Emendo Biotherapeutics’ next generation CRISPR gene editing technologies achieve breakthrough results with allele-specific approach for ELANE-related Severe Congenital Neutropenia as presented at ASGCT Annual Meeting

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  • Emendo Biotherapeutics revealed pre-clinical data for the treatment of ELANE-related Severe Congenital Neutropenia using an allele-specific editing approach, generating a highly specific editing composition that achieves both no off-targets and complete allele specificity, in oral presentation at ASGCT annual meeting by Emendo Executive VP R&D Rafi Emmanuel, Ph.D.

  • Emendo also presented three posters at ASGCT, highlighting applications of Emendo’s non-NGG PAM nuclease in immuno-oncology, a gene editing solution to enhance LDLR expression and boost LDL-C uptake in Familial Hypercholesterolemia, and challenges and inconsistencies in Type II CRISPR-associated nuclease subtype classification

NEW YORK, May 17, 2022--(BUSINESS WIRE)--Emendo Biotherapeutics presented the results of its next generation CRISPR-based gene editing approaches for several indications in an oral presentation and three posters at the 25th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) held May 16-19, 2022, in Washington, D.C.

Emendo presented pre-clinical data for the treatment of ELANE-related Severe Congenital Neutropenia using an allele-specific editing approach, demonstrating the power of Emendo’s dual technology platforms that enable the development of a highly specific editing composition that demonstrates no off-targets and complete allele specificity. Significantly, the lack of off-target achieved by Emendo’s engineered and optimized OMNI nuclease also eliminated any translocations. Edited patient derived CD34+ cells differentiated normally into neutrophils both in-vitro and in-vivo, showing full engraftment and reconstitution of all blood lineages, as required for the desired therapeutic effect.

"ELANE-based Severe Congenital Neutropenia, Emendo’s lead indication, is a devastating disease affecting pediatric patients that until now has been incurable," said David Baram, Ph.D., President & CEO of Emendo Biotherapeutics. "We are excited by the potentially curative treatment developed by our team and were pleased to present our pre-clinical results for this program and other diverse applications of our dual gene editing technology platforms at this year’s ASGCT meeting. And of course, we look forward to the discussion generated by our discoveries around Type II CRISPR nuclease classifications that promise to be ground-breaking in the field."

Senior members of the Emendo Biotherapeutics R&D team including Chief Technology Officer Lior Izhar, Ph.D. and Executive Vice-President Research & Development Rafi Emmanuel, Ph.D. presented Emendo’s research on-site at the conference.

Session Presentation

Title: A Novel Engineered CRISPR-Associated Nuclease Accurately Removes ELANE Mutated Allele and Shifts HSC Differentiation Towards Neutrophils in Severe Congenital Neutropenia

Session Title: Gene Therapy for Immunologic Diseases
Session Date/Time: Tuesday May 17, 2022 3:45 PM - 5:30 PM
Presentation Time: 3:45pm - 4:00pm
Room: Room 202
Final abstract number: 482

Poster Presentations

Title: A Unique CRISPR-Based Nuclease with a Non-NGG PAM Efficiently Targets Multiple Exclusive Genomic Sites for Immuno-Oncology Based Therapy

Session Title: Cancer - Targeted Gene and Cell Therapy I
Session Date/Time: Monday May 16, 2022 5:30 PM - 6:30 PM
Poster Board Number: M-215
Room: Hall D
Final abstract number: 334

Title: CRISPR-Based Gene Editing Enhances LDLR Expression and Boosts LDL-C Uptake in Familial Hypercholesterolemia

Session Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases II
Session Date/Time: Wednesday May 18, 2022 5:30 PM - 6:30 PM
Poster Board Number: W-125
Room: Hall D
Final abstract number: 999

Title: Challenges and Inconsistencies in Type II CRISPR-Associated Nuclease Subtype Classification

Session Title: Gene Targeting and Gene Correction II
Session Date/Time: Tuesday May 17, 2022 5:30 PM - 6:30 PM
Poster Board Number: Tu-61
Room: Hall D
Final abstract number: 556

About Emendo Biotherapeutics

Emendo Biotherapeutics, a subsidiary of AnGes, Inc., is a next generation CRISPR gene editing company leveraging dual proprietary technology platforms to enable high precision gene editing throughout the genome. Emendo’s novel nuclease discovery platform broadens the targetable range of the genome while its target-specific optimization platform enables highly precise editing, including allele specific editing, while maintaining high efficiencies. The capabilities of the OMNI technology platforms, along with deep expertise in genomic medicine, protein engineering and therapeutic development, provide Emendo with a unique advantage when addressing indications within hematology, oncology, ophthalmology and other disease areas. For more information please visit www.emendobio.com.

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Shahar Avni
Business Development Specialist

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