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
Arc'teryx Introduces System 0™, a New Framework for the Future of Product Design and Circularity26.8.2026 15:00:00 CEST | Press release
Sperro SV, a multisport mountain-grade hardshell built with circular design principles, available September 4, 2026 Arc'teryx Equipment, the global design company specializing in technical high-performance apparel and equipment, today introduced System 0™, a new framework that reimagines how products can be designed, manufactured, used, repaired, and ultimately regenerated. System 0 represents the next evolution for sustainability in the outdoor industry, as it embeds circular thinking across an entire product lifecycle while maintaining the uncompromising performance standards that define the Arc’teryx brand. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260826686539/en/ Arc’teryx athlete, Craig Murray, wearing the Sperro SV, a multisport mountain-grade hardshell built with circular design principles, available September 4, 2026. The first product being introduced as part of the System 0 platform is the Sperro SV jacket, a
Xsolla Announces Five-Year Partnership With the Global Esports Federation26.8.2026 15:00:00 CEST | Press release
Partnership Represents a Structural Shift in Esports, Placing Xsolla's Infrastructure Behind GEF’s Entire Ecosystem, Spanning Commerce, Payments, Wallet, Loyalty, and Distribution Across Global Esports Games, Global Esports Tour, and 180+ Member Federations Xsolla, a global commerce company, today announced a landmark five-year strategic partnership with the Global Esports Federation (GEF), the world-convening body for esports. As GEF's exclusive partner for commerce, payments, wallet, loyalty, and distribution, Xsolla will power the entire GEF ecosystem, including the flagship Global Esports Games, pro-series Global Esports Tour, global GEFcon, and 180+ Member Federations across five continents. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260826948844/en/ Graphic: Xsolla The announcement comes as GEF marks 100 days to go to the Los Angeles 2026 Global Esports Games (GEGLA26), adding further momentum to the road to Los An
NABEP Appoints Sara Chouraqui as General Counsel26.8.2026 15:00:00 CEST | Press release
Company expands legal team as it prepares for significant growth North American Blue Energy Partners (NABEP) has appointed Sara Chouraqui as General Counsel, with Elizabeth Collery and Victoria Jacobson each joining as Deputy General Counsel. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260826168543/en/ Sara Chouraqui joins NABEP as its General Counsel, bringing decades of legal expertise. These additions significantly strengthen NABEP’s senior leadership team and legal expertise as the company improves its operations, increases production and develops new strategic and commercial partnerships. As General Counsel, Chouraqui will lead NABEP’s global legal function and advise the company’s leadership on its legal, regulatory and corporate priorities, including major transactions and partnerships, governance, compliance and the management of legal and regulatory risk across the company’s operations. Her experience navigating
Grafana Labs Crosses 10,000-Customer Milestone as AI Adoption Accelerates Growth Across the Platform26.8.2026 15:00:00 CEST | Press release
Grafana Labs was recently recognized as a Leader in the 2026 Gartner® Magic Quadrant™ for Observability Platforms for the third consecutive year, launched six AI-native products during its inaugural AI Week, and surpassed $600M in Annual Recurring Revenue. Grafana Labs, the company behind the open observability cloud, today announced it has crossed 10,000 customers worldwide and surpassed $600 million in annual recurring revenue, as organizations standardize on open observability for the AI era. Teams need AI to help them understand their own systems faster, especially as complexity and overhead remain the top observability concern cited in Grafana Labs' 2026 Observability Survey – a need reflected by the adoption of Grafana Assistant, Grafana Cloud's context-aware AI agent for exploring and troubleshooting observability data, now used by more than 18,000 organizations. Teams also need observability purpose-built for AI systems themselves: When teams move AI into production, models dri
FlightSafety International Reaches Agreement to Acquire Acron Technologies’ Commercial Training Solutions26.8.2026 15:00:00 CEST | Press release
Agreement will expand FlightSafety’s training and simulation capabilities in the global commercial aviation market FlightSafety International Inc. (FSI), a leading global provider of aviation training and simulation technology, today announced it has signed a definitive agreement with Acron Technologies, a portfolio company of TJC LP, to acquire its Commercial Training Solutions (CTS) business, including its Training Systems, Training Services, Aviation Academy, and Driver Training portfolio. The agreement marks a significant expansion of FSI’s capabilities in the commercial aviation market, broadening and complementing its simulator manufacturing capabilities and global training footprint. CTS brings over eight decades of experience in commercial aviation training and simulation. With a long-standing heritage in commercial airline simulator manufacturing, CTS has delivered more than 850 training devices throughout its history, with over 300 full-flight simulators currently in operatio
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