Cellectis presents non-viral gene editing and base editing research at ASGCT meeting
Biotechnology company Cellectis has presented new research data on non-viral gene editing and base editing at the ASGCT annual meeting. The new methods aim to improve the efficiency and safety of cell therapies.
New York – Cellectis, a clinical-stage biotechnology company, has presented new research data on non-viral gene editing and base editing at the American Society of Gene and Cell Therapy (ASGCT) annual meeting.
The research focused on two key areas: TALEN®-mediated non-viral gene insertion for advancing cellular and gene therapies, and advancements in genetic editing using base editors (TALEB).
The non-viral gene editing approach aims to address challenges associated with traditional viral vectors, such as lentiviruses or AAV vectors, including manufacturing constraints, potential genomic toxicities, and limited payload sizes. Cellectis presented data showing that combining TALEN®-mediated gene editing with non-viral delivery of single-stranded DNA (ssDNA) can achieve efficient gene insertion in T-cells as well as hematopoietic stem and progenitor cells (HSPCs). Specifically, circular ssDNA (CssDNA) templates demonstrated advantages over traditional viral donor templates, promoting better maintenance of HSPC fitness and more stable gene editing.
Furthermore, the company showcased advancements in TALE base editors (TALEB), which enable the conversion of cytosine (C) to thymine (T) in DNA without requiring a DNA strand nick. The research indicated that TALEB technology can efficiently perform C-to-T edits, and that factors such as target sequence and editor architecture can be used to precisely control editing outcomes. Off-target editing risks were assessed, with no detectable editing observed in primary cells at previously described sites, highlighting TALEB's specificity for potential therapeutic applications.
These developments provide new tools for the advancement of gene therapies, potentially offering more efficient and safer treatment options for patients with conditions such as cancer, autoimmune diseases, and monogenic disorders.