Business Wire

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

Share

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.

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Media:
Shahar Avni
Business Development Specialist
shahara@emendobio.com

About Business Wire

Business Wire
Business Wire



Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

NetApp Empowers Customers to Securely “Talk to Their Data” in Collaboration with NVIDIA18.3.2024 23:00:00 CET | Press release

NetApp® (NASDAQ: NTAP) today announced it is collaborating with NVIDIA to advance retrieval-augmented generation (RAG) for generative AI applications. The new collaboration directly connects the just-announced NVIDIA NeMo Retriever microservices—coming to the NVIDIA AI Enterprise software platform for development and deployment of production-grade AI applications, including generative AI—to exabytes of data on NetApp’s intelligent data infrastructure. Every NetApp ONTAP® customer will now be able to seamlessly “talk to their data” to access proprietary business insights without having to compromise the security or privacy of their data. Enterprises want to leverage publicly available large language models to talk directly and securely to their corporate data with assurance that their private data will never be accessible outside the enterprise. Until now, enterprises that wanted to build internal chatbots, co-pilots, and applications that leverage corporate knowledge were faced with co

KPMG’s "Digital Gateway for Tax" Platform Raises Global Standards in Tax Functionality with Generative AI18.3.2024 18:38:00 CET | Press release

In a landmark move, KPMG has enhanced the capabilities of its Digital Gateway for Tax incorporating generative AI (genAI) as an independent component to communicate directly with data, enabling powerful insights and enhanced levels of efficiency. This pioneering Software as a Service (SaaS) is designed to provide an end-to-end solution for all members of the tax department and marks a significant milestone in tax technology, integrating generative AI for organizations embarking on their tax digital transformation journey. The KPMG Digital Gateway for Tax platform’s real strength lies in its content and transparent data enrichment capabilities. The solution empowers clients to use the Digital Gateway as their Centralized Knowledge Hub, where users can access company-specific documents through the unified genAI platform. The RAG approach (Retrieval Augmented Generation) is relied upon to effortlessly enrich models with additional content which helps ensure the information used to generat

Media invitation: World leaders must make headway for adaptation - there are no alternatives18.3.2024 18:06:00 CET | Press release

A coalition of international climate organizations will come together for an event in Copenhagen on the 20 March 2024 to deliver recommendations on integrating climate and development for climate finance to world leaders, ahead of a year of intense negotiations, starting at the Copenhagen Climate Ministerial. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240318208761/en/ Photo: Mathilde Utzon The year 2024 is of paramount importance for our future. We face an urgent need to handle the climate crisis, within the broader range of Sustainable Development Goals. This year’s high-level meetings leading up to COP29 will focus on climate finance, aiming to address the substantial funding gaps. To kick-start these discussions DanChurchAid, E3G, the International Institute for Environment and Development, CONCITO, NDC Partnerships, and the UN Foundation are coming together to co-host the virtual and in-person event ‘Integrating Clim

Verimatrix Releases Counterspy as a New Anti-Piracy Solution with First-Ever Defensive Capabilities18.3.2024 17:45:00 CET | Press release

Regulatory News: Verimatrix, (Euronext Paris: VMX), the leader in powering the modern connected world with people-centered security, today announced general availability of its new Verimatrix Counterspy anti-piracy solution -- the most advanced anti-piracy technology seen in the past decade. Counterspy leverages technology first developed by the company's cybersecurity team back in 2021 to offer an innovative new way to counter the rise in video piracy in an era where streaming apps are prevalent. Counterspy is a standalone product within Verimatrix's anti-piracy team, engineered to prevent video content theft, shield apps from attack, and provide ongoing piracy threat prediction, detection and response services. Counterspy goes beyond traditional methods to ensure top-tier security for media app subscribers, safeguarding content across various devices. It fills the gap in authentication created by the shift from operator-controlled set top box hardware to retail or app-based OTT clien

Centrico Spa (Gruppo Banca Sella) and Veracode Enter Deal to Help Secure the Application Development Life Cycle18.3.2024 15:42:00 CET | Press release

Centrico Spa, part of Gruppo Sella and a specialist in providing open banking systems for innovation-oriented financial institutions, has entered a collaboration with Veracode, a global leader in application security risk management. The deal will enhance the analysis, detection, and prioritization of flaws to expedite software fixes at every stage of Centrico’s application development life cycle. Modern banking and finance are exposed to a stringent and evolving landscape, not least as a result of the Digital Operational Resilience Act (DORA) and an increase in frequent and sophisticated cyberattacks. According to data collected in Veracode’s State of Software Security Report, 72 percent of financial organizations contain vulnerabilities—the lowest percentage of all sectors analyzed and an improvement from the previous year. Even so, financial organizations would benefit from increased automation and secure coding techniques to help them prevent, detect, and respond to vulnerabilities

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom
HiddenA line styled icon from Orion Icon Library.Eye