MaxCyte Inks Platform License With Vertex For Gene-Editing Program For Rare Blood Disorders


MaxCyte Inc MXCT has signed a strategic platform license with Vertex Pharmaceuticals Incorporated VRTX.

  • Under the terms of the agreement, Vertex obtains non-exclusive clinical and commercial rights to use MaxCyte's Flow Electroporation technology and ExPERT platform in developing its CRISPR/Cas9-based gene-edited therapy (exa-cel, formerly known as CTX001).
  • MaxCyte is entitled to receive platform licensing fees and program-related revenue.
  • Exa-cel is an investigational ex vivo CRISPR gene-edited cell therapy for transfusion-dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
  • The same MaxCyte technology was used in the development of exa-cel under an agreement between MaxCyte and CRISPR Therapeutics AG CRSP.
  • On Tuesday, the FDA granted exagamglogene autotemcel (exa-cel) a rolling review for the potential treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
  • Vertex will submit its biologics licensing application for exa-cel, beginning in November, and expects to complete the submission by the end of the first quarter of 2023. 
  • The company is on track to submit marketing applications to the European Medicines Agency by the end of the year.
  • Price Action: MXCT shares are up 10.2% at $6.40 on the last check Wednesday.
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