Emendo raises $61M to build on Takeda-partnered gene editing platform

A treatment for severe congenital neutropenia, a dominant indication, is in preclinical at Emendo Biotherapeutics. (LionFive / Pixabay)

Emendo Biotherapeutics has raised a $61 million series B to take forward programs based on its gene editing platform. The New York-based biotech has worked to expand the list of diseases amenable to treatment with gene editing, resulting in a pipeline of hematology and ophthalmology programs. 

While CRISPR has opened up new gene editing possibilities, the technology has limitations stemming from the specificity of nucleases. Emendo wants to eliminate the limitations with a technology, called OMNI, designed to have allele specificity, high activity and no off-target effects. Using its nucleases, Emendo aims to treat dominant, dominant negative and compound heterozygous indications. 

The potential of the platform has attracted drug developers and investors alike. For the series B, Emendo put together a syndicate led by AnGes, a Japanese biopharma company that wants access to the fruits of the OMNI platform. AnGes owns close to one-third of Emendo after the investment. 

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AnGes was joined in the series B syndicate by OrbiMed Advisors, OrbiMed Israel Partners and Takeda Ventures. The involvement of Takeda follows the formation of a licensing deal between the Japanese drugmaker and Emendo. Takeda struck the deal last year to gain the option to use OMNI to edit two genes. The collaborators achieved a milestone months after Emendo shared details of the deal. 

Last month, AnGes published a statement (PDF) outlining its investment in Emendo and explaining why it wants to build a close relationship with the company. 

“Emendo is developing high-precision enzymes that do not cut DNA other than the target sequences. In this way, it can be expected that, not only will they achieve highly safe genome editing, they will have more freedom in the selection of targets,” AnGes wrote.

To date, Emendo has used that freedom to develop treatments for hematology and ophthalmology indications while also exploring the application of the technology to cancers. A treatment for severe congenital neutropenia, a dominant indication, is in preclinical in partnership with the University of Washington School of Medicine. Treatments for primary immunodeficiency, bone marrow failure and inherited eye diseases are also in the pipeline. 

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