Editas Clears Australia Ethics Gate for EDIT-401 Trial

The Tip Desk · November 12, 2026 · 3 min read

Cash, cash equivalents and marketable securities stood at $190.2 million as of September 30, 2026, with a runway the company said extends into the second half of 2028.

Editas Medicine, Inc. (EDIT), a gene-editing company developing in vivo medicines, received Human Research Ethics Committee approval and completed the Clinical Trial Notification process with Australia’s Therapeutic Goods Administration to start the Phase 1/2 Strive study of EDIT-401 in patients with heterozygous familial hypercholesterolemia.

The company said it remains on track to begin patient dosing this year and to report initial safety and tolerability data in the first quarter of 2027. It expects to complete enrollment in Part 1 of the study, with topline safety and efficacy results from that dose-finding portion available in the second half of 2027.

That approval followed a second-quarter update in which Editas said it was on track to submit the Australian CTN that month. EDIT-401 is an experimental in vivo gene-editing medicine designed to edit the LDLR gene to increase LDLR protein expression and reduce LDL-C.

At the American Heart Association Scientific Sessions in November, Editas presented new preclinical data showing LDL-C reduction of at least 90 percent with EDIT-401 maintained over 10 months in an ongoing non-human primate study, extending the roughly six-month durability reported earlier in the year. The company also presented about 90 percent mean reductions in Lp(a) and ApoB maintained over three months in NHPs, and a safety profile it described as consistent with targeted liver delivery, transient liver-function-test elevations, no histopathologic findings, and no evidence of genotoxicity in relevant primary human cells.

In September, Editas appointed Dan Ory, M.D., as Chief Medical Officer to oversee clinical development strategy as EDIT-401 advances into the clinic.

A second IND/CTA was accepted for an alpha-beta T cell medicine under the company’s collaboration with Bristol Myers Squibb, triggering a milestone payment to Editas. BMS will advance BMS-986527, a CD70 healthy-donor allogeneic CAR-T therapy, in a Phase 1 study in clear cell renal cell carcinoma. Editas developed the Cas12a gene-editing technology used to engineer the CAR-T cells.

For the three months ended September 30, 2026, collaboration and other research and development revenues increased to $10.4 million from $7.5 million a year earlier. The company said the increase was primarily attributable to revenue recognized in connection with clinical development milestones achieved by a strategic partner.

Net loss attributable to common stockholders was $23.4 million, or $0.15 a share, compared with a net loss of $25.1 million, or $0.28 a share, for the same period in 2025. Weighted-average common shares outstanding, basic and diluted, were 153.6 million.

Research and development expenses increased by $1.4 million to $21.2 million from $19.8 million a year earlier. The company said the increase was primarily related to higher sublicense and license fees as well as increased costs associated with continued development of EDIT-401, partially offset by lower employee-related costs and external research and development costs.

General and administrative expenses increased by $1.2 million to $13.5 million from $12.3 million a year earlier, primarily attributable to higher intellectual property and patent-related fees due to increased legal activity related to intellectual property defense.

Cash, cash equivalents, and marketable securities as of September 30, 2026, were $190.2 million compared with $146.6 million as of December 31, 2025. Cash and cash equivalents were $93.2 million and marketable securities were $97.0 million. The company said existing cash, cash equivalents, and marketable securities will enable it to fund operating expenses and capital expenditure requirements into the second half of 2028.

President and Chief Executive Officer Gilmore O’Neill said the company is advancing EDIT-401 toward the clinic and looks forward to executing the Strive study and generating additional data in 2027.