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Cellectis to Present Data on TALE-Base Editors and Non-Viral Gene Therapy at the ESGCT 31st Annual Congress

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Cellectis, a clinical-stage biotechnology company, will present pre-clinical data on TALE base editors (TALEB) and non-viral gene insertion at the European Society of Cell and Gene Therapy 31st annual congress in Roma, Italy, from October 22-25, 2024. Two posters will be showcased:

1. 'Controlling C-to-T editing with TALE base editors' by Alexandre Juillerat, Ph.D., on October 24. This study enhances understanding of TALEB and allows for the design of efficient and specific tools potentially compatible with therapeutic applications.

2. 'Circular Single-Stranded DNA Enables Efficient TALEN-Mediated Gene Insertion in Long Term HSC' by Julien Valton, Ph.D., also on October 24. This research demonstrates that circularizing ssDNA increases gene insertion rates in long-term HSCs and may enhance their engraftment capacity in preclinical murine models, advancing non-viral gene therapy.

Cellectis, un'azienda biotecnologica in fase clinica, presenterà dati pre-clinici su editori di base TALE (TALEB) e inserimento genico non virale al 31° congresso annuale della Società Europea di Terapia Cellulare e Genica a Roma, Italia, dal 22 al 25 ottobre 2024. Saranno esposti due poster:

1. 'Controllare l'editing C-to-T con gli editori di base TALE' di Alexandre Juillerat, Ph.D., il 24 ottobre. Questo studio migliora la comprensione di TALEB e consente la progettazione di strumenti efficienti e specifici potenzialmente compatibili con applicazioni terapeutiche.

2. 'Il DNA circolare a singolo filamento consente un'inserzione genica efficiente mediata da TALEN in HSC a lungo termine' di Julien Valton, Ph.D., anch'esso il 24 ottobre. Questa ricerca dimostra che la circolarizzazione dell'ssDNA aumenta i tassi di inserimento genico negli HSC a lungo termine e potrebbe migliorare la loro capacità di innesto in modelli murini preclinici, facendo progredire la terapia genica non virale.

Cellectis, una empresa de biotecnología en etapa clínica, presentará datos preclínicos sobre editores de bases TALE (TALEB) y inserción génica no viral en el 31° congreso anual de la Sociedad Europea de Terapia Celular y Génica en Roma, Italia, del 22 al 25 de octubre de 2024. Se exhibirán dos pósteres:

1. 'Controlando la edición C-to-T con editores de bases TALE' por Alexandre Juillerat, Ph.D., el 24 de octubre. Este estudio mejora la comprensión de TALEB y permite el diseño de herramientas eficientes y específicas potencialmente compatibles con aplicaciones terapéuticas.

2. 'El ADN circular de cadena sencilla permite una inserción génica eficiente mediada por TALEN en HSC a largo plazo' por Julien Valton, Ph.D., también el 24 de octubre. Esta investigación demuestra que la circularización de ssDNA aumenta las tasas de inserción génica en HSC a largo plazo y puede mejorar su capacidad de injerto en modelos murinos preclínicos, avanzando en la terapia génica no viral.

Cellectis는 임상 단계의 생명공학 회사로서, TALE 기반 편집기(TALEB)비 바이러스 유전자 삽입에 대한 전임상 데이터를 유럽 세포 및 유전자 치료 학회 제31회 연례 총회에서 이탈리아 로마에서 2024년 10월 22일부터 25일까지 발표할 예정입니다. 두 개의 포스터가 전시될 예정입니다:

1. 'TALE 기반 편집기로 C-to-T 편집 조절하기' - Alexandre Juillerat 박사, 10월 24일. 이 연구는 TALEB의 이해를 높이고 치료 응용에 잠재적으로 적합한 효율적이고 구체적인 도구의 설계를 가능하게 합니다.

