Cellectis Presents Non-Viral Gene Editing and Base Editing Innovation at the ASGCT Annual Meeting
Cellectis unveiled groundbreaking research data on gene-editing technologies at the upcoming Society of Gene and Cell Therapy annual meeting in New Orleans (May 13-17, 2025). The company presented two significant innovations:
1. TALEN-Mediated Non-viral Transgene Insertion:
- Successfully combined TALEN gene editing with non-viral delivery methods
- Demonstrated high efficiency in T-cells and blood stem cells
- Showed better cell fitness and stability compared to traditional viral methods
- Addresses challenges of viral methods like manufacturing limits and toxicity risks
2. TALE Base Editors (TALEB) Development:
- Created new method for precise C-to-T DNA conversion
- Achieved efficient editing without DNA strand damage
- Showed no detectable off-target editing in primary cells
- Developed system to assess editing efficiency across different sequences
These breakthroughs could advance treatments for cancer, autoimmune diseases, and genetic disorders. The complete research data will be available on Cellectis' website when the event begins.
Cellectis ha presentato dati rivoluzionari sulla tecnologia di editing genico durante il prossimo incontro annuale della Society of Gene and Cell Therapy a New Orleans (13-17 maggio 2025). L'azienda ha illustrato due innovazioni importanti:
1. Inserimento di transgene mediato da TALEN senza virus:
- Combinazione riuscita della tecnologia TALEN con metodi di somministrazione non virali
- Alta efficienza dimostrata in cellule T e cellule staminali del sangue
- Maggiore integrità e stabilità cellulare rispetto ai metodi virali tradizionali
- Supera i limiti dei metodi virali come problemi di produzione e rischi di tossicità
2. Sviluppo degli editor basati su TALE (TALEB):
- Nuovo metodo per la conversione precisa del DNA da C a T
- Editing efficiente senza danneggiare il filamento di DNA
- Nessuna modifica off-target rilevabile nelle cellule primarie
- Sistema sviluppato per valutare l'efficienza dell'editing su diverse sequenze
Questi progressi potrebbero migliorare i trattamenti per cancro, malattie autoimmuni e disturbi genetici. I dati completi saranno disponibili sul sito di Cellectis all'inizio dell'evento.
Cellectis presentó datos innovadores sobre tecnologías de edición genética en la próxima reunión anual de la Society of Gene and Cell Therapy en Nueva Orleans (13-17 de mayo de 2025). La compañía mostró dos avances significativos:
1. Inserción de transgenes mediada por TALEN sin uso de virus:
- Combinación exitosa de la edición genética TALEN con métodos de entrega no virales
- Alta eficiencia demostrada en células T y células madre sanguíneas
- Mejor viabilidad y estabilidad celular en comparación con métodos virales tradicionales
- Soluciona problemas de los métodos virales como límites en la fabricación y riesgos de toxicidad
2. Desarrollo de Editores de Bases TALE (TALEB):
- Nueva técnica para la conversión precisa de ADN de C a T
- Edición eficiente sin dañar la cadena de ADN
- No se detectaron ediciones fuera del objetivo en células primarias
- Sistema desarrollado para evaluar la eficiencia de edición en diferentes secuencias
Estos avances podrían impulsar tratamientos para cáncer, enfermedades autoinmunes y trastornos genéticos. Los datos completos estarán disponibles en el sitio web de Cellectis al inicio del evento.
