Business Wire

Emendo Biotherapeutics’ next generation CRISPR gene editing technologies achieve breakthrough results with allele-specific approach for ELANE-related Severe Congenital Neutropenia as presented at ASGCT Annual Meeting

Share

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.

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Media:
Shahar Avni
Business Development Specialist
shahara@emendobio.com

About Business Wire

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

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

ADVA CEO Brian Protiva to step down, CTO Christoph Glingener appointed as successor6.7.2022 23:45:00 EEST | Press release

ADVA (FSE: ADV) today announced that Brian Protiva is to step down as CEO. After a transition period of several weeks, Protiva will assume the role of vice chairman of Acorn HoldCo, Inc. (the holding company of Adtran and ADVA). Christoph Glingener will be appointed ADVA’s new CEO and will also continue as the company’s CTO. During Protiva’s 25-year tenure as chief executive officer, he successfully steered the company from a small start-up to an industry leader in metro WDM and Ethernet access devices. As ADVA is about to complete its business combination with Adtran, Protiva will support the further unification of the companies over the coming months. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220706005829/en/ Brian Protiva, CEO, ADVA (Photo: Business Wire) “Working side-by-side with Brian for eight years, I was privileged to see one of the industry‘s true leaders develop a world-class company. During his 25 years as A

MSCI Schedules Earnings Call to Review Second Quarter 2022 Results6.7.2022 21:30:00 EEST | Press release

MSCI Inc. (NYSE: MSCI), a leading provider of critical decision support tools and services for the global investment community, announced today it will release its results for the second quarter 2022 on Tuesday, July 26, 2022, before the market opens. A copy of the earnings release, as well as an earnings presentation and a quarterly update, will be made available on MSCI's Investor Relations website. MSCI's senior management will review the second quarter 2022 results on Tuesday, July 26, 2022 at 11:00 AM Eastern Time. To listen to the live event via webcast, visit the events and presentations section of MSCI's Investor Relations website, https://ir.msci.com/events-and-presentations or to join via telephone, please register yourself at https://ir.msci.com/events-and-presentations. Upon registration, telephone participants will receive a confirmation email detailing how to join the conference call, including a dial-in number and a unique participant pin that can be used to access the c

Esri Recognizes Italian Rail Agency for Modernizing Infrastructure6.7.2022 16:50:00 EEST | Press release

Last year, the Italian rail agency Rete Ferroviaria Italiana (RFI) built an application using geographic information system (GIS) technology from Esri, the global leader in location intelligence. Called StationLAND, the application is designed to make railway stations the centerpieces of their communities by increasing connectivity to the stations themselves within towns and cities. StationLAND improves the understanding of how and why rail stations work and how they can work better. StationLAND was designed to address a very specific need. "In Italy, the railways are one of the things that work best. What lagged was our stations," said Luigi Contestabile, RFI head of facilities, who also led the team that built the application. As a result, RFI sought to use technology to understand how rail stations could be better connected to their communities, thereby encouraging more rail travel. "The stations' centricity in our planning is fundamental to developing the sustainable intermodality

Globality Named Best Innovative Provider at Trophées des Achats Procurement Awards6.7.2022 16:42:00 EEST | Press release

Globality, the company transforming the way global enterprises buy services with its leading AI-powered digital solution, was recently named “Lauréat d’Or”, or “Best Innovative Provider”, at the prestigious Trophées des Achats awards ceremony in Paris. Organised annually since 2007 by Republik, the Trophées des Achats is regarded as a leading procurement event in France with more than 900 procurement professionals gathering each year to celebrate the most innovative technology and solutions transforming the procurement industry. More than 60 companies competed across three categories with the Innovative Provider category rewarding the digital solutions which are improving the performance of procurement teams at leading global organizations. Globality received its award from the Chief Procurement Officer (CPO) of France Television, Ahlem Hamdi and Bertrand Pouilloux, CPO of Enedis. Other members of the jury included Veolia CPO Sophie Gour, Yasser Balawi, CPO at Sodiaal, Siemens CPO Oliv

BigCommerce Broadens Global Footprint into Denmark, Norway, Sweden, Austria and Peru to Empower Merchants to Scale and Grow Their Online Businesses6.7.2022 15:00:00 EEST | Press release

BigCommerce (Nasdaq: BIGC), a leading Open SaaS ecommerce platform for fast-growing and established B2C and B2B brands, today announced it has grown its European presence into Austria, Denmark, Norway and Sweden, and its Latin American presence into Peru. With its latest expansions and fully localized tech and agency partner ecosystem, BigCommerce will enable merchants of all sizes and complexities to build and scale their online businesses to deliver seamless shopping experiences to local and cross-border customers and drive revenue. “BigCommerce’s arrival into new DACH, Nordic and Latin American markets affords a localized, powerful and scalable open SaaS ecommerce platform to merchants in these regions that have not been previously offered these core tenets in one solution,” said Meghan Stabler, senior vice president at BigCommerce. “With our proven enterprise-grade ecommerce platform, merchants of all sizes will have the disruptive tools and innovative functionalities — from headle

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