2. '원형 단일 가닥 DNA가 TALEN 매개 유전자 삽입을 효율적으로 가능하게 한다' - Julien Valton 박사, 10월 24일. 이 연구는 ssDNA의 원형화가 장기 HSC에서 유전자 삽입률을 증가시키고 기본 연구용 마우스 모델에서 이식 능력을 향상시킬 수 있음을 보여주어 비 바이러스 유전자 치료의 발전에 기여합니다.

Cellectis, une entreprise de biotechnologie en phase clinique, présentera des données précliniques sur les éditeurs de base TALE (TALEB) et l'insertion génique non virale lors du 31ème congrès annuel de la Société Européenne de Thérapie Cellulaire et Génétique à Rome, en Italie, du 22 au 25 octobre 2024. Deux affiches seront présentées :

1. 'Contrôler l'édition C-to-T avec les éditeurs de base TALE' par Alexandre Juillerat, Ph.D., le 24 octobre. Cette étude améliore la compréhension des TALEB et permet la conception d'outils efficaces et spécifiques, pouvant être compatibles avec des applications thérapeutiques.

2. 'L'ADN single-brin circulaire permet une insertion génique efficace médiée par TALEN dans des HSC à long terme' par Julien Valton, Ph.D., également le 24 octobre. Cette recherche démontre que la circularisation de l'ADNss augmente les taux d'insertion génique dans les HSC à long terme et pourrait améliorer leur capacité d'engraftement dans des modèles murins précliniques, faisant avancer la thérapie génique non virale.

Cellectis, ein biopharmazeutisches Unternehmen in der klinischen Phase, wird beim 31. Jahreskongress der Europäischen Gesellschaft für Zell- und Gentherapie in Rom, Italien, vom 22. bis 25. Oktober 2024 präklinische Daten zu TALE-Basiseditoren (TALEB) und nicht-viraler Gen-insertion vorstellen. Zwei Poster werden präsentiert:

1. 'Kontrolle der C-to-T-Editierung mit TALE-Basiseditoren' von Alexandre Juillerat, Ph.D., am 24. Oktober. Diese Studie verbessert das Verständnis von TALEB und ermöglicht die Gestaltung effizienter und spezifischer Werkzeuge, die potenziell mit therapeutischen Anwendungen kompatibel sind.

2. 'Zirkuläres einzelsträngiges DNA ermöglicht effiziente TALEN-vermittelte Geninsertation in langfristigen HSC' von Julien Valton, Ph.D., ebenfalls am 24. Oktober. Diese Forschung zeigt, dass die Zirkularisierung von ssDNA die Geninsertionsraten in langfristigen HSC erhöht und deren Transplantationsfähigkeit in präklinischen Mausmodellen verbessern kann, was die nicht-virale Gentherapie vorantreibt.

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NEW YORK, Oct. 22, 2024 (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, announced today that they will be showcasing pre-clinical data that permit the design of an efficient and specific TALE base editors (TALEB) as well as a process to enhance the efficacy of non-viral gene insertion in hematopoietic stem and progenitor cells (HSPCs) at the European Society of Cell and Gene Therapy 31st annual congress, that will take place on October 22-25, 2024, in Roma, Italy.

The data will be presented in two posters:

Controlling C-to-T editing with TALE base editors

Presenter: Alexandre Juillerat, Ph.D., Vice-President Gene Editing & NY Lab Head at Cellectis

Date/Time: Thursday, October 24 from 2:00pm to 3:30pm CET

Poster number: P0666

  • TALE base editors (TALEB) are fusions of a transcription activator-like effector domain (TALE), split-DddA deaminase halves, and an uracil glycosylase inhibitor (UGI). The C-to-T class of TALEB edits double strand DNA by converting a cytosine (C) to a thymine (T) via the formation of an uracil intermediate.

  • Cellectis recently developed a strategy that allows the comprehensive characterization of C-to-T conversion efficiencies within the editing window. This method also takes advantage of a highly precise and efficient TALEN®-mediated ssODN knock-in in primary T cells to assess how target composition and spacer variations affect TALEB activity/efficiency.

