Emendo Biotherapeutics

Emendo Biotherapeutics specializes in gene editing to develop novel therapeutics across hematology, ophthalmology, immuno-oncology, and liver diseases, by leveraging CRISPR gene editing and protein engineering technologies. It uses its proprietary OMNI technology platform, which follows a disease-first approach. It creates and discovers new CRISPR nucleases customized to target specific alleles—two or more versions of a DNA—of diseases. The gene editing process with the identified nucleases is then optimized to develop a custom drug formulation for the specific target. This process intends to improve specificity and prevent off-target events. 

As of March 2024, the company had six programs in its pipeline of which the most advanced program EMD-101 for the treatment of Severe Congenital Neutropenia (SCN) is in the investigational new drug (IND) enabling stage. The remaining programs were all in the preclinical stages and lead optimization. 

In December 2020, Emendo was acquired by Japanese biopharma company Anges Inc for USD 250 million and has been operating independently.

Key customers and partnerships

Emendo’s pipeline consists of four programs in collaboration with educational and research institutes: EMD-101 for SCN with the University of Washington, EMD-102 for immunodeficiency and EMD-103 for bone marrow failure with the National Institutes of Health (NIH), and EMD-201 for Retinitis Pigmentosa with Columbia University.

The company secured a partnership with Takeda Pharmaceuticals in April 2019 to offer its OMNI platform and assist in editing two genes. Takeda also invested in Emendo through its venture capital arm to fund the development of the OMNI platform. It also teamed up with the Seattle Children’s Research Institute in December 2021 to assess how hematopoietic stem cells (HSCs) from SCN patients react to the CRISPR therapeutic.  

Funding and financials

The company raised USD 61 million in a Series B funding round in January 2020, led by Anges Inc. The proceeds were earmarked for advancing its gene editing platform across a broad range of therapeutic areas.