The latest clinical data of Vertex/CRISPR gene editing therapy will be announced

CompilationFan Dongdong

A few days ago, Vertex and CRISPR released 2 summary data of CRISPR/Cas9 gene editing CTX001 therapy, introduced the updated data of the ongoing CTX001 hemoglobinopathy clinical trial, and will be held at the European Hematology Association (EHA) on June 11.

One of the abstracts is numbered EP736, and the title of the abstract is "CTX001 for Sickle Cell Disease: Safety and Effectiveness of Ongoing CLIMB SCD-121 Autologous Crispr-Cas9 Modified CD34+ Hematopoietic Stem and Progenitor Cell Research result".

Another abstract, code-named EP733, will be published in the video conference, titled "CTX001 for blood transfusion-dependent thalassemia: the ongoing CLIMB Thal-111 study of autologous Crispr-Cas9-modified CD34+ hematopoietic stem and progenitor cells Safety and effectiveness.

Vertex and CRISPR reached a strategic research cooperation in 2015, hoping to develop CRISPR/Cas9 potential new therapies against the inherent genetic causes of human diseases.

Currently, CTX001 is undergoing two clinical trials as a potential, one-time treatment for patients with TDT and severe SCD.

According to other patient data and follow-up results (f/u) in the interim data summary on January 21, 2021, all TDT patients included in the CLIMB THAL-111 trial (NCT03655678) have been injected into the TDT analysis when f/u≥ CTX001 for 3 mo (N = 10).

All 10 patients reported adverse events; most were mild or moderate in severity, and the safety profile after CTX001 was usually consistent with the results of busulfan for bone marrow ablation.

At present, CTX001 has been granted regenerative medicine advanced treatment product (RMA), fast track qualification (FTD), orphan drug qualification (ODD), and rare pediatric disease qualification (RPDD) granted by the US FDA for the treatment of TDT and SCD.

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