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
Corona Global Named Most Valuable Beer Brand in Kantar BrandZ Rankings for Third Consecutive Year14.5.2026 06:01:00 CEST | Press release
AB InBev Holds 8 of the Top 10 Most Valuable Beer Brands Globally Corona global has been recognized as the most valuable beer brand in the world for the third consecutive year in Kantar's BrandZ 2026 Most Valuable Global Brands report, released today. Eight out of the top ten most valuable global beer brands belong to AB InBev (Brussel:ABI) (BMV:ANB) (JSE:ANH) (NYSE:BUD), according to the report ranking the best brands in the world. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260513211378/en/ In 2025, Corona led AB InBev’s performance, increasing revenue by 8.3% outside of its home market with double-digit volume growth in 30 markets, while Corona Cero delivered strong double-digit volume growth. As the brand celebrated its 100th anniversary, Corona launched its global “Corona 100” platform, including a multi-year sponsorship of a renowned concert at Copacabana Beach in Rio de Janeiro. Strong momentum continued in Q1 2026
The Bolivarian Republic of Venezuela Announces the Initiation of a Comprehensive Public Debt Restructuring Process13.5.2026 22:08:00 CEST | Press release
The Bolivarian Republic of Venezuela announces today its decision to initiate a comprehensive restructuring of its external public debt obligations. CONTEXT & RATIONALE Over the past decade, Venezuela's economy has been severely impacted by external shocks – from the end of the commodity super-cycle to foreign sanctions and COVID-19 – resulting in a sharp economic contraction, a protracted default since 2017, and a ballooning of public sector liabilities. The country nonetheless possesses a strong economic potential, including unique hydrocarbon and mineral endowments. A recovery is underway, and Venezuela is committed to accelerating it through a comprehensive reform agenda in support of growth, fiscal sustainability, monetary stabilization, and enhanced governance. Restoring public debt sustainability is central to this agenda: the current debt overhang constrains external financing, limits public investment capacity, and prevents full re-engagement with the international financial s
Boomi Announces Intent to Acquire Lunar.dev to Deliver Governed Agent Connectivity Across the Enterprise13.5.2026 21:00:00 CEST | Press release
Proposed acquisition extends Boomi’s AI gateway capabilities, enabling enterprises to govern massive volumes of traffic for production-ready AI Boomi, the data activation company for AI, today announced it has signed a letter of intent to acquire Lunar.dev, an innovator in AI and MCP gateway. The proposed acquisition is expected to enrich the Boomi Enterprise Platform and Boomi Connect with advanced capabilities to govern and scale AI usage across enterprise systems. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260513850557/en/ Boomi Announces Intent to Acquire Lunar.dev to Deliver Governed Agent Connectivity Across the Enterprise As organizations move from AI experimentation to production, controlling how agents and AI applications interact with LLMs at scale has become critical. Lunar.dev addresses this need with an AI gateway that delivers granular, policy-driven control over AI interactions, with the visibility, securi
BeOne Medicines’ BEQALZI™ (sonrotoclax) Approved by U.S. FDA as First and Only BCL2 Inhibitor for R/R Mantle Cell Lymphoma13.5.2026 19:27:00 CEST | Press release
BEQALZI is a foundational BCL2 inhibitor designed for greater potency and selectivity, with potential to improve efficacy, tolerability, and convenience versus others in the classApproval of BEQALZI marks the first new BCL2 inhibitor approved in a decade in the U.S. and the only BCL2 inhibitor approved in MCL, aiming to set a new standard of innovation BeOne Medicines Ltd. (“BeOne”) (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to BEQALZI™(bee-KAHL-zee; sonrotoclax), a foundational, next-generation BCL2 inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL), after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor. BEQALZI was designed to enhance BCL2 inhibition—with greater potency, selectivity, and a pharmacologic profile with potential to improve efficacy, tolerability, a
Boomi Unveils Innovations That Power the Agentic Enterprise13.5.2026 16:30:00 CEST | Press release
Orchestration capabilities simplify workflows, while advanced agent connectivity and an enhanced context layer activate data and scale AI Boomi, the data activation company for AI, today announced a major expansion of the Boomi Enterprise Platform at Boomi World 2026, introducing new capabilities across orchestrated agentic workflows, agentic engineering, governed agent connectivity, grounded agent context, and localized agent infrastructure. Together, these innovations are designed to power the agentic enterprise — where agents and humans work together to drive action and operationalize AI at scale. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260513996223/en/ Boomi Unveils Innovations That Power the Agentic Enterprise The enterprise has reached a defining moment as AI becomes the primary interface for work and MCP emerges as the new standard. While the shift toward a headless, agentic enterprise is inevitable, this visio
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