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.

Selected ex vivo gene-edited sickle cell projects in development
Project Company Description  Status/trial details
CTX001 Crispr/Vertex Crispr/Cas9 gene-edited cell therapy targeting BCL11a Filed in EU and UK Dec 2022; US submission expected to conclude by the end of Q1 2023
EDIT-301  Editas Crispr/Cas12a gene-edited cell therapy targeting beta-globin to increase foetal haemoglobin Update from Ph1/2 Ruby trial expected mid-2023
BEAM-101 Beam Therapeutics  Base-edited cell therapy promoting foetal haemoglobin Ph1/2 Beacon trial could report 2025
SAR445136 (BIVV003) Sangamo Zinc finger nuclease gene-edited cell therapy targeting BCL11a  Ph1/2 Precizn-1 trial will be completed, but no further investment will be made; company seeking a partner for Ph3 trials
GPH101  Graphite Bio Homology-directed repair gene-edited cell therapy targeting underlying beta-globin mutation Discontinued following case of pancytopenia in Ph1/2 Cedar trial
OTQ923 & HIX763 Intellia/Novartis Crispr/Cas9 gene-edited cell therapy targeting BCL11a Discontinued following poor Ph1/2 data 
BEAM-102 Beam Therapeutics Base-edited cell therapy recreating HbG Makassar variant Deprioritised in favour of BEAM-101 and other technologies
Source: Evaluate Pharma &

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