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Editas’s EDIT-101 Faces Uncertainty As LCA10 Data Lack BRILLIANCE

 
• By Alaric DeArment

Data showed efficacy limited to IVS26-mutant Leber congenital amaurosis 10, which affects only 300 patients in the US. The company is pausing enrollment as it seeks a partner.

Editas announced data from the Phase I/II BRILLIANCE trial of EDIT-101 in LCA10 • Source: Shutterstock

Editas Medicine is pausing enrollment in the Phase I/II BRILLIANCE study as it seeks a partner after data indicated the commercial opportunity in Leber congenital amaurosis 10 (LCA10) for its in vivo CRISPR/Cas9 genome-editing medicine EDIT-101 was too small for the company to viably pursue on its own. But the results have also raised doubts about whether the company will be able to find an ideal home for the product candidate.

On 17 November, the company announced data from BRILLIANCE showing that of 14 patients, three met a responder threshold, experiencing clinically meaningful improvements in best corrected visual acuity (BCVA); two...

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