In 2015, ViaCyte, which was acquired by Vertex Pharmaceuticals in 2022, partnered with CRISPR Therapeutics to develop treatments targeting the genetic causes of human disease.
The first treatment to emerge from this research programme is exagamglogene autoemcel (exa-cel), formerly known as CTX-001, an investigational, autologous, ex vivo clustered, regularly interspaced short palindromic repeats (CRISPR)-Cas9 therapy developed for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD). Clinical trials for exa-cel are underway and have shown promising results so far.
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