Sickle Cell Disease, SCD

For the inaugural episode of CMN Live on 26th January 2024, we spoke with Eric Kmiec, Ph.D. about the successes of therapeutic gene editing to date,...

Clinical Highlights From the Gene-Editing Field in...

CMN Clinical

Dec. 29, 2023

As 2023 draws to a close, we look back on some of the major clinical highlights from the year gone by.

Clinical Update: Phase 3 in Sight for Caribou's...

CMN Clinical

Dec. 13, 2023

Caribou Biosciences gets closer to Phase 3 with its gene-edited cell therapy candidate for large B-cell lymphoma, and Editas Medicine reports positive...

Startup Spotlight: Ariya Bio - A Unique...

Nov. 6, 2023

Located in the ETH Innovation & Entrepreneurship Lab in Zürich (Switzerland), Ariya Bio's goal is to develop safe and effective gene-editing therapies...

New FDA and EMA Designations for Two Gene-Editing...

Oct. 18, 2023

This update shares news about recent designations awarded by the US FDA and European Medicines Agency for gene-editing candidates for the treatment of...

Editing Stem Cells In Vivo: A Major Stride in Gene...

Oct. 9, 2023

By decorating lipid nanoparticles with a cell-specific surface marker, researchers from the University of Pennsylvania were able to edit...

Clinical Trial Roundup: Base Editors in the Clinic

Oct. 4, 2023

In this clinical trial roundup, we present an overview of the ongoing clinical trials involving base-editing therapeutic candidates. This article...

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...

Sickle Cell Disease, SCD

Sickle Cell Disease, SCD, (NCT06287099)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT06300723)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT06287086)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, and Transfusion-Dependent Beta-thalassemia, TDT, (NCT05145062)
Sponsors:
Sangamo Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Trait, (NCT06071377)
Sponsors:
National Alliance for Sickle Cell Centers

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT03167450)
Sponsors:
National Human Genome Research Institute (NHGRI)

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, and Transfusion-Dependent Beta-Thalassemia, 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