Cellectis Publishes a Novel Intronic Gene Editing Approach For the Treatment of Inborn Metabolic Diseases by Edited HSPCs - Seite 2

Research data showed that:

• The CD11b intron-specific gene insertion approach efficiently restricts the expression of a desired transgene to the myeloid lineage, preventing its overexpression by stem cells or by other differentiated lineages.

• The insertion of an IDUA transgene in the first intron of the CD11b gene enables to express IDUA (the enzyme missing in Mucopolysaccharidosis type I patients), in a myeloid-specific manner without affecting CD11b endogenous expression.

• Edited HSPC exhibited robust engraftment in the bone marrow of immunodeficient mice, displayed multi-lineage differentiation in various hematopoietic tissues and showed significant presence in the brain as myeloid cells.

The article is available on Molecular Therapy website by clicking on this link:
https://doi.org/10.1016/j.ymthe.2024.04.001

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 over 24 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).

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