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Cellectis Shares Jump on Breakthrough Gene Editing Data

MarketDash Editorial Team
18 days ago
Cellectis published research in Nature Communications demonstrating that circular single-stranded DNA dramatically improves gene insertion efficiency in stem cells, offering a safer alternative to viral methods for next-generation therapies.

Cellectis A/S (CLLS) just gave investors a reason to get excited about the future of gene therapy, and the stock responded accordingly.

The company published research Wednesday in Nature Communications that establishes circular single-stranded DNA (CssDNA) as a highly efficient non-viral template for inserting genes into hematopoietic stem and progenitor cells (HSPCs). Think of HSPCs as the bone marrow's master cells that can self-renew and eventually mature into every type of blood cell your body needs, including red blood cells, white blood cells, and platelets.

Why does this matter? Because editing HSPCs offers the potential for long-term therapeutic benefits. Fix the stem cells, and you potentially fix all the blood cells they create downstream.

The problem has been delivery. Viral vectors like AAV6 are commonly used to insert genes, but they come with safety and efficacy concerns that make researchers nervous. Over the past decade, non-viral DNA templates have emerged as promising alternatives. Scientists have used them with nucleases to target short single-stranded linear DNA corrective templates in HSPCs, but these approaches were initially limited to making only small corrections within defective genes.

Cellectis changed the game by combining its TALEN technology with CssDNA donor templates to develop a gene insertion process that enables precise and efficient integration of large genetic sequences within therapeutically relevant subpopulations of HSPCs. This significantly expands what non-viral gene therapy can accomplish.

The results are impressive. CssDNA achieved 3-5 times higher knock-in efficiency than linear single-stranded DNA (LssDNA), with success rates surpassing 40%. The technology works across multiple genetic locations in HSPCs and applies to other therapeutically interesting cell types, including primary T cells.

Perhaps most importantly, comparative studies showed that CssDNA-edited HSPCs demonstrated a higher propensity to engraft and maintain gene edits in a murine model compared to AAV6-edited HSPCs. That's the kind of data that suggests real therapeutic staying power.

"These results establish the CssDNA process as an efficient non-viral gene insertion strategy, and mark a pivotal advance towards the development of next-generation cell and gene therapies," said Julien Valton, Vice President of Gene Therapy at Cellectis.

The company has some runway to work with too. As of September 30, 2025, Cellectis had $225 million in consolidated cash, cash equivalents, restricted cash, and fixed-term deposits. Management believes this will fund operations into the second half of 2027.

Price Action: CLLS stock was up 8.27% at $4.45 at last check on Thursday.

Back to News

Cellectis Shares Jump on Breakthrough Gene Editing Data

MarketDash Editorial Team
18 days ago
Cellectis published research in Nature Communications demonstrating that circular single-stranded DNA dramatically improves gene insertion efficiency in stem cells, offering a safer alternative to viral methods for next-generation therapies.

Cellectis A/S (CLLS) just gave investors a reason to get excited about the future of gene therapy, and the stock responded accordingly.

The company published research Wednesday in Nature Communications that establishes circular single-stranded DNA (CssDNA) as a highly efficient non-viral template for inserting genes into hematopoietic stem and progenitor cells (HSPCs). Think of HSPCs as the bone marrow's master cells that can self-renew and eventually mature into every type of blood cell your body needs, including red blood cells, white blood cells, and platelets.

Why does this matter? Because editing HSPCs offers the potential for long-term therapeutic benefits. Fix the stem cells, and you potentially fix all the blood cells they create downstream.

The problem has been delivery. Viral vectors like AAV6 are commonly used to insert genes, but they come with safety and efficacy concerns that make researchers nervous. Over the past decade, non-viral DNA templates have emerged as promising alternatives. Scientists have used them with nucleases to target short single-stranded linear DNA corrective templates in HSPCs, but these approaches were initially limited to making only small corrections within defective genes.

Cellectis changed the game by combining its TALEN technology with CssDNA donor templates to develop a gene insertion process that enables precise and efficient integration of large genetic sequences within therapeutically relevant subpopulations of HSPCs. This significantly expands what non-viral gene therapy can accomplish.

The results are impressive. CssDNA achieved 3-5 times higher knock-in efficiency than linear single-stranded DNA (LssDNA), with success rates surpassing 40%. The technology works across multiple genetic locations in HSPCs and applies to other therapeutically interesting cell types, including primary T cells.

Perhaps most importantly, comparative studies showed that CssDNA-edited HSPCs demonstrated a higher propensity to engraft and maintain gene edits in a murine model compared to AAV6-edited HSPCs. That's the kind of data that suggests real therapeutic staying power.

"These results establish the CssDNA process as an efficient non-viral gene insertion strategy, and mark a pivotal advance towards the development of next-generation cell and gene therapies," said Julien Valton, Vice President of Gene Therapy at Cellectis.

The company has some runway to work with too. As of September 30, 2025, Cellectis had $225 million in consolidated cash, cash equivalents, restricted cash, and fixed-term deposits. Management believes this will fund operations into the second half of 2027.

Price Action: CLLS stock was up 8.27% at $4.45 at last check on Thursday.