Vertex Pharmaceuticals, Inc.

Disease Roundup: Four Gene-Editing Candidates in...

Sep. 20, 2023

There are currently four clinical-stage gene-editing candidates in clinical trials for sickle cell disease, which affects millions of people worldwide...

Clinical Roundup: Gene-Edited Therapies for Beta...

Jul. 26, 2023

In this roundup, we provide a summary of the ongoing gene-editing trials for beta thalassemia. This update includes trials sponsored by Vertex...

Clinical Trial Update: Positive Results for...

Jun. 22, 2022

This week's clinical update summarises data shared at the recent European Hematology Congress (EHA2022). The updates include positive data from...

CMN Weekly (29 October 2021) - Your Weekly CRISPR...

Oct. 29, 2021

Some of the best links we picked up around the internet

Clinical Update: Sickle Cell Disease IND Advances...

May. 12, 2021

In this week’s update, we present a CRISPR-based therapy for sickle cell disease that has moved from the investigational new drug (IND) stage into a...

Vertex Pharmaceuticals, Inc.

Sickle Cell Disease, SCD, and Transfusion Dependent Beta-Thalassaemia, TDT, (NCT05477563)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Sickle cell disease, SCD, (NCT05951205)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, and Transfusion Dependent Beta-Thalassaemia, TDT, (NCT04208529)
Sponsors:
Vertex Pharmaceuticals

IND Enabling

Phase I

Phase II

Phase III

Transfusion-Dependent β-Thalassemia, TDT, (NCT05356195)
Sponsors:
Vertex Pharmaceuticals

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT05329649)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (2018-001320-19)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, BT, (NCT03655678)
Sponsors:
Vertex Pharmaceuticals Inc.

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT03745287)
Sponsors:
Vertex Pharmaceuticals

IND Enabling

Phase I

Phase II

Phase III