Cellectis가 2025년 5월 13일부터 17일까지 뉴올리언스에서 열리는 유전자 및 세포 치료 학회 연례회의에서 혁신적인 유전자 편집 기술 연구 데이터를 공개했습니다. 회사는 두 가지 중요한 혁신을 발표했습니다:
1. TALEN 매개 비바이러스성 전사인자 삽입:
- TALEN 유전자 편집과 비바이러스 전달 방식을 성공적으로 결합
- T세포 및 혈액 줄기세포에서 높은 효율성 입증
- 전통적인 바이러스 방법보다 세포 건강도와 안정성 우수
- 제조 한계 및 독성 위험 등 바이러스 방법의 문제점 해결
2. TALE 기반 편집기(TALEB) 개발:
- C에서 T로의 정확한 DNA 변환을 위한 새로운 방법 개발
- DNA 가닥 손상 없이 효율적인 편집 달성
- 일차 세포에서 비표적(off-target) 편집 검출되지 않음
- 다양한 서열에서 편집 효율을 평가할 수 있는 시스템 개발
이러한 돌파구는 암, 자가면역 질환 및 유전 질환 치료를 발전시킬 수 있습니다. 전체 연구 데이터는 행사 시작 시 Cellectis 웹사이트에서 확인할 수 있습니다.
Cellectis a dévoilé des données de recherche révolutionnaires sur les technologies d'édition génétique lors de la prochaine réunion annuelle de la Society of Gene and Cell Therapy à la Nouvelle-Orléans (13-17 mai 2025). L'entreprise a présenté deux innovations majeures :
1. Insertion de transgène non virale médiée par TALEN :
- Combinaison réussie de l'édition génétique TALEN avec des méthodes de délivrance non virales
- Efficacité élevée démontrée dans les cellules T et les cellules souches sanguines
- Meilleure vitalité et stabilité cellulaire comparée aux méthodes virales traditionnelles
- Répond aux défis des méthodes virales tels que les limites de fabrication et les risques de toxicité
2. Développement des éditeurs de bases TALE (TALEB) :
- Création d'une nouvelle méthode pour la conversion précise de l'ADN de C en T
- Édition efficace sans endommager le brin d'ADN
- Aucune édition hors cible détectable dans les cellules primaires
- Système développé pour évaluer l'efficacité de l'édition sur différentes séquences
Ces avancées pourraient faire progresser les traitements contre le cancer, les maladies auto-immunes et les troubles génétiques. Les données complètes seront disponibles sur le site de Cellectis dès le début de l'événement.
Cellectis stellte bahnbrechende Forschungsergebnisse zu Gen-Editing-Technologien auf der bevorstehenden Jahreskonferenz der Society of Gene and Cell Therapy in New Orleans (13.-17. Mai 2025) vor. Das Unternehmen präsentierte zwei bedeutende Innovationen:
1. TALEN-vermittelte nicht-virale Transgen-Einfügung:
- Erfolgreiche Kombination von TALEN-Gen-Editing mit nicht-viralen Liefermethoden
- Hohe Effizienz in T-Zellen und Blutstammzellen nachgewiesen
- Bessere Zellfitness und Stabilität im Vergleich zu herkömmlichen viralen Methoden
- Bewältigt Herausforderungen viraler Methoden wie Produktionsgrenzen und Toxizitätsrisiken
2. Entwicklung von TALE-Baseneditors (TALEB):
- Neue Methode für präzise C-zu-T-DNA-Umwandlung entwickelt
- Effizientes Editieren ohne DNA-Strangschäden erreicht
- Keine nachweisbaren Off-Target-Effekte in Primärzellen
- System zur Bewertung der Editier-Effizienz über verschiedene Sequenzen entwickelt
Diese Durchbrüche könnten Behandlungen von Krebs, Autoimmunerkrankungen und genetischen Störungen voranbringen. Die vollständigen Forschungsdaten werden auf der Website von Cellectis zum Veranstaltungsbeginn verfügbar sein.
- Successful development of non-viral transgene insertion method that overcomes limitations of traditional viral methods, potentially reducing manufacturing costs
- High efficiency gene insertion demonstrated in T-cells and HSPCs with better cell viability
- Circular ssDNA shows improved HSPC fitness and more stable gene editing compared to traditional methods
- New TALE base editors (TALEB) technology shows high efficiency in C-to-T conversion with no detectable off-target editing in primary cells
- Technology still in research phase, not yet proven in clinical applications
- No commercial products or revenue generation mentioned from these developments
Insights
Cellectis advances non-viral gene editing technology, addressing key limitations in current gene therapy approaches with improved efficiency and safety profiles.
