Universal Cells is a therapeutic genome editing company using a nuclease-free technology platform to efficiently and accurately edit any gene, without off-target cutting.
The company is developing therapeutics that target the underlying genetic causes of diseases and HLA-engineered Universal Donor Cells that are rejection-free, off-the-shelf stem cell products that are compatible with everyone. No immune suppression. No donor matching. No rejection.
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. Stem cells are targeted with our patented stem cell-tropic AAV3b serotype.
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). The diagram displays the introduction of a disrupting mutation into the Beta-2-Microglobulin gene (B2M).
Universal Cells' technology enables the complete engineering of HLA class I and class II expression. We eliminate the expression of all polymorphic HLA proteins, and reintroduce only the specific HLA molecules required for a given indication.
For example, we can remove all HLA class I molecules from the cell surface and simultaneously re-introduce a non-polymorphic HLA-E molecule to prevent lysis by Natural Killer cells. Customized HLA molecules can also be expressed in order to present specific peptides.
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Genetic engineering of HLA class I
Parent cells express 6 polymorphic HLA class I molecules (HLA-A, B & C) that need to be matched to each transplant recipient (colored circles).
Both alleles of B2M are knocked out by gene editing with rAAV to prevent HLA-A, B & C expression, while a single-chain, non-polymorphic HLA-E molecule (gray circles) is simultaneously reintroduced by knock-in at one B2M allele.