A Harsh Reset for a CRISPR Pioneer

On November 7, 2024, Editas Medicine announced a dramatic restructuring, laying off approximately 65% of its workforce after failing to secure a development partner for its sickle cell disease program. The Cambridge-based biotech, once a flagship name in CRISPR gene editing, is now narrowing its focus to EDIT-301, a gene therapy candidate for Leber congenital amaurosis 10 (LCA10)—a rare inherited eye disorder.

The layoffs affect roles across R&D, clinical operations, and corporate functions. Editas stated the move was necessary to extend its cash runway and prioritize programs with the highest likelihood of success. The company’s sickle cell program, once seen as a competitor to Vertex and CRISPR Therapeutics, will be shelved unless external funding or partnerships materialize.

This marks one of the most significant downsizing events in the gene editing space in 2024, reflecting broader challenges in translating CRISPR breakthroughs into commercial therapies.

🔬 Strategic Pivot: Betting on Ophthalmology

With its resources now concentrated on EDIT-301, Editas is doubling down on ophthalmology—a field where gene editing has shown promise due to:

• Localized delivery via subretinal injection
• Immune-privileged environment of the eye
• Clear clinical endpoints like visual acuity and retinal structure
• Smaller patient populations suited for rare disease targeting

EDIT-301 uses CRISPR to correct mutations in the CEP290 gene, which causes LCA10. Early clinical data have shown encouraging signs of safety and potential efficacy, with patients reporting improved light perception and retinal response.

Editas plans to:

• Advance EDIT-301 into Phase 2 trials in 2025
• Seek FDA orphan drug and breakthrough therapy designations
• Explore additional ophthalmic indications using its proprietary editing platform

This pivot aligns with a growing trend: gene editing companies are moving toward niche indications with clearer regulatory paths, rather than competing in crowded areas like hemoglobinopathies.