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Phase 1-2 Study: Safety and efficacy of EDIT-101 CRISPR-Cas9 treatment for CEP290-associated retinal degeneration – N Engl J Med

25 May, 2024 | 19:42h | UTC

This study evaluates the safety and effectiveness of EDIT-101, a CRISPR-Cas9 gene-editing therapy, in treating inherited retinal degeneration caused by CEP290 IVS26 variants. Conducted as a phase 1-2, open-label, single-ascending-dose trial, it involved 14 participants (12 adults aged 17-63 and 2 children aged 9 and 14) who received subretinal injections of EDIT-101. Treatment doses varied, with two participants at a low dose, seven at an intermediate dose, and five at a high dose. The primary safety assessment showed no serious adverse events or dose-limiting toxic effects. Notably, 64% of participants exhibited significant improvements in visual acuity, retinal sensitivity to red light, or mobility. Additionally, improvements in vision-related quality of life were documented in six participants. These promising results suggest that EDIT-101 is safe and potentially effective, warranting further investigation into CRISPR-Cas9 gene therapy for similar genetic retinal conditions.

 

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Reference (link to abstract – $ for full-text):

Pierce, E. A. et al. (2024). Gene Editing for CEP290-Associated Retinal Degeneration. New England Journal of Medicine, (May 6, 2024), 10.1056/NEJMoa2309915.

 


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