Editas Medicine is ousting its ex vivo sickle cell and beta thalassemia gene therapy to instead focus on an in vivo treatment for the diseases.
The company announced Tuesday that it’s seeking to partner or out-license its lead clinical program, known as reni-cel. The press release also detailed the gene editing company’s new and ambitious focus to develop an in vivo treatment for sickle cell disease and beta thalassemia, and divulged some initial results for the therapy in mice.
Gilmore O’NeillDuring an analyst call on Tuesday morning, Editas CEO Gilmore O’Neill said the company is not yet sharing a timeline for when it plans to begin human studies with the in vivo therapy. To date, no in vivo gene editing treatments for sickle cell disease or thalassemia have been studied in clinical trials.
“We believe there are several beneficial financial differences versus making ex vivo autologous cell therapies,” Editas CFO Erick Lucera said during the call. “Among them is our belief that in vivo medicines will provide larger market opportunities and do so with lower cost of goods and lower clinical trial costs.”
Editas’ pivot comes as the companies that brought to market two sickle cell gene therapies, which require a patient’s blood stem cells to be extracted and then returned to their body, have seen slow commercial launches.
While Vertex and bluebird bio have said that the launches will take time, the broader cell and gene therapy field has also seen cooling interest from investors and pharma companies. And bluebird bio, which is solely focused on gene therapies, has been forced to renegotiate a loan, lay off staff and cut costs in recent months.
Editas would have been a distant third-to-market with reni-cel, which uses gene editing to modify blood stem cells that are taken from the patient so they make fetal hemoglobin. The edited cells are then infused back into the patient. Beam Therapeutics is also developing a base-edited ex vivo sickle cell gene therapy.
“Investors had grown increasingly skeptical and cautious on reni-cel’s commercial prospects,” Stifel analyst Dae Gon Ha told investors on Tuesday.
Editas’ shares $EDIT fell about 8% Tuesday morning.
The shift to in vivo gene editing marks yet another pivot for Editas, which was founded in 2013 to develop new therapies from CRISPR technology. The company had been working on a treatment for a rare inherited form of blindness, as well as cancer programs, before shifting its focus in 2023 to its ex vivo sickle cell disease and beta thalassemia therapy, which commanded a larger market than the rare form of blindness it had been working on.
The in vivo approach would deliver a gene editor using a fatty acid envelope targeted at cells outside the liver to induce fetal hemoglobin expression in blood stem cells directly in the body. Typically, these fatty acid particles called LNPs like to accumulate in the liver, and delivery beyond the liver has been a white whale for genetic medicine developers.
In mice engrafted with human blood stem cells, Editas said that researchers observed 29% editing efficiency with a single dose of therapy, and that approximately 20% of fetal hemoglobin-expressing red blood cells populated the host at one month.
A handful of other companies, including Tessera Therapeutics, are also trying to develop in vivo gene editing treatments for sickle cell disease.
The company also announced on Monday that it signed a non-exclusive license deal with Genevant Sciences for LNP technology for two undisclosed targets. The agreement would give Genevant up to $238 million in upfront and milestone payments plus royalties. Editas declined to disclose the upfront payment of the deal.