The company had said in October said that it was looking to license out its ex vivo editor renizgamglogene autogedtemcel to focus its resources on its in vivo platform.
Editas on Thursday kicked off a cost-cutting transition initiative after its hunt for a development sponsor for its sickle cell disease therapy renizgamglogene autogedtemcel turned up empty.
As part of the effort, Editas will terminate its work on the ex vivo therapy. The biotech will likewise let go of around 65% of its staff over the next six months, a move that will also affect several members of the company’s management, including its Chief Medical Officer Baisong Mei. These adjustments will help the biotech extend its runway into the second quarter of 2027.
In connection with these changes, Emma Reeve and Meeta Chatterjee will step down from Editas’ Board of Directors effective December 31. Jessica Hopfield will serve as the new chair of the company’s Board.
Thursday’s strategic changes follow a previous announcement, in October, that Editas was looking to out-license renizgamglogene autogedtemcel. At the time, the biotech touted that it was “on-track” to release “substantive” clinical data for the candidate in sickle cell disease and that its Phase I/II/III trial for the asset was ongoing.
Moving forward, Editas will focus its resources and expertise on developing in vivo CRISPR-editing assets. This pivot will capitalize on several recent promising preclinical proof-of-concept findings in various tissues.
For instance, Editas on Thursday also revealed in its press announcement that company scientists were able to achieve approximately 40% editing of the promoter site of the HBG1/2 gene in human hematopoietic stem cells using the company’s proprietary targeted lipid nanoparticle platform.
The HBG1/2 gene encodes for proteins involved in the production of fetal hemoglobin and has been, according to the biotech, “validated and derisked” based on its studies for renizgamglogene autogedtemcel. This in vivo editing approach is still in animal studies.
Editas on Thursday also reported that it had achieved high efficiency editing for its in vivo editor in the liver of nonhuman primates. The biotech is exploring this program in collaboration with Genevant.
Editas president and CEO Gilmore O’Neill in a statement said that these “scientific breakthroughs by the Editas team” have given the biotech confidence regarding the “near-term viability of in vivo CRISPR-edited medicines.” The company aims to produce human proof-of-concept evidence for its in vivo approach in around two years.
“We believe the ability to provide in vivo gene editing that functions via gene upregulation across tissues holds the potential to significantly expand the addressable therapeutic possibilities for CRISPR-based gene editing,” O’Neill added.
In a late Thursday note to investors, Truist Securities analysts were not as optimistic, writing that because human proof-of-concept is still two years away, they “see little reason to be a buyer of EDIT now.”
“Given rapidly evolving competitive dynamics, it’s difficult to have a view on EDIT’s in vivo program at this juncture,” the analysts wrote, adding that “due to lack of clear path to value creating catalysts in next 12-18 months we choose to stay on the sidelines.”