Editas Medicine announced its “strategic transition” to an in vivo gene editing company, eyeing human proof-of-concept within the next two years.
The pivot comes with a 65% reduction in headcount over the next six months, along with the departure of management personnel, including the company’s chief medical officer. Editas will also end development of reni-cel — the company’s ex vivo autologous treatment targeting sickle cell and transfusion dependent beta thalassemia — after failing to find a commercial partner.
Editas had communicated its desire to seek a global partner or out-license reni-cel back in October, while simultaneously announcing its achievement of in vivo preclinical proof of concept of hematopoietic stem and progenitor cell (HSPC) editing and fetal hemoglobin (HbF) induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells.
Editas has also achieved in vivo proof of concept of high efficiency editing in the liver in non-human primates through a collaboration with Genevant Sciences that uses Genevant’s lipid nanoparticle patent portfolio.
According to Editas CEO Gilmore O’Neill, these “recent scientific breakthroughs” have accelerated the timelines around the “near-term viability” of in vivo CRISPR-edited medicines for sickle cell disease and beta thalassemia.
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