If Editas Medicine’s CRISPR gene-editing therapy for rare blood diseases reaches the market, it will be in the hands of a different company. The biotech on Tuesday announced plans to partner or out-license that clinical-stage ex vivo therapy, choosing instead to focus its resources on in vivo R&D that now has preclinical proof-of-concept data.
The in vivo therapy, renizgamglogene autogedtemcel (reni-cel), has reached late-stage clinical development for severe sickle cell disease. Reni-cel is also in early-stage development for transfusion-dependent beta thalassemia. Patients who have these rare blood disorders already have ex vivo gene-editing treatment options. Last winter Vertex Pharmaceuticals’ Casgevy landed FDA approvals for sickle cell disease and beta thalassemia. Bluebird Bio’s last winter received FDA approval for Lygenia for sickle cell disease. A different Bluebird gene-editing therapy named Zynteglo won FDA approval in 2022 for beta thalassemia.
Editas’s reni-cel would be third to market, but the Cambridge, Massachusetts-based company has said it could be best in the class of ex vivo therapies, made by editing a patient’s hematopoietic stem cells in a lab and infusing those cells back into the patient. These modified cells are intended to correct the anemia caused by these blood disorders and increase levels of fetal hemoglobin.
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Dosing is continuing in reni-cel’s pivotal sickle cell study with 28 adult patients dosed to date; dosing is being scheduled for adolescent patients. Editas said data will be presented during the upcoming American Society of Hematology meeting in December. The beta thalassemia study is on track to present clinical data by the end of this year.
Speaking during a Tuesday webcast, Editas CEO Gilmore O’Neill said the company is mindful of the financial cost that comes with commercializing an ex vivo autologous therapy. Placing reni-cel in the hands of another company would allow Editas to substantially reduce its 2025 spending.
“We have previously said that we would be open to partnering reni-cel, but now, with the progress we’re making on in vivo editing, we are actively undertaking a process to partner or out-license reni-cel to most effectively drive it to commercialization while allowing us to apply our full attention and capital to in vivo medicines,” O’Neill said. “The in vivo data we are sharing today makes us even more confident in this decision.”
The data that has Editas excited show proof of concept for its in vivo editing approach in mouse models for both sickle cell disease and beta thalassemia. Specifically, the company reported its in vivo therapy enabled an editing level of 29% in hematopoietic stem cells after a single dose. This treatment led to fetal hemoglobin induction (HbF), indicated by the presence of HbF-expressing human red blood cells populating in the host by one month. O’Neill said this level of in vivo editing in mice after a single dose is “highly competitive” compared to data in the public domain for the development of an in vivo medicine for sickle cell disease and beta thalassemia.
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In a note sent to investors, Leerink Partners analyst Mani Foroohar said the decision to seek an external partner for reni-cel makes sense given the cost of commercializing autologous ex vivo therapies. The near-term focus for Editas shares is the additional reni-cel data and the extent to which the company can show differentiation for the therapy to drive business development interest in this program.
Editas said it has engaged investment bank Moelis & Company to lead the global process to partner or out-license reni-cel. Meanwhile, the company has secured non-dilutive financing to support its in vivo gene-editing focus. Earlier this month, Editas sold to DRI Healthcare Trust certain future license fees and payments owed under a Vertex Pharmaceuticals’ licensing agreement for Cas9 gene-editing technology. The DRI deal paid Editas $57 million up front. Editas reported its cash position as of the end of the third quarter of this year was about $265 million; or about $320 million including the DRI payment. O’Neill said the company has enough capital to fund operations into 2026.
Public domain image by Flickr user NIH Image Gallery