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
Nordson EFD Launches PICO Pµlse XP HV Jetting System to Meet Assembly Market Demand for Dispensing High-Viscosity Materials9.11.2026 16:00:00 CET | Press release
Dispensing Innovation delivers Precise Performance, Ease of Use and Automation Integration Nordson EFD,a Nordson company (NASDAQ: NDSN), today announced the launch of its PICO Pµlse® XP HV jetting system designed for extremely precise (XP) assembly applications utilizing high-viscosity (HV) materials like thick silicones, greases, and UV-cure adhesives. New formulations are rapidly entering the industrial marketplace due to evolutionary new product development across multiple industries including electronics, medical, and automotive. Nordson leveraged its core competencies and time-tested structural architecture to innovate the piezoelectric jetting system for non-contact precision dispensing of this emerging genre of viscous materials. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260921406991/en/ Caption 1: Engineered for performance, the PICO Pμlse XP HV jetting system excels in demanding applications, such as optical bo
Tashkent Makes History with Central Asia’s First Golden Globes Gala9.11.2026 14:42:00 CET | Press release
Two-time Academy Award winner Adrien Brody joins leading cultural figures as the inaugural Golden Globes Silk Road Tribute Gala brings the awards to the region for the first time Tashkent hosted the inaugural Golden Globes Silk Road Tribute Gala, bringing together internationally recognised actors, filmmakers and cultural figures from Central Asia, the Caucasus and beyond. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261109918777/en/ Tashkent Makes History with Central Asia’s First Golden Globes Gala (Photo credit: © Organising Committee of the Golden Globes Silk Road Tashkent) Guests of honour included Otabek Umarov, First Deputy Chairman of the National Olympic Committee of Uzbekistan, Vice President of the Olympic Council of Asia and Member of the FIA Senate, and Shakhnoza Mirziyoyeva, First Deputy Director of the National Agency for Social Protection, First Deputy Chairman of the National Paralympic Committee of Uzbeki
Latifa bint Mohammed Inaugurates ISEA20269.11.2026 14:30:00 CET | Press release
Her Highness Sheikha Latifa bint Mohammed bin Rashid Al Maktoum, Chairperson of Dubai Culture and Arts Authority, inaugurated the 31st International Symposium on Electronic/Emerging Art (ISEA2026 Dubai), held under the theme ‘ELYAH: Constellating Place, Data, and Identity.’ Bringing together artists, researchers and innovators from around the world, the symposium, in its largest-ever edition, explores how art, science and technology can expand creative expression, deepen cultural understanding, and shape the future of electronic and emerging art. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261109319593/en/ Burj Khalifa lights up to celebrate the opening of ISEA2026 Dubai (Photo: AETOSWire) Her Highness marked the inauguration by launching the interactive ELYAH show at ARTE MUSEUM Dubai. Inspired by Dubai’s celestial heritage, the experience combined light, music, performance and digital technology to explore the relations
Muslim World League’s “Minhaj” App Participates in Hajj Conference and Exhibition 2026 in Jeddah9.11.2026 14:04:00 CET | Press release
The “Minhaj” app, developed by the Muslim World League (MWL), is participating in the sixth edition of the Hajj Conference and Exhibition 2026, which commenced today in Jeddah under the patronage of the Custodian of the Two Holy Mosques, King Salman bin Abdulaziz Al Saud (may Allah protect him). Organized by the Ministry of Hajj and Umrah in cooperation with the Pilgrim Experience Program, one of the programs of Saudi Vision 2030, the event is being held from November 8 to 11 at the Jeddah Superdome under the theme “From Makkah to the World.” This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261109411972/en/ The pavilion of the Muslim World League (MWL) at the sixth edition of the Hajj Conference and Exhibition 2026 in Jeddah, Saudi Arabia. (Photo: AETOSWire) Minhaj’s participation in this international event aims to introduce its integrated Islamic digital ecosystem, launched from Makkah to serve Muslims worldwide. The app off
CAIRDAC Adds Operational Expertise to Its Board of Directors With the Appointment of Rinda Sama9.11.2026 13:44:00 CET | Press release
CAIRDAC, SA, a pre-revenue-stage medtech company developing ALPS™, a leadless, self-powered cardiac pacing system, today announced the appointment of Mr. Rinda Sama to its Board of Directors. His appointment adds deep manufacturing, quality and operational experience to the Board and follows the recent appointment of Raymond W. Cohen as Chairman. “Mr. Sama’s extensive experience in manufacturing, quality systems and operational scale-up for complex implantable devices will be extremely valuable to our Company as we prepare for clinical evaluation and the next stages of development,” said Edouard Fonck, Chief Executive Officer of CAIRDAC. Mr. Sama currently serves as CEO of Spiro Medical, a company focused on treating asthma employing a proprietary neuromodulation system. He previously served as Chief Operating Officer of publicly traded Axonics, Inc. where he was accountable for manufacturing and quality and the operational infrastructure that supported the company’s commercial growth.
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