Vertex wins third CF approval in Europe; ERS Genomics grants DefiniGEN access to CRISPR/Cas9 tech

Marcus Yeo

→ Vertex $VRTX has scored its third cystic fibrosis treatment approval, with the European Commission giving Symkevi (tezacaftor/ivacaftor) in combination with Kalydeco (ivacaftor) the nod for CF patients with certain mutations in the CFTR gene. The treatment is already approved the US and Canada and is aimed at patients with residual function mutations and those who cannot tolerate Orkambi (lumacaftor/ivacaftor).

→ Dublin-based ERS Genomics has agreed to provide Cambridge-based DefiniGEN access to CRISPR/Cas9 genome editing technology patents. DefiniGEN is hoping to use its stem cell differentiation platform with ERS’ CRISPR/Cas9 technology to produce preclinical human models for its customers in the drug discovery sector, optimising the “development of therapeutics for multiple rare liver diseases, Type 2 diabetes and non-alcoholic fatty liver disease,” said Marcus Yeo, CEO of DefiniGEN.


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Biotech Investment Analyst
SV Health Investors Boston, MA
Director, Program Management
Contrafect Corporation New York, NY
Director, Translational Sciences
Cadent Therapeutics Cambridge, MA

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