Cellectis's research demonstrates significant technical progress in gene editing methodologies, with two distinct approaches showing promise. The TALEN®-mediated non-viral transgene insertion represents an innovative solution to critical challenges facing gene therapy development.
Their data reveals that non-viral templates (both linear and circular ssDNA) achieve high-efficiency gene insertion in T-cells and hematopoietic stem cells while preserving cell viability. The circular ssDNA approach particularly stands out by enabling better maintenance of HSPC fitness and more stable gene editing compared to viral vector methods.
This advancement directly addresses key limitations of traditional viral delivery methods, including manufacturing constraints and payload size limitations. By eliminating viral components, this approach may potentially reduce manufacturing complexity while enhancing safety profiles.
The company's TALE base editor technology also shows promising results, with efficient C-to-T editing capability and high specificity. Their research has established methods to characterize and control editing outcomes, with data indicating no detectable off-target editing in primary cells.
These technologies expand Cellectis's gene editing toolkit, potentially enabling applications across multiple therapeutic areas including cancer, autoimmune diseases, and monogenic disorders. The research stage advancements demonstrate continued innovation in their foundational technology platform.
Cellectis strengthens gene editing platform with research advancements addressing key industry challenges, though commercial applications remain long-term prospects.
The research advancements outlined in Cellectis's presentation strengthen their technological foundation in the gene editing space. By developing non-viral delivery methods for gene insertion, the company addresses a critical industry bottleneck - the manufacturing and payload limitations associated with viral vectors.
Transcriptomic analysis and in vivo data demonstrating improved cell fitness with their circular ssDNA approach suggest potential advantages in creating more functional cell therapies. The technology could potentially improve manufacturing scalability while maintaining therapeutic effectiveness - a significant consideration for eventual commercialization.
Their TALE base editing technology further diversifies their technical capabilities beyond their core TALEN® platform. The high specificity demonstrated, with no detectable off-target editing in primary cells, addresses a key safety consideration for gene editing therapeutics.
These research-stage developments represent progress in overcoming fundamental challenges in gene therapy development, particularly for ex vivo cell engineering. While these advancements don't impact Cellectis's near-term financial performance, they strengthen the company's technological position and potential future therapeutic applications.
The presentation of these data at ASGCT highlights Cellectis's continued innovation in their core platform technology, supporting their focus on developing life-saving cell and gene therapies for conditions including cancer, autoimmune diseases, and monogenic disorders.
NEW YORK, April 28, 2025 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today unveils research data on TALEN®-mediated non-viral transgene insertion for advancing cellular and gene therapies, and advancements in genetic editing using TALE base editors (TALEB), at the Society of Gene and Cell Therapy (ASGCT) annual meeting, that will be held on May 13-17, 2025 in New Orleans.
The data are presented in two posters:
Title: TALEN®- Mediated non-viral Transgene Insertion for the Advancement of Cellular and Gene Therapies.
Cell and gene therapy approaches can use gene-editing tools and introduce transgenes to modify disease-associated genes, thus providing a potential therapeutic solution for a wide array of diseases.
In this work, Cellectis combines TALEN®-mediated gene editing with non-viral delivery of transgene for advancing cellular and gene therapies, and explores gene insertion-efficacy and cellular health using single-stranded DNA (ssDNA) for payload delivery in different cell types.
This innovative approach has the potential to address the challenges associated with traditional lentiviral viral methods or AAV-mediated transgene insertion such as manufacturing constraints, potential genomic toxicities or limited payload size.
Research data show:
- Non-viral methods for gene editing: TALEN® mediated gene editing combined with non-viral templates (linear and circular ssDNA) can be used for highly efficient gene insertion in T-cells as well as hematopoietic stem and progenitor cells (HSPCs) promoting viability and insertion specificity.
