Editas and Genevant Collaborate on Gene Editing Therapies

Editas Medicine and Genevant Sciences have entered into a collaborative agreement to develop gene editing therapies by leveraging their respective CRISPR and lipid nanoparticle (LNP) technologies. Editas, based in Cambridge, Massachusetts, will utilize its CRISPR/Cas12a gene editing system in conjunction with Genevant’s LNP technology, which is headquartered in Basel, Switzerland. This partnership is aimed at creating in vivo gene editing medicines targeting two yet-to-be-disclosed conditions, aligning with Editas’ strategy focused on genetic upregulation.

Under the terms of the agreement, Genevant will grant Editas a nonexclusive, worldwide license to the products resulting from their collaboration. In return, Genevant stands to receive up to $283 million in upfront and milestone payments, in addition to royalties on future sales.

Dr. Linda Burkly, Chief Scientific Officer of Editas, and Dr. James Heyes, Chief Scientific Officer of Genevant, both highlighted the synergistic potential between their companies’ technologies. This announcement triggered a significant increase in Editas stock trading, with over 4.5 million shares exchanged on the day of the announcement, a notable rise from the 860,000 shares traded the previous Friday. As of now, Editas has a market capitalization of $310 million.

In recent years, Editas has strategically entered into licensing agreements to stabilize its financial standing. In December 2023, the company licensed its technology to Vertex Pharmaceuticals for $100 million. Additionally, in October 2024, Editas traded a portion of the revenue from this deal to a DRI Healthcare subsidiary in exchange for a $57 million payment.

Among the licensed assets is Casgevy (exagamglogene autotemcel), the world’s first CRISPR-based gene therapy, which is approved to treat sickle cell disease and beta thalassemia. According to projections by GlobalData, this asset is expected to generate an annual revenue of $857 million for Vertex by 2030.

Editas' current leading candidate, renizgamglogene autogedtemcel, is a CRISPR/Cas12a-based therapy currently undergoing a Phase III clinical trial for sickle cell disease (NCT04853576) and a Phase II trial for beta thalassemia (NCT05444894). This therapy received orphan drug designations for both indications from the US Food and Drug Administration (FDA) in 2023.

Genevant has also been active in licensing its LNP technology to other companies. In July 2018, Genevant collaborated with BioNTech to develop therapies for rare diseases. Further, in March 2022, Genevant entered into a collaboration with Takeda Pharmaceutical to develop gene therapies targeting liver disease.

This collaboration between Editas Medicine and Genevant Sciences marks a significant step in the advancement of gene editing therapies, merging cutting-edge technologies to potentially provide groundbreaking treatments for genetic disorders. The successful integration of CRISPR/Cas12a and LNP technologies could pave the way for new in vivo gene editing solutions, offering hope for patients with rare genetic conditions.