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
Incyte Data to Be Highlighted in Four Rapid Oral Presentations at the European Society for Medical Oncology (ESMO) Congress 2026 Support Efforts to Improve Outcomes in Difficult-to-Treat Cancers17.7.2026 19:10:00 CEST | Press release
Rapid oral presentations will highlight new Phase 1 data across Incyte’s solid tumor portfolioBoth INCB161734, an investigational, potent, selective and orally bioavailable KRAS G12D inhibitor, and INCA338901, a TGFβR2×PD-1 bispecific antibody, are being evaluated in ongoing Phase 3 programs as first-line treatments for patients with advanced pancreatic ductal adenocarcinoma (PDAC) and microsatellite stable (MSS) colorectal cancer, respectively Incyte (Nasdaq:INCY) today announced that it will highlight data from several programs in its oncology portfolio in six presentations at the European Society of Medical Oncology (ESMO) Congress 2026, being held October 23 - 27, 2026, in Madrid. “The data at ESMO will further illustrate Incyte’s commitment to advancing innovation for patients with cancer,” said Pablo J. Cagnoni, M.D., President, Incyte and Global Head of Research and Development. "Among the presentations are important updates from our KRAS G12D inhibitor in advanced pancreatic ca
STL Expands Its Optical Connectivity Portfolio in the US with the CONCAT Solution17.7.2026 14:24:00 CEST | Press release
STL Optical Connectivity NA, LLC, (STLOC), a U.S. subsidiary of STL (Sterlite Technologies Ltd.) [NSE: STLTECH], a leading connectivity solutions provider for AI-ready digital infrastructure, today announced that it launched its new advanced FTTH solution, CONCAT, after successfully completing field trials on the networks of one of the largest telecom service providers in the United States. Following successful field validation, CONCAT is now available to digital infrastructure providers seeking faster fiber rollouts with reduced deployment complexity and labor. It enables up to 71% in labor cost savings by eliminating most field splicing through factory-assembled, pre-connectorized fiber segments that deliver plug-and-play installation. CONCAT simplifies fiber deployment by shifting critical fiber preparation and termination into a controlled manufacturing environment ensuring consistent quality, faster installation, and lower operational risk in the field. CONCAT solution is ideal fo
Aqemia and Sanofi Expand Their Research Collaboration17.7.2026 12:30:00 CEST | Press release
A new target nomination and a milestone payment mark the next step of the multi-year partnership first announced in December 2023 Aqemia, the drug invention company combining generative AI and quantum-inspired physics to invent small molecule drugs, today announced the expansion of its multi-year research collaboration with the global pharmaceutical company, Sanofi. The expansion is marked by the nomination of a new therapeutic target and an additional payment. The collaboration, first announced in December 2023, makes Aqemia eligible to receive up to a total of $140 million in upfront and milestone payments across programs. It spans the drug discovery journey from the identification of the very first hits to the selection of a development candidate. Aqemia leverages Qemi, its proprietary physics-based generative AI platform, to design novel molecules addressing Sanofi’s targets of choice, working in close collaboration with Sanofi scientific teams. Sanofi leads wet lab research, devel
China's ~$900B Live-Commerce Market Now Approaches US E-Commerce Scale17.7.2026 12:30:00 CEST | Press release
The consumer habits reshaping global retail were built in the East — and most Western shoppers haven't yet adopted them. NIQ report shows that brands still treating live, social, and quick commerce as "emerging" risk being left behind. The center of gravity in global retail has shifted East. The formats now driving the fastest growth in global retail (live shopping, social commerce, and delivery in minutes) were pioneered and scaled in Asia, and most Western consumers have yet to adopt them. According to NIQ (NYSE: NIQ), a leading consumer intelligence company, in its global report The Commerce Revolution: Where East Meets West, the gap between East and West is still vast. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260717253489/en/ China´s USD 900bn live shopping boom now approaches the scale of the US E-commerce The scale is already substantial. China's live-commerce market alone was worth roughly $900 billion in 2025,
Ant International’s Alipay+ Connects Argentina's National QR Payment Scheme via PVS,Enabling for Cross-Border Digital Payment Nationwide at Millions of Merchants17.7.2026 12:17:00 CEST | Press release
Alipay+, a global digital payment gateway under Ant International, today announced that it will enable global travellers to make QR code payments at millions of merchants across Argentina through integration with the country's national QR payment scheme Transferencias 3.0, in partnership with PVS, a fintech company specialized in developing customized payment solutions in Latin America. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260717276406/en/ By connecting to Argentina's national QR payment scheme, Alipay+ now enables global travellers in the country to make convenient QR code payments at merchants nationwide. This service helps to enhance global travellers' travel experiences in Argentina, allowing them to pay seamlessly at restaurants, malls and tourist attractions. Using an Alipay+ partner payment app, they can now scan the national QR code displayed at all merchants to make cross-border payments across Argentina,
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