Sickle cell pipeline gets edited down

The pipeline for gene-edited ex vivo projects for sickle cell disease, once full to bursting, now looks a lot less crowded. Three contenders have dropped out of the race in the past two days: Intellia/Novartis’s OTQ923, Graphite’s nula-cel and Sangamo’s BIVV003. The writing was already on the wall for OTQ923 and nula-cel following disappointing data at Ash 2022 and a clinical hold respectively. Intellia is now focused on an in vivo editing approach in sickle cell, which is in preclinical development. Sangamo produced decent data with SAR445136; Sanofi handed back its rights to the project around 12 months ago after deciding to refocus on allogeneic approaches. Sangamo is now looking for a new partner, but the project might be too far behind Crispr/Vertex’s exa-cel to draw much interest. Attention now turns to Editas, which expects to give a mid-year update on the Ruby study of EDIT-301.