Editas Medicine Realigns Strategy to Focus on In Vivo Gene Editing, Targets Human Proof Of Concept By 2026

On Thursday, Editas Medicine Inc. EDIT announced a strategic realignment to focus on in vivo gene editing.

In vivo refers to studies or experiments conducted on living organisms or cells instead of dead organisms or tissue extracts. The opposite of in vivo is in vitro, which means outside the body or in a laboratory.

The shift is designed to optimize costs, extend its cash runway into the second quarter of 2027, and accelerate its progress toward achieving human proof of concept within two years.

The transition follows recent in vivo preclinical research advancements, including proof of concept in multiple tissue types.

For hematopoietic stem cells (HSCs), Editas demonstrated ~40% editing of the HBG1/2 promoter using a proprietary targeted lipid nanoparticle (tLNP).

Also Read: Editas Medicine Downgraded: Analyst Sees Limited Near-Term Catalysts

The single-dose delivery of Cas12a editing machinery directly to human HSCs in mice yielded significant functional outcomes. Within one month, human red blood cells expressed fetal hemoglobin (HbF) at an average of 20%.

The achievement builds on validated HBG1/2 biology from the RUBY trial with Editas’ reni-cel program.

In liver applications, Editas achieved high-efficiency gene editing in non-human primates in collaboration with Genevant.

The company will share detailed preclinical data and development timelines in early 2025.

As part of its transition, Editas announced a workforce reduction, eliminating approximately 65% of its employees over the next six months.

This includes several leadership team members, such as Chief Medical Officer Baisong Mei. The company stated that these changes align with its renewed focus on in vivo therapies.

By restructuring operations and prioritizing its in vivo platform, Editas aims to create a more streamlined and efficient organization.

Cash, cash equivalents, and marketable securities as of September 30, 2024, were $265.1 million.

The company expects the existing cash, cash equivalents, and marketable securities, the upfront cash payment from DRI, and the retained portions of the payments payable under the license agreement with Vertex to fund operating expenses and capital expenditures into the second quarter of 2026.

Price Action: At last check on Friday, EDIT stock was down 8.99% at $1.72 during the premarket session.

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