- Advantages of Circular ssDNA over viral vectors: Transcriptomic analysis and in vivo data demonstrate that CssDNA-mediated cell engineering allows better maintenance of HSPC fitness as well as more stable gene editing, compared to traditional viral donor template-mediated transgene delivery.
« The implementation of these gene-editing techniques holds significant potential for the development of next-generation therapies, aiming to provide alternative efficient, and safe therapeutic options for patients with cancer, autoimmune diseases, monogenic disorders, and various other conditions. » said Beatriz Aranda Orgilles, Ph.D., Associate Director – IO and business development analyst at Cellectis.
Title: High fidelity C-to-T editing with TALE base editors
TALE base editors (TALEB) are fusions of a transcription activator-like effector domain (TALE), split-DddA deaminase halves, and a uracil glycosylase inhibitor (UGI). The C-to-T class of TALEB edits double-stranded DNA by converting a cytosine (C) to a thymine (T) and does not involve DNA strand nick.
Cellectis has developed a method to characterize the efficiency of this conversion and examined various factors influencing TALEB activity. This method also takes advantage of a highly precise and efficient knock-in of ssODN in primary T cells to develop an assay to assess how the composition and spacer variations of target sequences affect TALEB activity/efficiency.
Research data show:
- Efficiency of C-to-T editing: TALEB enables efficient conversion of C to T. Variations in target sequences and surrounding bases affecting editing efficiency. An educated choice of the TALEB architecture further allows to control the editing outcome.
- Assessment of off-target editing risks: Studies have been conducted to evaluate off-target editing, showing no detectable editing in primary cells at previously described sites, highlighting the specificity of TALEB for potential therapeutic applications.
«It is inspiring to see the advancement of Cellectis’ TALE technology into a new tool that is available in our gene editing toolbox. Our ability to understand and fine-tune the editing capacity of TALE base editors has equipped us with another efficient and specific approach that can be used to support novel gene editing and gene therapy applications. » said Louisa Mayer, Ph.D., Scientist II and Supervisor – Innovation & Gene Editing at Cellectis.
Overall, the results of this study enhance the control and use of TALEB, allowing for the design of highly efficient and specific TALEB compatible with future therapeutic applications.
The abstracts are live on the ASGCT website. The posters will be available on Cellectis’ website the first day of the event.
About Cellectis
Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. The company utilizes an allogeneic approach for CAR T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to develop gene therapies in other therapeutic indications. With its in-house manufacturing capabilities, Cellectis is one of the few end-to-end gene editing companies that controls the cell and gene therapy value chain from start to finish.
Cellectis’ headquarters are in Paris, France, with locations in New York and Raleigh, NC. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more, visit www.cellectis.com and follow Cellectis on LinkedIn and X.
TALEN® is a registered trademark owned by Cellectis.
Cautionary Statement
This press release contains “forward-looking” statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as” can,” “potential,” “has the potential to,” “aiming to,” and “will” or the negative of these and similar expressions. These forward-looking statements are based on our management’s current expectations and assumptions and on information currently available to management. Forward-looking statements include statements about the potential of our innovation programs, in particular our gene editing innovations. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including those described in our Annual Report on Form 20-F as amended and in our annual financial report (including the management report) for the year ended December 31, 2024 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, which are available on the SEC’s website at www.sec.gov, as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.
For further information on Cellectis, please contact:
Media contacts:
Pascalyne Wilson, Director, Communications, + 33 (0)7 76 99 14 33, media@cellectis.com
Patricia Sosa Navarro, Chief of Staff to the CEO, +33 (0)7 76 77 46 93
Investor Relations contact:
Arthur Stril, Chief Financial Officer & Chief Business Officer, investors@cellectis.com
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FAQ
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What are the advantages of Cellectis (CLLS) circular ssDNA technology?
How effective is Cellectis TALE base editor (TALEB) for gene editing?
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