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NEWS
Editas Medicine Announces Strategic Transition to in vivo Gene Editing Company with Intent to Achieve Human Proof of Concept in Approximately Two Years
Thursday, 12th December at 4:00 pm
• Focus on in vivo CRISPR-edited medicines based on Editas researchers' recent scientific progress in multiple tissues:
◦ Achieved pre-clinical in vivo proof of concept of high level HBG1/2 promoter editing and HbF induction in a humanized mouse model for treatment of sickle cell disease and beta thalassemia with a single dose of an HSC-targeted lipid nanoparticle (tLNP) formulation
◦ Achieved in vivo proof of concept of high efficiency editing in the liver in non-human primates
• Ending development of reni-cel after extensive search did not yield a commercial partner
◦ The Company will work closely with the clinical trial sites, regulators, and other parties to determine the path forward for patients enrolled in the RUBY and EdiTHAL trials
• Initiating cost savings measures and reduction in headcount to align workforce and resources to in vivo pipeline, extending cash runway into Q2 2027
• Conference call and webcast today at 5:00 p.m. ET
Thursday, 12th December at 4:00 pm
• Focus on in vivo CRISPR-edited medicines based on Editas researchers' recent scientific progress in multiple tissues:
◦ Achieved pre-clinical in vivo proof of concept of high level HBG1/2 promoter editing and HbF induction in a humanized mouse model for treatment of sickle cell disease and beta thalassemia with a single dose of an HSC-targeted lipid nanoparticle (tLNP) formulation
◦ Achieved in vivo proof of concept of high efficiency editing in the liver in non-human primates
• Ending development of reni-cel after extensive search did not yield a commercial partner
◦ The Company will work closely with the clinical trial sites, regulators, and other parties to determine the path forward for patients enrolled in the RUBY and EdiTHAL trials
• Initiating cost savings measures and reduction in headcount to align workforce and resources to in vivo pipeline, extending cash runway into Q2 2027
• Conference call and webcast today at 5:00 p.m. ET
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