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
One Biosciences Receives BOOST Funding From Paris-Saclay Cancer Cluster to Advance the First Single-Cell Assay Approach for ADC Therapies22.6.2026 22:15:00 CEST | Press release
One Biosciences, a techbio company pioneering clinical-grade single-cell tumor profiling, today announced financial support via a Paris-Saclay Cancer Cluster (PSCC) BOOST grant to develop the first single-cell assay for antibody-drug conjugates (ADC) therapies in oncology. The rapid growth of ADCs is driving demand for more sophisticated biomarker strategies. By capturing tumor complexity at cellular resolution, single-cell profiling has the potential to enhance patient selection and support the development of next-generation ADCs. Ultimately, it will help match the right patients to the right therapies, maximizing therapeutic benefit. The PSCC BOOST-funded project seeks to bring a new level of precision to ADC development. It will be conducted in collaboration with Adcytherix, a clinical-stage biotech company developing differentiated ADCs for cancers with high unmet medical need. The collaboration will combine Adcytherix’s expertise in ADC development with One Biosciences’ AI-powered
Interactive Brokers Expands AI Integration Capabilities – Adding ChatGPT and Grok to Its Growing Suite of Agentic Trading Tools22.6.2026 16:00:00 CEST | Press release
Additional Asset Classes Available Including Options and Futures Interactive Brokers (Nasdaq: IBKR), an automated global broker, today announced the expansion of its agentic trading capabilities with the addition of ChatGPT and Grok, further broadening a growing ecosystem of AI platform integrations that began with Claude. Available through certified AI connector marketplaces across multiple leading platforms, these integrations allow clients to research, analyze, and generate instructions with speed and efficiency to uncover new trading and investing opportunities instantly. With this release, Interactive Brokers also extends the selection of products available for order instructions to include support for options, futures and futures options in addition to equities and ETFs. “We continue to see growing interest from investors in using artificial intelligence as a more natural way to interact with financial markets,” said Milan Galik, Chief Executive Officer of Interactive Brokers. “A
IQM Named Among Quantum Collaborators in HPE's New Hybrid Quantum-HPC Platform22.6.2026 15:22:00 CEST | Press release
The collaboration will advance practical and scalable hybrid classical-quantum applications. This follows IQM's first on-premises quantum computer installation in the United States, at Oak Ridge National Laboratory. IQM has sold 23 quantum systems globally to date and intends to list on Nasdaq in the United States by mid 2026. Hewlett Packard Enterprise has named IQM Quantum Computers among the companies collaborating on its hybrid classical-quantum computing platform, announced at HPE Discover Las Vegas. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260622035759/en/ IQM Radiance quantum computer at HPE Discover Las Vegas 2026 IQM, a global leader in full-stack superconducting quantum computers, is contributing superconducting quantum processor technology to the effort, in which HPE is integrating multiple quantum modalities with its Cray supercomputing infrastructure. This collaboration strengthens IQM's global expansion p
Return Abuse Goes Mainstream as Nearly Half of Consumers Use AI in Return Claims, New Riskified Research Finds22.6.2026 14:30:00 CEST | Press release
New global study shows normalization of strategic returns, widening regional differences, and rapid AI adoption reshaping ecommerce post-purchase risk Riskified (NYSE: RSKD), a global leader in ecommerce fraud detection and risk intelligence, today released a new global report, “Rewriting the Rules on Returns”, exploring how consumer attitudes and behaviors around ecommerce returns are evolving in the age of artificial intelligence (AI). The Riskified-commissioned study, conducted by eTail Insights, is based on a survey of 2,091 consumers across seven countries, alongside in-depth interviews with senior leaders from many of the largest retail companies in the world. The research finds that return abuse behaviors are increasingly normalized, while nearly half of consumers already use generative AI tools to assist with return or refund claims. At the same time, merchants are responding by tightening return policies, shortening return windows, and deploying advanced AI detection to better
Forrester Unveils Agendas For 2026 Technology & Innovation Forums In North America And EMEA22.6.2026 14:00:00 CEST | Press release
The Forums will equip technology leaders with the capabilities needed to operationalize AI and build high-performing IT organizations that deliver measurable business outcomes Forrester (Nasdaq: FORR) today announced the full conference agendas for its Technology & Innovation Forum Central (Austin, September 14–15, 2026), Technology & Innovation Forum EMEA (London, September 30–October 1, 2026), and Technology & Innovation Forum East (New York City, November 4–5, 2026). This year’s theme, “The IT Singularity,” dives into the new AI era in which AI has become ubiquitous and no longer a competitive advantage on its own. To succeed, technology leaders including chief information officers, chief data officers, and chief AI officers need to integrate AI into their operating models, modernize software development and IT operations, strengthen data and governance practices, and align technology investments with business strategy. Across the Forums, tech leaders and their teams will learn how
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