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
Kubota Vision Inc. Signs Supply and Licensing Agreement with Laboratoires KÔL for Stargardt Disease Treatment Candidate Under Compassionate Use4.5.2026 17:16:00 CEST | Press release
Kubota Vision Inc. (“Kubota Vision”), a wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Minato-ku, Tokyo, Japan) announced signing of a Supply and Licensing Agreement with Laboratoires KÔL (Clermont-Ferrand, France) for the provision of a Stargardt disease (STGD1) treatment candidate under compassionate use authorization. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260414590785/en/ The purpose of this Agreement is for Kubota Vision and KÔL to collaborate on providing “Emixustat” for treatment of Stargardt Disease (STGD1) through compassionate use authorization in France. Kubota Vision will exclusively manufacture and supply Emixustat final products to KÔL and KÔL will have exclusive rights to distribute Emixustat in France under compassionate use access. Ryo Kubota, Chairman, President, and CEO of Kubota Pharmaceutical Holdings, stated, “We are extremely pleased to enter into this Agreement with Labor
Incentive Market Set to Surpass $80B by 2030s — IMA Launches Tools to Help the Industry Keep Pace4.5.2026 16:28:00 CEST | Press release
With combined business spend on loyalty, employee recognition, and channel incentives projected to exceed $80 billion by the 2030s, the incentive industry is entering a period of rapid scale and strategic pressure. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260422729016/en/ In response, the Incentive Marketing Association (IMA) launched a new suite of resources designed to help incentive professionals navigate an environment that’s becoming more specialized, fragmented, and central to business performance. “It’s not just more money coming in—it’s more complexity,” said Vince Chiofolo, President of the Incentive & Engagement Solution Providers (IESP). “Buyers are demanding programs that evolve fast, integrate deeply, and feel personal at scale. These new tools were built for that.” IMA’s Suite of Tools Includes: New Industry Report — Inside the Incentive Buyer’s Mind Drawn from insights of 50+ program owners, the report f
Tigo Energy Breaks Global Growth Benchmark; Boosts U.S. Energy Feature in Predict+4.5.2026 15:00:00 CEST | Press release
Advanced energy prediction platform adds real-time energy pricing for North American Independent System Operator (ISO) customers, accelerates meters-connected growth. Tigo Energy, Inc. (NASDAQ: TYGO) (“Tigo” or “Company”), a leading provider of intelligent solar and energy solutions, today announced that the Predict+ platform now offers integrated real-time spot market pricing for ISO customers in the United States. Predict+ provides utilities with deep insights into grid demand, renewable generation, and energy market dynamics, enhancing the precision, scalability, and robustness of energy forecasting to up to 97.5% accuracy through machine learning and artificial intelligence. For energy providers, Predict+ helps streamline operations, reduce volatility, and maximize performance. Predict+ empowers utility operators to adapt to real-world demand challenges when balancing renewable and baseload generation sources, particularly during extreme weather events and market disruptions. On th
Airship Announces 2026 Elevate Customer Experience Award Winners4.5.2026 15:00:00 CEST | Press release
Southwest Airlines, The Economist, and OneFootball among brands recognized at Elevate’26 Customer Experience Summit for setting the standard in mobile-first and AI-centered customer experiences Airship, the mobile-first customer experience company, today announced the winners of the 2026 Elevate Customer Experience Awards. Global brand leaders from The Economist, Southwest Airlines, and OneFootball were among those honored at the Elevate’26 Customer Experience Summit. The Elevate Awards spotlight global companies that have developed exceptional mobile-first customer experiences for their customers, created innovative campaigns, leveraged AI agents to reach new levels of productivity, and achieved real business results. "Evolving mobile-first consumer behaviors and unprecedented advancements in AI are driving a seismic shift that is reshaping how brands interact with their customers," said Brett Caine, CEO, Airship. "The 2026 Elevate Award winners aren’t just adapting, they’re leading t
Alnylam to Present New Analyses at Heart Failure 2026 Underscoring Consistent Clinical Profile of Vutrisiran in ATTR-CM4.5.2026 14:30:00 CEST | Press release
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced it will present new data analyses for vutrisiran in patients with the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) at Heart Failure 2026, a scientific congress of the European Society of Cardiology, taking place May 9-12, 2026, in Barcelona, Spain. ATTR-CM is a heterogeneous and rapidly progressive disease, and patients often present with complex clinical profiles. At Heart Failure 2026, Alnylam will present multiple new analyses from the Phase 3 HELIOS-B study of vutrisiran, which continue to support its use as a first-line treatment for patients with ATTR-CM, and further characterize its clinical profile across patients with a high disease burden, and in the context of concomitant therapies. Vutrisiran is the first and only transthyretin (TTR) silencer for ATTR-CM that delivers rapid knockdown of TTR at the source. Additional analyses being pr
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