Takeda Pharmaceutical has advanced further into gene editing, inking a licensing option agreement with and, through its venture capital arm, investing in Emendo Biotherapeutics, the gene editing discovery platform developer said today.

The value of the agreement and the investment were not disclosed.

Through the licensing option agreement, Emendo has granted Takeda the option to use its proprietary nuclease program OMNI to edit two genes as part of the pharma’s R&D efforts to treat unspecified genetic disorders.

Headquartered in New York City, with an R&D center in Ness-Ziona, Israel, Emendo focuses on engineering site-specific nucleases for an accurate, sequence-specific and efficient correction mechanism that is homology-directed repair (HDR) mediated, and effective in post-mitotic cells. Emendo uses proprietary protein engineering and selection platforms that are designed to generate ultra-specific and highly active CRISPR OMNI nucleases.

“We are working towards a well-established platform that will enable us to address a large pool of genetic diseases, many of which have been untreatable until now,” Emendo states on its website.

In the agreement with Takeda, Emendo agreed to apply its novel gene editing engineering platform to optimize the OMNI nuclease, with the aim of achieving the highest specificity and activity on Takeda’s chosen genes, which were not disclosed in Emendo’s announcement.

“This agreement further positions Emendo as a leading, next-generation, high-precision, gene editing company and promotes its allele-specific editing programs that can potentially cure severe genetic disorders,” Emendo CEO David Baram, PhD, said in a statement.

In addition, Takeda agreed to issue Emendo convertible notes through its venture arm Takeda Ventures, with funds from the investment intended to further advance Emendo’s novel nuclease gene editing discovery platform and proprietary product development programs.

Financing toward clinical trials

The notes will be converted to a future Series B financing round being planned by Emendo, which the company said is intended to allow it to advance its high precision gene editing biotherapies into initial clinical trials.

Emendo said it is moving several preclinical programs through optimization, in preparation for moving towards the clinic in a number of “promising” clinical indications.

The transactions expand an unspecified Takeda investment in Emendo that stretches back to the founding of Emendo in 2015, when Takeda Ventures invested in the company along with OrbiMed, a healthcare investment firm that manages approximately $14 billion across public and private company investments worldwide.

“Extending our investment in Emendo and licensing its nuclease discovery platform and gene editing technology is another example of Takeda’s commitment to investing in transformative advances in science via mutually advantageous partnerships,” stated Takeda Ventures senior partner Rob Woodman, PhD.

Takeda has disclosed its partnership with Emendo in the lists of partnerships with other companies that are included in its data supplements to the quarterly results it furnishes to investors.

Over the past year, those lists have also included two other partnerships that incorporate gene or genome editing. Takeda has teamed up with the Japan Agency for Medical Research and Development (AMED) through its Cyclic Innovation for Clinical Empowerment (CiCLE) program toward the development of a hypertrophic cardiomyopathy drug that uses induced pluripotent stem cell-derived cardiomyocytes with disease-causing mutations that were induced by gene-editing technology.

Takeda also disclosed retaining a financial interest in spinout company GenAhead Bio, a Fujisawa City, Japan, company to which Takeda provides “efficient genome editing, fee for service and/or collaboration in cell/gene therapies,” as well as nucleic acid delivery on specific cell types.

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