Next-generation CRISPR gene editing technologies from Emendo Biotherapy make a real breakthrough with an allele-specific approach to severe congenital neutropenia associated with the ELANE gene…

  • Emendo Biotherapy presents pre-clinical data on the treatment of ELANE gene-associated severe congenital neutropenia with an allele-specific editing approach, creating a highly specific editing composition without precise targets and full allelic specificity in an oral presentation at the annual ASGCT conference. Rafi Emmanuel, PhD, Executive Vice President of Research and Development at Emendo

  • Emendo also presented three posters at ASGCT highlighting the use of Emendo nucleases other than NGG PAM in immuno-oncology, a gene editing solution to optimize LDLR expression and promote LDL cholesterol uptake in familial hypercholesterolemia, and difficulties and differences in nuclease classification. subtypes associated with CRISPR type II


Emendo Biotherapy’s Next Generation CRISPR Gene Editing Technologies Breakthrough Allele-Specific Approach to Treatment of Gene-Related Severe Congenital Neutropenia ELANpresented at the ASGCT Annual Conference

Emendo Biotherapy presented the results of its CRISPR-based next generation gene editing approaches for various indications in an oral presentation and three poster presentations at the 25th conference.and Annual Meeting of the American Society for Gene and Cell Therapy (ASGCT) May 16-19, 2022 in Washington DC.

Emendo presents preclinical data on the treatment of gene-related severe congenital neutropenia ELAN using an allele-specific editing approach, demonstrating the power of Emendo’s two technology platforms that allow the development of a highly specific editing composition that exhibits aimless and comprehensive allele-specificity. In addition, the lack of non-target activity of the Emendo-designed and optimized OMNI nucleases also ruled out any translocation. Patient-derived CD34+ edited cells normally differentiated into neutrophils and also in vitro thatin vivodemonstrating complete engraftment and recovery for all blood lines, which is required to achieve the desired therapeutic effect.

“Severe congenital neutropenia associated with the genome ELAN, Emendo’s primary indication, is a devastating disease affecting pediatric patients that has so far remained untreatable,” said David Baram, Ph.D., president and CEO of Emendo Biotherapys. “We are very excited about this potentially curative treatment developed by our team and are excited to present our preclinical results for this program and other diverse applications of our two ASGCT gene editing technology platforms. We welcome discussion of the ideas generated by our findings regarding the classifications of CRISPR type II nucleases, which promise to be revolutionary in this field.”

Members of the Emendo Biotherapy R&D team, including Chief Technology Officer Lior Izhar, Ph.D., and Executive Vice President of Research and Development, Rafi Emmanuel, Ph.D., presented research on Emendo Biotherapys.

Presentation sessions

Title: A novel CRISPR-associated nuclease accurately abolishes the ELANE mutation allele and transposes HSC differentiation into neutrophils in severe congenital neutropenia.

Session Title: Gene Therapy for Immunological Diseases
Date and time of the meeting: Tuesday, May 17, 2022, from 15:45 to 17:30.
Presentation time: 15:45 – 16:00.
Room: 202
Final abstract number: 482

Poster presentations

Title: Unique CRISPR-associated nuclease with non-NGG PAM effectively targets multiple proprietary genomic sites for immuno-oncological therapy

Session Title: Cancer – Targeted Gene and Cell Therapy I
Date and time of the meeting: Monday, May 16, 2022, from 17:30 to 18:30.
Poster number: M-215
Venue: Hall D.
Final abstract number: 334

Title: CRISPR-based gene editing optimizes LDL expression and stimulates LDL cholesterol uptake in familial hypercholesterolemia.

Session Title: Metabolic, Accumulative, Endocrine, Hepatic and Gastrointestinal Diseases II
Date and time of the meeting: Wednesday, May 18, 2022, from 17:30 to 18:30.
Poster Number: W-125
Venue: Hall D.
Final abstract number: 999

Title: Difficulties and differences in the classification of nuclease subtypes associated with CRISPR type II

Session Title: Genetic Targeting and Correction II
Date and time of the meeting: Tuesday, May 17, 2022, from 17:30 to 18:30.
Poster number: Tu-61
Venue: Hall D.
Final abstract number: 556

About Emendo Biotherapy

Emendo Biotherapys, a subsidiary of AnGes, Inc., is a next-generation CRISPR gene-editing company using two proprietary technology platforms to provide high-precision, genome-wide gene editing. Emendo’s new nuclease detection platform expands the targeted genomic range, while its targeted optimization platform enables high-precision editing, including allele editing, while maintaining high efficiency. The power of OMNI’s technology platforms, combined with deep knowledge of genomic medicine, protein engineering, and therapeutic development, gives Emendo a unique advantage in determining indications in hematology, oncology, ophthalmology, and other disease areas. For more information, visit

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