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
The Estée Lauder Companies Announces Expanded Roles for Brian Franz and Amber English8.9.2026 23:05:00 CEST | Press release
Appointments Support the Company's Ongoing Transformation Through Beauty Reimagined and Continued Focus on Consumer-Centric Growth The Estée Lauder Companies Inc. (NYSE: EL) today announced expanded leadership roles for two members of its Executive Team. Brian Franz has been appointed Chief Technology & Transformation Officer,expanding his responsibilities to include leading the company's enterprise-wide transformation. Amber English has been named President, Digital & Online, The Americas and Global Amazon Lead, assuming new enterprise-wide responsibility for The Estée Lauder Companies’ global relationship with Amazon. Both will continue to report directly to Stéphane de La Faverie, President and Chief Executive Officer, and remain members of The Estée Lauder Companies’ Executive Team. Both appointments are effective immediately. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260908417554/en/ Brian Franz, Chief Technology &
Samsung and Mistral AI Announce Strategic Partnership for Intelligence-Driven Semiconductor Infrastructure8.9.2026 18:15:00 CEST | Press release
Collaboration will focus on development and implementation of on-premises AI technologies across Samsung’s chip operationsSamsung makes lead investment in Mistral’s funding round Samsung Electronics Co., Ltd., today announced it has entered into a strategic partnership with Mistral AI to enhance its semiconductor engineering and manufacturing capabilities that will reinforce its leadership in AI chip technologies. The announcement of the partnership was made during the state summit held in Paris between South Korea and France. Under the agreement, Samsung will integrate Mistral’s AI services and solutions—including its flagship large language model, Mistral Large—across its semiconductor operations to develop customized on-premises AI models optimized for intelligence-driven infrastructure. Mistral’s powerful AI platform is expected to provide Samsung with a unique stack of tools that will transform how semiconductors are designed and manufactured. The on-premises enterprise solutions
Sensereo Airo Wins IFA Berlin Innovation Award8.9.2026 17:00:00 CEST | Press release
Prestigious Accolade Recognizes Sensereo’s Next-Generation Approach to Creating Healthier, Smarter Indoor Environments Sensereo, an environmental intelligence company developing next-generation sensing products for safer, healthier and smarter living, was presented with the prestigious IFA Berlin Innovation Award for its Airo modular indoor air quality system. The award was presented during IFA Berlin on Thursday, Sept. 4, recognizing Airo for its innovative approach to helping consumers better understand and manage the quality of their indoor environments. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260906292649/en/ Winner of a 2026 IFA Innovation Award, Airo is Sensereo’s flagship environmental intelligence platform, combining advanced sensing technology with a modular design that allows users to monitor multiple aspects of indoor air quality and environmental conditions through a single, consumer-friendly system. Desig
Verdantis Rebrands as the AI Super-Agent for MRO, Sets Sights on AI-Native Enterprise Asset Management8.9.2026 16:06:00 CEST | Press release
Nine AI agents now run the MRO decision chain for Fortune 500 plants; new tagline “Anticipate. Resolve. Learn.” Verdantis, the AI company for spare parts and MRO inventory optimization in asset-intensive industries, today unveiled its new brand identity as the AI Super-agent for MRO and announced it is building toward a fully AI-native Enterprise Asset Management (EAM) platform. Verdantis’s super-agent orchestrates nine specialized AI agents across the decisions that decide plant uptime: spare parts, criticality, demand, reorder, and work order planning. These decisions set working capital, maintenance spend, and whether a critical asset runs and, in most plants, they are still made in spreadsheets and disconnected systems. The positioning is for the teams who own uptime - maintenance, reliability, and MRO supply chain leaders in oil and gas, mining, utilities, chemicals, and manufacturing. And it reflects an AI-first architecture: agents decide and act by default; people supervise, ov
HyperLight Welcomes VentureTech Alliance to Series C, Deepening TFLN Integration Across the Semiconductor Ecosystem8.9.2026 16:00:00 CEST | Press release
HyperLight Corporation ("HyperLight"), a leader in thin-film lithium niobate (TFLN) photonics, today announced that VentureTech Alliance ("VTA") has joined its Series C financing round. VTA is a venture investment firm focused on the semiconductor industry, with a portfolio built across the semiconductor technology stack — including circuit design, foundry-adjacent process technology, EDA, advanced packaging, and photonics. VTA's participation extends the ecosystem alignment that defined the round, which brought together investors spanning silicon ICs, foundry manufacturing, electronics manufacturing services, networking, and global infrastructure capital. With VTA's addition, the syndicate now reaches further into the semiconductor manufacturing and design-enablement layers on which high-volume photonics production depends. "The transition to TFLN is not a component decision — it is an ecosystem decision," said Mian Zhang, CEO of HyperLight. "Getting TFLN into volume requires the proc
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