Cellectis announced on November 19 that it has published a paper in Nature Communications describing a novel non‑viral gene‑editing platform that uses circular single‑stranded DNA (CssDNA) as a donor template for efficient gene insertion into hematopoietic stem and progenitor cells (HSPCs).
The platform leverages Cellectis’ proprietary TALEN technology and achieves 3‑5 times higher knock‑in efficiency than linear single‑stranded DNA, with efficiencies exceeding 40%. In preclinical mouse models, CssDNA‑edited HSPCs showed higher engraftment rates and better maintenance of gene edits compared with AAV6‑edited cells, demonstrating a safer and potentially more scalable alternative to viral vectors.
This breakthrough positions Cellectis as a leader in non‑viral gene‑editing, addressing key safety and scalability concerns that have limited the broader adoption of viral vector‑based therapies. By expanding its gene‑editing toolbox, the company can accelerate the development of next‑generation cell and gene therapies across multiple therapeutic areas, strengthening its intellectual‑property moat and competitive advantage.
Following the publication, Cellectis’ shares rose 6.2% in the U.S. market and 10% in Paris, reflecting investor confidence in the technology’s commercial potential. Analysts upgraded ratings and raised price targets, citing the platform’s superior efficiency and safety profile as a catalyst for future growth.
The new platform aligns with Cellectis’ broader strategy to broaden its gene‑editing capabilities and accelerate clinical translation. It complements prior work on universal CAR T cells and sickle cell disease, and positions the company to address limitations of current viral vector‑based approaches. Management remains optimistic about scaling the CssDNA platform into its pipeline, with plans to integrate it into upcoming clinical trials and commercial programs.
Cellectis’ leadership in non‑viral gene‑editing underscores its commitment to innovative, scalable solutions that can transform the gene‑therapy landscape, offering a clear pathway to expand therapeutic options and enhance patient outcomes.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.