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
Xsolla Sponsors Game Camp France, Reinforcing Commitment to France’s Dynamic Games Ecosystem17.6.2026 15:47:00 CEST | Press release
Game Camp France supports regional developer communities across the French games ecosystem Xsolla, a global video game commerce company that helps developers launch, grow, and monetize their games, today announced its sponsorship of Game Camp France this 18-19 June 2026. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260617351463/en/ Graphic: Xsolla The French video game industry is widely recognized as one of the most vibrant in Europe, with revenue reaching approximately €5.8 billion in 2025. Rather than a centralized market, it is a patchwork of regional scenes, each with its own communities, creative identity, and business dynamics. Game Camp France plays a key role in bringing these diverse voices together, creating a centralized space for collaboration, exchange, and growth. By sponsoring Game Camp France, Xsolla is investing directly in the local ecosystem and the developers who power it. Xsolla will be on-site to eng
The Estée Lauder Companies and Jo Malone London Launch Experience That Turns Visual Taste into a Personalized Fragrance Wardrobe, exclusively on Pinterest17.6.2026 15:26:00 CEST | Press release
The Estée Lauder Companies Inc. (NYSE: EL) and Jo Malone London today announced the launch of Scent Scanner, a first-of-its-kind experience available exclusively on Pinterest and rolling out in the US and France. The experience translates the visual preferences people have expressed across their Pinterest boards into personalized Jo Malone London fragrance recommendations. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260617962807/en/ Building on the success of Jo Malone London's AI Scent Advisor, introduced in 2025, Scent Scanner offers a new way for consumers to find their personalized scent, shifting the starting point from words to images. While the AI Scent Advisor invited consumers to describe the scent they were seeking, Scent Scanner reads visual inspiration – imagery, color palettes, textures, destinations, rituals and aesthetics – to suggest fragrances suited to each individual. The custom Pinterest experience bri
Azul Addresses the Java Runtime Security Blind Spot Autonomous AI Can Now Exploit17.6.2026 15:00:00 CEST | Press release
Launches free JVM vulnerability risk assessment to give enterprises estate visibility before AI threat actors find the gaps Azul, the trusted leader in enterprise Java for today’s AI and cloud-first world, today launched a free JVM vulnerability risk assessment to address the blind spot that autonomous AI exploitation tools are increasingly able to find. With mean time to exploit (MTTE) collapsing from months to days or hours, the unmanaged Java estate has become an urgent enterprise security vulnerability. Azul’s assessment gives DevOps and SecOps teams complete visibility into the hidden risks embedded in the runtime of their Java estate before threat actors get there first, and is designed to complement the broader security, licensing and compliance solutions and services delivered by Azul’s trusted partners. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260617352753/en/ Azul's JVM Vulnerability Risk Assessment dashboard
Fortegra Strengthens Actuarial Leadership with Appointment of Anthony Katz as SVP, Reserving17.6.2026 15:00:00 CEST | Press release
The Fortegra Group, Inc., a global specialty insurer, today announced the appointment of Anthony Katz as Senior Vice President, Reserving. Mr. Katz will lead Fortegra’s team of professional actuaries, with responsibility for reserving, credit insurance, statistical reporting, and key initiatives including IFRS 17. “Anthony brings more than 30 years of actuarial expertise and a genuine commitment to building the capabilities our distribution partners depend on,” said Rick Kahlbaugh, Chief Executive Officer at Fortegra. “His background across reserving, actuarial transformation, and international markets will be instrumental as we continue to support our distribution partners.” A credentialed actuary holding FCAS, FSA, and MAAA designations, Mr. Katz brings more than 30 years of experience across reserving, pricing, and actuarial transformation. He has held senior leadership roles at ToaRe, Everest Re, Arch Insurance, Ernst & Young, and ACE, and most recently served as an independent con
Hitachi Expands Its Work With OpenAI to Accelerate AI-Driven Modernization and Cybersecurity17.6.2026 15:00:00 CEST | Press release
Hitachi and OpenAI’s Forward Deployed Engineer (FDE) will begin developing AI solutions for legacy system modernization, starting with financial institutions, aiming to expand across a wide range of industries. Through OpenAI’s “Trusted Access for Cyber,” Hitachi will strengthen its internal cybersecurity in practice and leverage the insights gained from this initiative to enhance its cybersecurity capabilities. Through this work, Hitachi will enhance its FDE capabilities in modernization and cybersecurity and further advance HMAX. Hitachi, Ltd. (TSE:6501, "Hitachi") today announced that it will expand its work with OpenAI*1 to accelerate AI-driven modernization and cybersecurity. The work combines OpenAI’s frontier AI technology with Hitachi’s domain expertise and know-how in supporting mission-critical systems and social infrastructure to accelerate AI transformation (AX) for customers and society. Working together, the two companies’ FDE teams will leverage OpenAI’s AI agent “Codex”
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