Editas Medicine, a biotech firm specialized in CRISPR gene-editing technology, is planning to shift its focus towards developing in vivo therapies by partnering or out-licensing its ex vivo gene-editing treatment for rare blood diseases. This strategic pivot was announced as part of the company’s goal to concentrate on in vivo research and development which has shown promising preclinical proof-of-concept data.
Currently, Editas has an ex vivo therapy called renizgamglogene autogedtemcel (reni-cel) that is making progress in late-stage clinical trials for severe sickle cell disease, and it’s in early development stages for transfusion-dependent beta thalassemia. Reni-cel operates by editing a patient’s hematopoietic stem cells in a laboratory setting, followed by reinfusing them into the patient where they are intended to correct anemia by increasing levels of fetal hemoglobin. Despite its potential, this treatment would only be the third of its kind to enter the market, following behind other FDA-approved treatments like Vertex Pharmaceuticals’ Casgevy for sickle cell disease and beta thalassemia, and Bluebird Bio’s Lygenia for sickle cell disease and Zynteglo for beta thalassemia introduced by last winter.
During a recent webcast, Editas CEO Gilmore O’Neill emphasized that the company is aware of the financial burdens associated with commercializing autologous ex vivo therapies. By finding a suitable partner or licensee for reni-cel, Editas aims to significantly reduce its projected 2025 expenses, freeing up resources to focus on advancing in vivo treatments. This strategic realignment is supported by promising new data from their in vivo research showing successful gene editing in mouse models for both sickle cell disease and beta thalassemia using a single dose approach. The in vivo therapy showed an editing level of 29% in hematopoietic stem cells, leading to the induction of fetal hemoglobin, a crucial factor in treating these blood disorders.
The promising outlook of in vivo editing, coupled with the operational and financial efficiency of focusing resources on this approach, has reinforced the company’s decision to out-license reni-cel. O’Neill expressed confidence in this direction due to the competitiveness and potential of their in vivo editing capabilities demonstrated in preclinical models.
To facilitate the partnering or licensing of reni-cel, Editas has enlisted the expertise of Moelis & Company, an investment bank, to manage the global process. This move comes as Editas has also secured additional non-dilutive financing to bolster its shift towards in vivo gene-editing research. Earlier this month, Editas arranged a deal with DRI Healthcare Trust, selling certain future license fees and payments owed under a Vertex Pharmaceuticals licensing agreement. This agreement has provided Editas with an immediate $57 million, contributing to a total cash position of about $320 million when combined with its third quarter financials, thus extending its operational runway into 2026.
Leerink Partners analyst Mani Foroohar commented on the decision, noting that focusing on additional reni-cel data could enhance the therapy’s distinctiveness and attractiveness to potential partners or licensees, which makes financial and strategic sense for Editas given the high costs associated with commercializing autologous ex vivo therapies.
This strategy enables Editas to streamline its efforts towards potentially revolutionizing gene therapy, targeting the treatment of genetic diseases directly inside the patient’s body, thereby reducing complications and costs associated with laboratory-based gene editing of stem cells followed by reinfusion into patients. With this adjusted focus, Editas is positioned to explore and potentially harness the full promise of in vivo gene editing technologies for treating severe genetic disorders.
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