotemcel (exa-cel) in Sickle Cell Disease (SCD) crisprtx.com ↗
The U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee is meeting today to review the Biologics License Application (BLA) for exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease (SCD) in people ages 12 and older with recurrent vaso-occlusive crises. |
| 2023-08-01 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Reports Second Quarter 2023 Financial Results vrtx.com ↗
FDA has accepted the exa-cel BLAs in both severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT); Priority Review granted for SCD with PDUFA date of December 8, 2023 |
| 2023-06-09 | Vertex Pharmaceuticals Incorporated | Results 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 ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that both pivotal trials for exagamglogene autotemcel (exa-cel) in patients with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD) met primary and key secondary endpoints at pre-specified interim analyses. |
| 2023-06-09 | CRISPR Therapeutics | Results 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 crisprtx.com ↗ |
| 2023-06-08 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia vrtx.com ↗ |
| 2023-06-08 | CRISPR Therapeutics | Regulatory FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia crisprtx.com ↗ |
| 2023-04-03 | Vertex Pharmaceuticals Incorporated | Regulatory 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 ↗
The BLAs include requests for Priority Review, which if granted, would shorten the FDA’s review of the application to eight months from the time of submission versus a standard review timeline of 12 months. |
| 2023-04-03 | CRISPR Therapeutics | Regulatory CRISPR Therapeutics and Vertex 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 crisprtx.com ↗
CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the completion of the rolling Biologics License Applications (BLAs) to the U.S. Food and Drug Administration (FDA) for the investigational treatment exagamglogene autotemcel (exa-cel) for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). |
| 2022-09-27 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex and CRISPR Therapeutics Announce Global exa-cel Regulatory Submissions for Sickle Cell Disease and Beta Thalassemia in 2022 vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that Vertex has concluded discussions with the U.S. Food and Drug Administration (FDA), and the FDA granted exagamglogene autotemcel (exa-cel) a rolling review for the potential treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). |
| 2022-09-27 | CRISPR Therapeutics | Regulatory CRISPR Therapeutics and Vertex Announce Global exa-cel Regulatory Submissions for Sickle Cell Disease and Beta Thalassemia in 2022 crisprtx.com ↗
the FDA granted exagamglogene autotemcel (exa-cel) a rolling review for the potential treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). |
| 2022-06-11 | Vertex Pharmaceuticals Incorporated | Results 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 ↗
Forty-two of 44 patients with TDT were transfusion-free with follow-up ranging from 1.2 to 37.2 months after exa-cel infusion. |
| 2022-06-11 | CRISPR Therapeutics | Results 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 crisprtx.com ↗
Forty-two of 44 patients with TDT were transfusion-free with follow-up ranging from 1.2 to 37.2 months after exa-cel infusion. |
| 2021-06-11 | Vertex Pharmaceuticals Incorporated | Results Vertex and CRISPR Therapeutics Present New Data in 22 Patients With Greater Than 3 Months Follow-Up Post-Treatment With Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at European Hematology Association Annual Meeting vrtx.com ↗
All 15 patients with TDT, including six who have the beta zero/beta zero or other severe genotypes, were transfusion-free at last follow-up, and all seven patients with severe SCD were free of vaso-occlusive crises (VOCs) from CTX001 infusion through last follow-up. |
| 2021-06-11 | CRISPR Therapeutics | Results Vertex and CRISPR Therapeutics Present New Data in 22 Patients With Greater Than 3 Months Follow-Up Post-Treatment With Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at European Hematology Association Annual Meeting crisprtx.com ↗
All 15 patients with TDT, including six who have the beta zero /beta zero or other severe genotypes, were transfusion-free at last follow-up |
| 2021-04-26 | CRISPR Therapeutics | Regulatory Vertex and CRISPR Therapeutics Announce Priority Medicines (PRIME) Designation Granted by the European Medicines Agency to CTX001™ for Transfusion-Dependent Beta Thalassemia crisprtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of transfusion-dependent beta thalassemia (TDT). |
| 2020-12-05 | Vertex Pharmaceuticals Incorporated | Results CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at American Society of Hematology Annual Meeting and Exposition, Together With Publication in the New England Journal of Medicine vrtx.com ↗
today announced new data on a total of 10 patients treated with the investigational CRISPR/Cas9-based gene-editing therapy, CTX001, that show a consistent and sustained response to treatment. |
| 2020-12-05 | CRISPR Therapeutics | Results CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at American Society of Hematology Annual Meeting and Exposition, Together With Publication in the New England Journal of Medicine crisprtx.com ↗
CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new data on a total of 10 patients treated with the investigational CRISPR/Cas9-based gene-editing therapy, CTX001, that show a consistent and sustained response to treatment. |
| 2020-09-22 | Vertex Pharmaceuticals Incorporated | Regulatory CRISPR Therapeutics and Vertex Pharmaceuticals Announce Priority Medicines (PRIME) Designation Granted by the European Medicines Agency (EMA) to CTX001™ for the Treatment of Sickle Cell Disease vrtx.com ↗
CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD). |
| 2020-09-22 | CRISPR Therapeutics | Regulatory CRISPR Therapeutics and Vertex Pharmaceuticals Announce Priority Medicines (PRIME) Designation Granted by the European Medicines Agency (EMA) to CTX001™ for the Treatment of Sickle Cell Disease crisprtx.com ↗
CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Inco r porated (Nasdaq: VRTX) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD). |
| 2020-06-12 | Vertex Pharmaceuticals Incorporated | Results CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001™ in Severe Hemoglobinopathies at the 25th Annual European Hematology Association (EHA) Congress vrtx.com ↗
Two patients are transfusion independent at 5 and 15 months after CTX001 infusion; data demonstrate clinical proof-of-concept for CTX001 in transfusion-dependent beta thalassemia |
| 2020-06-12 | CRISPR Therapeutics | Results CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001™ in Severe Hemoglobinopathies at the 25th Annual European Hematology Association (EHA) Congress crisprtx.com ↗
Data presented today at EHA demonstrate clinical proof-of-concept for CTX001 in TDT. |
| 2020-05-11 | Vertex Pharmaceuticals Incorporated | Regulatory CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001™ for the Treatment of Severe Hemoglobinopathies vrtx.com ↗
ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 11, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). |
| 2020-05-11 | CRISPR Therapeutics | Regulatory CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001™ for the Treatment of Severe Hemoglobinopathies crisprtx.com ↗
“RMAT designation is another important regulatory milestone for CTX001 and underscores the transformative potential of a CRISPR-based therapy for patients with severe hemoglobinopathies,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. |
| 2019-11-19 | Vertex Pharmaceuticals Incorporated | Results CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data From First Two Patients Treated With Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001® for Severe Hemoglobinopathies vrtx.com ↗
CRISPR Therapeutics (NASDAQ: CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. |
| 2019-11-19 | CRISPR Therapeutics | Results CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data From First Two Patients Treated With Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001® for Severe Hemoglobinopathies crisprtx.com ↗
CRISPR Therapeutics (NASDAQ: CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. |
| 2019-04-16 | Vertex Pharmaceuticals Incorporated | Regulatory CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Beta Thalassemia vrtx.com ↗
ZUG, Switzerland and CAMBRIDGE and BOSTON, Mass. – April 16, 2019 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the treatment of transfusion-dependent beta thalassemia (TDT). |
| 2019-04-16 | CRISPR Therapeutics | Regulatory CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Beta Thalassemia crisprtx.com ↗
CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the treatment of transfusion-dependent beta thalassemia (TDT). |
| 2019-01-04 | Vertex Pharmaceuticals Incorporated | Regulatory CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease vrtx.com ↗
CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the treatment of sickle cell disease (SCD). |
| 2019-01-04 | CRISPR Therapeutics | Regulatory CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease crisprtx.com ↗
CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the treatment of sickle cell disease (SCD). |
| 2018-10-10 | Vertex Pharmaceuticals Incorporated | Regulatory CRISPR Therapeutics and Vertex Announce FDA Has Lifted the Clinical Hold on the Investigational New Drug Application for CTX001 for the Treatment of Sickle Cell Disease vrtx.com ↗ |
| 2018-10-10 | CRISPR Therapeutics | Regulatory CRISPR Therapeutics and Vertex Announce FDA Has Lifted the Clinical Hold on the Investigational New Drug Application for CTX001 for the Treatment of Sickle Cell Disease crisprtx.com ↗
CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold and accepted the Investigational New Drug application (IND) for CTX001 for the treatment of sickle cell disease (SCD). |
| 2018-05-30 | Vertex Pharmaceuticals Incorporated | Regulatory CRISPR Therapeutics and Vertex Provide Update on FDA Review of Investigational New Drug Application for CTX001 for the Treatment of Sickle Cell Disease vrtx.com ↗
the U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Investigational New Drug Application (IND) for CTX001 for the treatment of sickle cell disease |
| 2018-05-30 | CRISPR Therapeutics | Regulatory CRISPR Therapeutics and Vertex Provide Update on FDA Review of Investigational New Drug Application for CTX001 for the Treatment of Sickle Cell Disease crisprtx.com ↗
the U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Investigational New Drug Application (IND) for CTX001 |
| 2017-12-10 | CRISPR Therapeutics | Results CRISPR Therapeutics Announces Oral Presentation of New Data on CTX001, a CRISPR Gene-Edited Therapy for β-Thalassemia and Sickle Cell Disease, at the ASH Annual Meeting crisprtx.com ↗
Data presented at ASH demonstrate that CRISPR Therapeutics’ proprietary CRISPR gene-editing approach results in high editing efficiency, with >90% of the hematopoietic stem cells edited at the target site. |
| 2017-12-07 | CRISPR Therapeutics | Regulatory CRISPR Therapeutics Submits First Clinical Trial Application for a CRISPR Gene-Edited Therapy, CTX001 in β-thalassemia crisprtx.com ↗
CRISPR Therapeutics (NASDAQ:CRSP) today announced the submission of a Clinical Trial Application (CTA) for CTX001 in β-thalassemia. |
All press releases naming this drug 74 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 9 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | EXAGAMGLOGENE AUTOTEMCEL | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095474 ↗ |
| Known as | Casgevy | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095474 ↗ |
| Known as | CTX001 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07577817 ↗ |
| Action | Restore | “designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing” PMID 38657265 ↗ Apr 2024 |
| Modality | CRISPR / gene editing | “modified with CRISPR-Cas9” NCT05477563 ↗ |
| Route | Intravenous | “Administered by IV infusion following myeloablative conditioning with busulfan” NCT03655678 ↗ |
| Target | BCL11A | “CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene)” NCT03655678 ↗ |