Emendo Biotherapeutics
Emendo Biotherapeutics, acquired by AnGes, Inc. in December 2020, specializes in CRISPR-based gene editing technology with a focus on allele-specific editing to treat Severe Congenital Neutropenia (SCN). Their OMNI™ technology platform enables precise gene editing for various genetic diseases, supported by a team of over 90 scientists and engineers.
Acquisition by AnGes, Inc.
Emendo Biotherapeutics was acquired by AnGes, Inc. in December 2020. This acquisition marked a significant milestone for the company, enabling it to further its development in the field of gene editing technologies. AnGes, Inc., a biopharmaceutical company, is known for its focus on gene therapy and DNA-based vaccines, aligning well with Emendo Biotherapeutics' expertise in CRISPR-based gene editing.
Funding and Investment Partners
In a notable funding round, Emendo Biotherapeutics raised $61 million, with Takeda Pharmaceuticals participating as a key investor. This funding supported the advancement of their CRISPR-based gene editing programs and technological development. Such substantial financial backing underscores the confidence of investors in the potential and innovative approach of Emendo Biotherapeutics in the gene editing space.
CRISPR-Based Gene Editing Technology
Emendo Biotherapeutics focuses on CRISPR-based gene editing technology, developing customized nucleases designed for specific genetic diseases. Their technology platform, named OMNI™, enables extremely precise gene editing and is adaptable for a variety of in-vivo delivery systems and ex-vivo cell therapy. The OMNI™ platform is essential for ensuring high activity and specificity while minimizing off-target effects.
Lead Program: Severe Congenital Neutropenia (SCN)
Emendo Biotherapeutics’ lead program targets Severe Congenital Neutropenia (SCN) through allele-specific editing. This approach focuses on ex-vivo editing of patient-derived hematopoietic stem cells (HSCs) to create therapeutic effects. The lead program is currently in the IND-enabling phase, showing promise in the potential cure of SCN by precisely targeting and editing specific alleles responsible for the disease.
Pipeline and Scientific Collaboration
Emendo Biotherapeutics boasts a robust pipeline that includes programs in hematology, cardiovascular, and ophthalmology. Their collaborative team, comprising more than 90 scientists and engineers, works on developing novel approaches and technologies in gene editing. The team’s efforts are concentrated on optimizing nucleases and guide-RNAs, ensuring their platform can address various genetic disorders with high specificity and efficacy.