NY-EMENDO-BIO
17.5.2022 22:17:09 CEST | Business Wire | Press release
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 .
View source version on businesswire.com: https://www.businesswire.com/news/home/20220517006030/en/
Link:
About 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
Financial Services Increases AI Investment Faster Than Wider Market, While Maturity Remains Level28.10.2026 09:56:00 CET | Press release
HTEC research finds 42% of financial services and insurance executives report significantly increased AI investment, compared with 28% across industries, while AI maturity remains in line with the wider market HTEC, the global AI technology and engineering leader, today released its research on AI Value realization in financial services and Insurance. Organizations in this sector are increasing AI investment more aggressively than the wider market, but the sector has yet to establish a corresponding lead in AI maturity, according to new research from HTEC. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261028854882/en/ HTEC, the global AI technology and engineering leader, today released its research on AI Value realization in financial services and Insurance. Organizations in this sector are increasing AI investment more aggressively than the wider market, but the sector has yet to establish a corresponding lead in AI matur
Powens Launches Connector Agentic Factory, an AI System to Build and Fix Open Finance Connectors28.10.2026 09:00:00 CET | Press release
A new chapter in Powens' ambition: Connector Agentic Factory is Powens' new AI system to build, extend and repair Open Finance connectors, a key step toward becoming the most complete and reliable Open Finance infrastructure in EuropeAlready at work: 12 new connectors delivered across Europe, and repairs completed in minutes rather than daysNo control loss: every connector the AI proposes is still reviewed and approved by Powens engineers before it goes live Powens, a leading European Open Finance infrastructure provider, today announced the launch of its Connector Agentic Factory, an internal AI system that is capable of building new Open Finance connectors and repairing existing ones. Connectors are the technical links that let banks, fintechs, and financial apps securely retrieve a customer's financial data, or make payments, from a bank or other institution. Powens maintains more than 1,800 of them across Europe, and they underpin the services that banks, fintechs, lenders, wealth
Ant International CEO: Achieving Inclusion Is Critical to Re-Globalisation, and AI Will Accelerate It by Democratising Opportunities28.10.2026 07:24:00 CET | Press release
Speaking at the United Nations Trade and Development (UNCTAD) World Investment Forum 2026 panel, “Investing in AI for an Inclusive Digital Future”, Ant International CEO Peng Yang called for a new, inclusive “re-globalisation” project that harnesses AI and digital connectivity to give small and medium-sized enterprises (SMEs) greater access to global markets and opportunities. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261027567337/en/ Ant International CEO Peng Yang speaks at the United Nations Trade and Development (UNCTAD) World Investment Forum 2026 “Globalisation 1.0 mostly benefited large enterprises, and a lot of SMEs very rarely got to participate in the global economy,” said Yang. “Achieving inclusion is critical to re-globalisation, and AI will accelerate it by democratising opportunities.” 1. The limitation of Globalisation 1.0 and how AI and other innovations offer a chance to improve Yang said one of the gre
LTM Launches GCC Origin: A New Independently Operated Business Unit for Next-Generation Global Capability Centers28.10.2026 06:04:00 CET | Press release
LTM, the Business Creativity partner to the world’s largest enterprises, today announced the launch of GCC Origin, a dedicated business unit to help enterprises establish, operate, scale and transform Global Capability Centers in India. As GCC needs evolve in the AI era, enterprises are seeking greater scale, speed and integration. GCC Origin represents the evolution of LTM’s GCC strategy to meet these changing needs. Operating as a standalone business unit within LTM, GCC Origin will have dedicated leadership, sales and delivery teams, specialist expertise and significant investment. LTM has appointed Shailendra Singh to lead the business unit, reporting to Venu Lambu, Chief Executive Officer and Managing Director, LTM. GCC Origin brings together strategy, AI, technology, talent, infrastructure, operations and ecosystem partnerships under one integrated model. Spanning site selection, workplace and infrastructure, operational readiness and turnkey facilities across Tier 1 and Tier 2 c
Hitachi Unveils a New Global Digital Services Company Turning Cloud, Data and AI Into Measurable Business Outcomes: Hitachi Digital28.10.2026 01:00:00 CET | Press release
Uniting GlobalLogic and Hitachi Digital Services under a single global brand, Hitachi Digital will deliver end-to-end capabilities that help organizations in complex, mission-critical industries drive business transformation Hitachi, Ltd. (TSE:6501, “Hitachi”) today announced plans to launch Hitachi Digital Inc. (“Hitachi Digital”), a new global digital services company, on April 1, 2027, in accordance with the integration plan announced in January 20261. Hitachi Digital will unite Hitachi’s U.S. subsidiaries, GlobalLogic Inc. (“GlobalLogic”) and Hitachi Digital Services LLC (“Hitachi Digital Services”) under a single company using the Hitachi brand, with a brand campaign launching today. Hitachi Digital will steadily guide enterprise digital transformation at the convergence of cloud, data and AI toward concrete business results. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261027531796/en/ Hitachi unveils a new global di
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