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
Special Olympics Airlift Takes Flight Nationwide; Dove 1 Arrives at St. Paul Downtown Airport19.6.2026 17:09:00 CEST | Press release
Approximately 130 Cessna, Beechcraft and Hawker aircraft and volunteer pilots mobilize to transport more than 800 Special Olympics athletes and coaches to the 2026 Special Olympics USA Games The 2026 Special Olympics Airlift officially took flight today as all participating Cessna, Beechcraft and Hawker aircraft, known as Doves, departed from airports across the country. Dove 1 for arrival day, a Cessna Citation Latitude generously operated by Prent Corporation, landed at St. Paul Downtown Airport (STP) carrying Special Olympic athletes and delegation members, signaling the start of Airlift arrivals for the Special Olympics USA Games. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260619085293/en/ Special Olympics Airlift takes flight nationwide; Dove 1 arrives at St. Paul Downtown Airport (Photo credit: Textron Aviation). The arrival signals the start of the world’s largest cumulative peacetime airlift spanning more than 40
Record Currency Management Participates in Innovative European Bank for Reconstruction and Development (EBRD) -Backed Mongolian Tugrik Transaction19.6.2026 16:53:00 CEST | Press release
Record Currency Management Ltd (RCM), subsidiary of London-listed Record plc (Record Financial Group), is pleased to announce its participation in an innovative local currency bond transaction issued by the European Bank for Reconstruction and Development (EBRD), supporting the development of Mongolia's capital markets while providing institutional investors with access to attractive frontier market opportunities. RCM is the UK currency management arm of Record Financial Group, the London-listed specialist investment group managing USD 115 billion of assets on behalf of institutional clients worldwide. Record's client base comprises pension funds, foundations, sovereign institutions and other asset managers, with whom the Group has built long-standing relationships through its focus on bespoke investment and risk management solutions. Headquartered in London, Record has offices in Hamburg, Zurich, Zug, New York, and Hong Kong. The investment forms part of Record Financial Group's broad
Plasma One Launches to Herald a New Era of Stablecoin Banking19.6.2026 14:29:00 CEST | Press release
Plasma has today announced the launch of Plasma One, its flagship financial product designed to make digital dollars usable for everyday spending, sending and earning. Plasma One is designed to make stablecoins feel like money, only better - more accessible, reliable and efficient. For years, stablecoin adoption has been held back by a fragmented system and poor user experience. We have seen wallets in one place, exchanges in another, and costly off-ramps standing between digital dollars and daily life. Plasma One brings that experience together in a single app, giving users a simple way to spend, send and earn with stablecoins from one account with zero fees. Headquartered in London, the city that gave rise to Revolut, Wise and Monzo, Plasma is making a bigger bet, that the next generation of consumer banking will be built on stablecoin rails, not legacy banking infrastructure. Underneath Plasma One is the Plasma Network, Plasma’s own blockchain, purpose-built to move stablecoins inst
IQM Appoints Craig Ciesla, Former Illumina VP, as CTO; Inés de Vega Becomes Chief Scientist19.6.2026 09:00:00 CEST | Press release
Ciesla is a seasoned deep tech executive with more than 25 years of experience delivering products across industries — from startups to Fortune 500 companies. De Vega brings more than 20 years of experience advancing quantum technologies from fundamental research to intellectual property and industrial applications. IQM Quantum Computers, the global leader in superconducting quantum computers, today announced the appointments of Dr. Craig Ciesla as Chief Technology Officer (CTO) and Dr. Inés de Vega as Chief Scientist, as she transitions from her role as Vice President of Quantum Solutions, deepening IQM´s leadership as the company prepares for its planned Nasdaq listing through a merger with Real Asset Acquisition Corp. (Nasdaq: RAAQ). This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260619693929/en/ From left to right: Dr. Inés de Vega, Chief Scientist, and Dr. Craig Ciesla, Chief Technology Officer. Ciesla, an experienced d
Teijin Receives Approval for Additional Indications of XEOMIN® for Cervical Dystonia and Blepharospasm in Japan19.6.2026 09:00:00 CEST | Press release
Teijin Pharma Limited, the core company of the Teijin Group’s healthcare business, and Merz Therapeutics GmbH, a leading player in neurology-focused specialty pharma, jointly announced today that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved the additional indications for XEOMIN® (incobotulinumtoxinA) for the treatment of cervical dystonia and blepharospasm. This approval represents the fourth and fifth indications for XEOMIN® authorized in Japan, alongside its existing approvals for the treatment of upper limb spasticity, lower limb spasticity and chronic sialorrhea. Cervical dystonia is a focal dystonia characterized by involuntary contractions of the neck muscles, resulting in abnormal or repetitive movements and postures.1 Blepharospasm is also a focal dystonia, characterized by excessive involuntary contractions of the muscles surrounding the eye.2 Both conditions significantly impact patients’ quality of life. The approval by MHLW is based on Phase III clinic
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