Editas Medicine is pushing its in vivo gene therapy strategy to the top of its agenda, boasting proof-of-concept data and signing a $238 million biobucks pact at the same time as it searches for a partner for its more advanced ex vivo sickle cell disease (SCD) medicine.
The biotech announced this morning that it had proven that its in vivo candidate was capable of hematopoietic stem and progenitor cell (HSPC) editing and fetal hemoglobin induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells.
Specifically, the candidate enabled an editing level of 29% in HSPCs after a single dose by “utilizing a hematopoietic stem cell targeting strategy and tLNP formulation optimization,” the company said.
“Achieving in vivo preclinical proof of concept in a desired, extrahepatic target cell type, delivered utilizing a proprietary Editas LNP that works outside the liver, puts us on a clear path to develop a potentially first- and best-in-class in vivo gene-edited medicine for the treatment of sickle cell disease and beta thalassemia,” Editas CEO Gilmore O’Neill said in a release this morning.
While Editas' in vivo candidate uses targeted lipid nanoparticles (LNP) that were developed in-house, the biotech also expanded its LNP expertise via a collaboration with Genevant yesterday. The collaboration will see Editas’ CRISPR Cas12a genome editing systems combined with Genevant’s LNP tech to develop in vivo gene editing medicines aimed at two undisclosed targets.
“As we investigated the delivery landscape to identify systems for our in vivo upregulation strategy that would best complement our gene editing technology, we quickly identified Genevant, an established leader in the LNP space, and we are delighted to launch this collaboration,” Editas chief scientific officer Linda Burkly, Ph.D., explained in a statement yesterday.
The Roivant offshoot will be in line to receive up to $238 million from Editas—including an undisclosed upfront fee as well as milestone payments—plus tiered royalties should a med make it to market.
Editas also shared an update on the reni-cel program this morning, namely that the company will now seek out a partner for this SCD medicine. Despite the FDA’s approval of two SCD gene therapies late last year in the form of Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia, Editas has remained confident it has a potentially "best-in-class" therapy. Now, though, the biotech has decided the best financial path is to partner or out-license the 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,” O’Neill said in this morning's 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.”
A spokesperson for the company told Fierce Biotech that despite the hunt for a partner, work on reni-cell has not been halted. The biotech has now fully enrolled the adult and adolescent cohorts of the RUBY trial of the SCD med, with a “substantive clinical data set” due to be unveiled at the American Society of Hematology Annual Meeting in December, the spokesperson pointed out.
“We also continue to dose patients in the EdiTHAL trial of reni-cel for the treatment of beta thalassemia and will share additional clinical data this year,” they added.
On a call with analysts this morning, O'Neill said the biotech remains “highly committed” to working towards an FDA approval application for reni-cell, and the company has “made excellent progress in that direction.”