Universal Cells is a therapeutic genome editing company. The company uses a nuclease-free technology platform for efficient and accurate editing of any chromosomal gene, without off-target cutting.
Universal Cells is developing HLA-engineered Universal Donor Cells that are off-the-shelf stem cell products compatible with everyone. No immune suppression. No donor matching. No rejection.
In addition, the company is developing therapeutics that target and correct the underlying genetic causes of diseases.
Universal Cells patented technology is based on recombinant adeno-associated virus (rAAV)-mediated gene editing, which offers high accuracy and safety, and the best vehicle available for cellular delivery.
Recombinant adeno-associated virus (rAAV) vectors are single-stranded DNA vectors that efficiently infect stem cells and edit the genome by homologous recombination. The rAAV genome (green) is engineered to contain DNA sequences homologous to a chromosomal target gene (blue arrows). The rAAV sequence can include mutations to knockout gene expression or knock-in new genes of interest (red).