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
Cardiac Dimensions® Announces Three Major Publications Demonstrating Long-Term Durability, Real-World Performance, and Broad Patient Benefit of the Carillon Mitral Contour System®8.7.2026 16:05:00 CEST | Press release
New data highlight multi-center 5-year durability, 10-year survival, and the largest single-center experience across both HFrEF and HFpEF patients with functional mitral regurgitation Cardiac Dimensions, a leader in transcatheter therapies for heart failure, today announced the publication of three major manuscripts that together form the most comprehensive evidence base ever assembled for transcatheter indirect mitral annuloplasty. These publications span a five-year multi-center commercial registry, the largest single-center experience across both preserved and reduced ejection fraction, and long-term survival results through ten years. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260707656796/en/ Carillon Mitral Contour System® The Carillon Mitral Contour System is the only commercially available indirect mitral annuloplasty device, designed to treat a broad and expanding population of patients living with functional mi
First Randomized Controlled Trial Shows Promise of a Ketogenic Diet in Psychotic Disorders8.7.2026 15:00:00 CEST | Press release
Trial reports correlations between ketone levels and cognitive and psychiatric symptoms in individuals with schizophrenia-spectrum and bipolar-1 disorders Published today in Schizophrenia Bulletin, a first-of-its-kind randomized controlled trial (RCT) from researchers at the University of California, San Francisco (UCSF), and funded in part by the National Institute of Mental Health (NIMH), adds to growing literature on the potential benefit of a ketogenic diet for treating psychotic disorders. The study, which enrolled participants with schizophrenia-spectrum or bipolar-1 disorders, demonstrated rapid metabolic improvements with a ketogenic diet compared to diet-as-usual during an initial one-month RCT open-label phase. Furthermore, those who continued with the optional four-month single-arm ketogenic diet extension saw meaningful gains across metabolic, psychiatric, and cognitive measures. This press release features multimedia. View the full release here: https://www.businesswire.co
Teamily AI Publicly Launches Human+AI Social Platform to Make Building and Growing a Company Easy for Every Team8.7.2026 15:00:00 CEST | Press release
Teamily AI (https://Teamily.ai), together with its Agentic AI Infra platform TensorOpera AI (https://TensorOpera.ai), today announced the public launch of its Human+AI social platform, a product stack that has already served more than 5 million users around the world. Starting today in Palo Alto and rolling out simultaneously across dozens of countries, this first public launch opens the full stack to everyone with a single mission: to make building and growing a company easy for every person and every team. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260708584390/en/ "This is the moment we open our doors to the world," said co-founders Dr. Aiden Chaoyang He and Professor Salman Avestimehr. "We want every person and every team to be able to move from idea to product, from product to market, and from market to growth and investment, with an AI-native team by their side." At its core, Teamily AI enables humans and AI agents
Leo Cancer Care Raises $65M Series D to Scale Its Integrated Upright Cancer Care Platform8.7.2026 14:30:00 CEST | Press release
Oversubscribed round follows the world-first compact upright proton treatment at Stanford Medicine — funding a single upright platform that will span imaging and treatment across multiple radiation modalities. Leo Cancer Care, the medical technology company working to reinvent how patients are imaged and treated by designing systems around the body’s natural upright position, today announced the close of an oversubscribed $65 million Series D financing. The round was led by Silicon Valley’s Yu Galaxy and welcomes new investors including Eventide Asset Management, alongside continued support from the company’s existing investors. Leading cancer institutions are already adopting the upright approach. Stanford Medicine delivered the world’s first compact upright proton therapy treatment on 4 June 2026. Dana-Farber Cancer Institute and McLaren Health Care are among the institutions bringing the upright platform into their programmes — adoption that spans world-leading academic centers and
LUMI AI Factory Selects IQM to Deploy Advanced Quantum Computer, Accelerating Hybrid HPC and AI Development8.7.2026 14:00:00 CEST | Press release
The Halocene H4 quantum computer, named LUMI-IQ, will be delivered and installed in 2027 The system will be hosted at CSC – IT Center for Science in Finland and integrated into the LUMI AI Factory The system will provide researchers, industry innovators, and developers across Europe with a unique, advanced experimental platform where quantum computing and artificial intelligence converge The LUMI AI Factory, led by CSC – IT Center for Science, has selected IQM Quantum Computers (Nasdaq: IQMX) to deliver IQM Halocene H4, an advanced quantum computer aimed at accelerating hybrid high-performance computing, artificial intelligence, and quantum computing capabilities. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260708006791/en/ LUMI AI Factory selects IQM to deploy advanced quantum computer, accelerating hybrid HPC and AI development IQM Halocene H4 is the first and most advanced on-premises superconducting quantum computer o
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