NEW YORK – Editas Medicine on Tuesday said it will seek a global development and commercialization partner or an out-licensing deal for an investigational treatment for severe sickle cell disease and transfusion-dependent beta thalassemia (TDT) so that it can focus resources on its pipeline of in vivo gene-edited therapies.
Cambridge, Massachusetts-based Editas' renizgamglogene autogedtemcel (reni-cel), previously called EDIT-301, comprises autologous CD34-positive hematopoietic stem and progenitor cells that are edited at the gamma globin genes HBG1 and HBG2 promoters ex vivo in an effort to promote production of fetal hemoglobin. Reni-cel is the company's first clinical-stage candidate.
"We are laser focused on the optimal use of capital as we continue development of reni-cel, invest in our in vivo pipeline and vision, and map to the future," Editas President and CEO Gilmore O'Neill said in a statement. "We believe that the best option for both patients and our shareholders is for us to seek an alternative such as a global partner or out-licensing, which would allow for further development and ultimately commercialization of reni-cel with or by another party and would allow us to substantially reduce spend in 2025."
Editas has engaged the investment bank Moelis & Company to lead the process of identifying a development and commercialization partner or securing an out-license deal for reni-cel.
In January, O'Neill had said Editas could file a biologics license application with the US Food and Drug Administration for reni-cel by late 2025.
Now, Editas said it plans to focus on a separate, newly announced sickle cell disease and TDT treatment, for which it recently demonstrated preclinical proof-of-concept in a study of humanized mice. This preclinical candidate involves in vivo editing of hematopoietic stem and progenitor cells using the company's targeted lipid nanoparticle formulation.
"I am delighted with our concrete progress on in vivo functional upregulation as we drive towards our long-term vision to be a leader in in vivo programmable gene editing," O'Neill said.
Both gene-editing treatments would be a competitor to Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy (exagamglogene autotemcel), which was the first-ever CRISPR gene-editing therapy to receive marketing approval in the US and other countries and is a treatment for sickle cell disease and TDT that involves editing the BCL11A gene ex vivo.
This latest update follows Editas' announcement earlier this month that it had sold certain licensing fees and other payments it is slated to receive from Vertex under a CRISPR license agreement to a subsidiary of DRI Healthcare Trust for $57 million in upfront cash. Editas said at the time that it will use the funds to advance its therapeutic pipeline and other strategic priorities.
Editas on Tuesday also reported that it ended Q3 2024 with about $265 million in cash, cash equivalents, and marketable securities. Once it receives the upfront cash payment from DRI, it will have roughly $320 million.
Separately, Editas on Monday announced a collaboration with Genevant Sciences to develop in vivo gene-editing therapeutics directed at two undisclosed targets, combining Editas' CRISPR Cas12a gene-editing systems with Genevant's lipid nanoparticle technology. Genevant has granted Editas a nonexclusive worldwide license to its technology and is eligible to receive up to $238 million in upfront and possible milestone payments, as well as tiered royalties on potential product sales as part of the deal.