After making a pivot to in vivo gene editing medicines and dramatically reducing its head count last year, Editas Medicine has shared more clues about its future direction.
Tuesday, the company said it has nominated EDIT-401 as its lead in vivo development candidate. The asset is a one-time therapy designed to significantly reduce LDL cholesterol (LDL-C) levels, having shown a 90% mean reduction in nonhuman primates, according to the company's Sept. 2 release.
The drug is designed to treat high cholesterol by editing the LDLR gene to increase LDLR protein expression and slash LDL-C levels, Editas says. In preclinical testing, the asset has shown a "favorable" profile in terms of both efficacy and tolerability, according to the Massachusetts-based biotech.
The new direction marks a pivot from Editas' research in rare genetic diseases such as sickle cell disease. In pursuing LDL-C reductions, Editas' preclinical prospect could one day serve a market in atherosclerotic cardiovascular disease that's projected to be responsible for more than $300 billion in U.S. healthcare spending in 2035, according to an estimate provided by the company.
In its release, Editas noted that standard of care therapies typically show a 40% to 60% reduction in LDL-C levels. Editas sees its preclinical prospect as a potential "one-time treatment" that's designed to offer "lifelong benefit."
Of course, it's still early days for the program. Under Editas' current plan, the company aims to submit IND or clinical trial filings to the FDA by the middle of 2026. This would potentially allow Editas to deliver in vivo human proof-of-concept data by the end of next year.
The biotech's current stockpile of cash and equivalents, sitting at about $178.5 million, should help fund operations into the second quarter of 2027, Editas said.
The new direction comes after Editas prioritized in vivo gene editing last fall. At the time, the company said it would seek to partner its prior lead program reni-cel, an ex vivo gene therapy aimed at SCD and beta thalassemia.
Two months later, after failing to find a partner for that prospect, Editas laid off 65% of its staff and parted ways with its chief medical officer.
Besides EDIT-401, Editas is continuing research in its hematopoietic stem cell program in order to optimize candidates, but the company says it's funneling most of its resources toward the new lead asset.