EHA 2023 – Editas hopes to be better, although late

“We remain confident that we have not just a competitive product, but … a differentiated medicine.” So said Editas’s chief executive officer, Gilmore O’Neill, during a conference call today to discuss the group’s Crispr-edited sickle cell and beta-thalassaemia contender EDIT-301. In reality, the claim of differentiation remains hard to assess, given that Editas presented data on just one beta-thalassemia and four sickle cell subjects at EHA on Saturday. Results in sickle cell looked good, with robust increases in foetal haemoglobin and, just as importantly, no vaso-occlusive crises. But, as Stifel analysts pointed out, they also look broadly similar to data with Vertex/Crispr’s rival project exa-cel, which is due an approval decision before year end. Stifel wrote that EDIT-301's expected later entrance into the market without a clear differentiation could be a disadvantage. Editas plans to have dosed 20 patients in the Ruby sickle cell trial by the end of this year, and hinted today that this might be enough to file. In beta-thalassaemia, short follow-up makes the EDIT-301 data even harder to critique.

For a deeper dive on Editas, what might help differentiate EDIT-301, and the gene editing space more broadly, download our recent report.

Source: EHA & company presentation.

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