Drug intelligence / Profile preview

RND1

Development stage
Preclinical
Lead developer
Lineage Cell Therapeutics
Modality
iPSCs → Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies
01

Overview

RND1 is a preclinical-stage hypoimmune induced pluripotent stem cell (iPSC) line being developed by Lineage Cell Therapeutics in collaboration with Factor Bioscience. The cell line is engineered using gene-editing technology to reduce immunogenicity, facilitating the creation of allogeneic (off-the-shelf) cell transplant therapies. Key genetic modifications include the targeted deletion of the Beta-2-microglobulin (B2M) gene to inhibit rejection by CD8+ T cells and the targeted insertion of the HLA-E gene to prevent adverse natural killer (NK) cell responses. A third undisclosed genetic edit is also included to provide clinical differentiation and a competitive advantage. RND1 is intended for differentiation into cell transplant product candidates for the treatment of central nervous system (CNS) diseases and other neurological indications.

Other names
RND1RND-1RND 1
02

Targets

B2M (Beta-2-microglobulin)HLA-E (Human leukocyte antigen-e)

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