Strategic Transformation 2023
2024 Strategic Objectives
Drive reni-cel (EDIT-301) toward
BLA and Commercialization
Continue enrollment and dosing in the RUBY and EdiTHAL trials of reni-cel
Initiate the adolescent cohort in the RUBY trial
• Present a substantive clinical data set of Sickle cell patients with considerable
clinical follow-up in the RUBY study in mid-2024 and by year-end 2024
Strengthen and Focus Discovery
to Build in vivo Editing Pipeline
• Establish in vivo preclinical proof-of-concept for an undisclosed indication
o Focus on disease targets with high probability of technical, clinical,
regulatory, and commercial success
o Initial focus on hematopoietic stem cells (HSCS)
Increase Business Development
Activities and Monetize IP
Derive revenue from the Company's foundational IP, building on the recently
announced license agreements with Vertex Pharmaceuticals and Vor Bio
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