Gene Editing
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Actualités
ADVANCED THERAPIES | Vertex, CRISPR To Submit Exa-Cel to FDA For β-Thalassaemia And Sickle Cell Disease
Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review, beginning in November 2022 and expects to complete the submission package by the end of Q1 2023.…
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Actualités
La Repubblica: Gene Editing Cured Two Siblings With Thalassaemia In Italy
Cured of thalassemia, thanks to gene editing. According to a recent news report published in the Italian newspaper La Repubblica, Erika and Emanuele Guarini, two siblings from Pistoia, Tuscany, aged…
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Actualités
A Head-to-Head Comparison of BMT, Gene Therapy & Gene Editing Through A Brand New TIF Infographic
The expansion of management options and therapies for patients with thalassaemia is nowadays a reality, thanks to the application of new, revolutionary scientific procedures. However, patients’ understanding on the distinct…
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Clinical News
Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
The U.S. Food & Drug Administration (FDA) has cleared an investigational new drug (IND) application for the experimental gene editing therapy GPH101 by Graphite Bio to initiate a Phase 1/2…
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Actualités
EdiGene Announces Approval in China of its IND Application for CRISPR/Cas 9 Gene Editing Therapy ET-01 in β-thalassaemia
The Center for Drug Evaluation (CDE) of China‘s National Medical Products Administration (NMPA) has recently approved EdiGene’s application for ET-01, an investigational CRISPR-Cas9 gene editing therapy for patients with transfusion dependent β-thalassaemia. This marks the first…
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Clinical News
EdiGene Application of CRISPR/Cas 9 Gene-editing Therapy ET-01 For β-Thalassaemia Has Been Accepted For Review
Τhe Chinese biotechnology company EdiGene announced yesterday that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has accepted for review the Company’s Investigational New Drug…
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Actualités
Gene-editing Therapy CTX-001 Granted Priority Medicines Designation for Severe SCD in EU
CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of severe sickle cell disease (SCD). The announcement was made by CRISPR Therapeutics and Vertex Pharmaceuticals, which are jointly…
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