Vertex Receives CHMP Positive Opinion For The First CRISPR/Cas9 Gene-Edited Therapy CASGEVY

RTTNews | 607天前
Vertex Receives CHMP Positive Opinion For The First CRISPR/Cas9 Gene-Edited Therapy CASGEVY

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) announced Friday that the European Medicines Agency's (EMA's) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

If approved, exa-cel would be the only genetic therapy for patients in the European Union who are 12 years of age and older with either severe SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.

An approval decision by the European Commission is expected in February 2024.

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Vertex Pharma Inks Licensing Agreement To Use CRISPR Therapeutics' Gene Editing Technology

Vertex Pharma Inks Licensing Agreement To Use CRISPR Therapeutics' Gene Editing Technology

Biotechnology company Vertex Pharmaceuticals, Inc. (VRTX) and gene editing company CRISPR Therapeutics, Inc. (CRSP) announced Monday that they have entered into a new non-exclusive licensing agreement for the use of CRISPR Therapeutics' gene editing technology, known as CRISPR/Cas9, to accelerate the development of Vertex's hypoimmune cell therapies for type 1 diabetes (T1D).
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