Cellectis Unveils Advances in Gene Therapy at ESGCT Conference
Innovative Gene Therapy Insights from Cellectis
NEW YORK — Cellectis (Euronext Growth: ALCLS - NASDAQ: CLLS) is at the forefront of biotechnology, focusing on groundbreaking gene editing techniques to foster advancements in life-saving therapies. Their cutting-edge research was recently spotlighted at a prominent conference that gathered leading experts from the field.
Non-Viral Gene Editing Breakthrough
Cellectis introduced compelling data emphasizing the application of circular single-stranded DNA (CssDNA) as a transformative non-viral gene therapy template. This presentation showcased findings about the efficacy of CssDNA in enhancing genetic modifications within hematopoietic stem and progenitor cells (HSPCs), along with a comprehensive analysis of the safety of TALE base editors.
Poster Presentation Highlights
The poster titled Circularization of Single-Stranded DNA Donor Template Unleashes the Power of Non-Viral Gene Delivery for Long-Term HSCs Editing was presented by Julien Valton, Ph.D., the Vice President of Gene Therapy at Cellectis. The findings revealed two critical outcomes:
- CssDNA editing demonstrated a significant increase in gene insertion frequency within viable HSPCs.
- CssDNA-optimized HSPCs exhibited a superior capability for engrafting and maintaining genetic changes in murine models compared to traditional adeno-associated virus (AAV) methods.
These outcomes strongly indicate CssDNA's potential as a streamlined and efficient mechanism for advancing gene therapy applications.
TALE Base Editing Safety Analysis
Another vital component of the research presented at the event was an exploration into TALE base editors (TALEB). This innovative editing tool merges a transcription activator-like effector domain with specialized enzymatic components that facilitate precise genetic edits without causing DNA breaks.
Comprehensive Safety Evaluation
Maria Feola, Ph.D., Senior Scientist and Team Leader at Cellectis, presented work on the safety profile of TALEB, which provided insights into potential off-target effects during the editing process. The analysis utilized advanced bioinformatics coupled with experimental validations, confirming no significant biases toward unintended C-to-T edits in the vicinity of essential genomic sites associated with DNA organization and expression.
These findings establish a robust foundation for the safe utilization of TALEB in future therapeutic applications aimed at both nuclear and mitochondrial genomes.
Future Perspectives on Gene Therapy
Cellectis continues to position itself as a leader in the biotechnology landscape by innovating within the realm of gene therapies. The company not only focuses on therapeutic applications but also incorporates a comprehensive approach from research and development through to manufacturing. Their allogeneic CAR T-cell therapies stand as a testament to their vision of delivering ready-to-use treatments designed to combat cancer effectively.
This integrated operation underlines Cellectis' unique capability to manage the entire gene therapy process, ensuring quality and efficiency at every stage. With its headquarters situated in Paris and additional locations in New York and Raleigh, North Carolina, Cellectis is well-positioned to meet the growing demands in the therapeutic landscape.
Contact Information for Further Inquiries
For more details on their presentations or inquiries about their ongoing research, interested parties can reach out to:
Media Contact:
Pascalyne Wilson, Director, Communications, +33 (0)7 76 99 14 33, media@cellectis.com
Investor Relations:
Arthur Stril, Chief Financial Officer & Chief Business Officer, investors@cellectis.com
Frequently Asked Questions
What recent advancements were shared by Cellectis at the ESGCT Congress?
Cellectis presented innovative data focusing on non-viral gene therapy using CssDNA and the safety analysis of TALE base editors.
Who were the key presenters at the event?
Julien Valton, Ph.D., presented on CssDNA, while Maria Feola, Ph.D., discussed TALE base editors' safety.
How does CssDNA improve gene therapy outcomes?
CssDNA enhances gene insertion frequency and improves engraftment success in stem cells compared to traditional methods.
What impact does the TALE base editing tool have?
TALEB allows precise edits in the DNA sequence without inducing breaks, which reduces potential for off-target effects.
How does Cellectis manage its gene therapy process?
Cellectis oversees the entire therapy production cycle, from development to manufacturing, ensuring quality and efficiency in treatments.
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