  • The datasets obtained in this study enhanced our understanding of TALEB and permitted the design of efficient and specific tools that could be compatible with the potential development of therapeutic applications.

Circular Single-Stranded DNA Enables Efficient TALEN-Mediated Gene Insertion in Long Term HSC

Presenter: Julien Valton, Ph.D., Vice-President Gene Therapy at Cellectis

Date/Time: Thursday, October 24 from 2:00pm to 3:30pm CET

Poster number: P0585

  • Non-viral alternatives such as linear single-stranded DNA (LssDNA) and circular single-stranded DNA (CssDNA) are emerging as promising options to vectorized DNA donor template for nuclease-mediated gene insertion in hematopoietic stem and progenitor cells (HSPCs) used for gene therapy applications.

  • Capitalizing on its TALEN® technology, Cellectis has devised a gene editing process that incorporates non-viral DNA donor template delivery (LssDNA or CssDNA) to enhance gene insertion in HSPCs.

  • The circularization of ssDNA increases gene insertion rates in long term HSCs and has the potential to enhance their engraftment capacity in preclinical murine model, thereby to facilitate the advancement of next-generation cell therapies. This research marks a crucial step towards enhancing the efficacy of non-viral gene therapy.

The posters will be published on Cellectis’ website after the presentations.

About Cellectis       

Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. Cellectis 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 make therapeutic gene editing in hemopoietic stem cells for various diseases. As a clinical-stage biopharmaceutical company with 25 years of experience and expertise in gene editing, Cellectis is developing life-changing product candidates utilizing TALEN®, its gene editing technology, and PulseAgile, its pioneering electroporation system to harness the power of the immune system in order to treat diseases with unmet medical needs. Cellectis’ headquarters are in Paris, France, with locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS).       

To find out more, visit our website: www.cellectis.com

Follow Cellectis on social networks @cellectis on LinkedIn and X (formerly Twitter)

TALEN® is a registered trademark owned by Cellectis. 

Forward-looking Statements    

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 "will”, “could”, “potential”, “has the potential to” and emerging or the negative of these and similar expressions. These forward-looking statements, which are based on our management’s current expectations and assumptions and on information currently available to management, include statements about the potential of our research programs, including without limitation the TALEB and TALEN technologies. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including with respect to the numerous risks associated with biopharmaceutical product candidate development. Furthermore, many other important factors, including those described in our Annual Report on Form 20-F and the financial report (including the management report) for the year ended December 31, 2023 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, 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, Interim Chief Financial Officer, +1 (347) 809 5980, investors@cellectis.com              

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FAQ

What will Cellectis (CLLS) present at the ESGCT 31st Annual Congress in 2024?

Cellectis will present pre-clinical data on TALE base editors (TALEB) and non-viral gene insertion in hematopoietic stem and progenitor cells (HSPCs) at the ESGCT 31st Annual Congress in Roma, Italy, from October 22-25, 2024.

What is the focus of Alexandre Juillerat's poster presentation for Cellectis (CLLS)?

Alexandre Juillerat's poster, titled 'Controlling C-to-T editing with TALE base editors,' focuses on a strategy to characterize C-to-T conversion efficiencies within the editing window of TALE base editors (TALEB).

What are the potential benefits of Cellectis' (CLLS) research on circular single-stranded DNA?

Cellectis' research shows that circular single-stranded DNA (CssDNA) increases gene insertion rates in long-term HSCs and may enhance their engraftment capacity in preclinical murine models, potentially advancing non-viral gene therapy applications.

When and where will Cellectis (CLLS) present its research at the ESGCT congress in 2024?

Cellectis will present its research on October 24, 2024, from 2:00pm to 3:30pm CET at the European Society of Cell and Gene Therapy 31st annual congress in Roma, Italy.

What technology is Cellectis (CLLS) using for gene insertion in HSPCs?

Cellectis is using its TALEN® technology along with non-viral DNA donor template delivery (LssDNA or CssDNA) to enhance gene insertion in hematopoietic stem and progenitor cells (HSPCs).

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