Universal Cells Inc. products

Universal Cells uses recombinant Adeno-Associated Virus (rAAV)-mediated gene editing to engineer chromosomal genes without the use of genotoxic nucleases. Serotype 3B rAAV vectors efficiently infect pluripotent stem cells and their single-stranded DNA genomes pair with homologous chromosomal sequences during replication to precisely alter human genomic sequences.

Pluripotent stem cells (PSCs) such as embryonic stem cells (ESCs) and induced PSCs (iPSCs) are undifferentiated stem cells that can differentiate into any cell type found in the body. Scientists around the world are using PSCs to produce more mature cell types with diverse therapeutic functions. These new cell therapies have the potential to change the lives of patients suffering from diseases of nearly every organ system.

Realizing the potential of pluripotent stem cells requires that the therapeutic cells derived from PSCs be accepted by patients. PSC-derived cells that have not been engineered to avoid allogeneic rejection will be eliminated soon after transplantation unless they are HLA-matched to their recipients, delivered to immune privileged sites, or administered along with potentially dangerous immunosuppression.

Combining class I and class II engineering to create Universal Donor Cells (UDCs) that are appropriate for developing numerous differentiated cell products.

Class I molecules present peptides to CD8+ T cells and are composed of B2M paired with diverse heavy alpha chains. Human cells express two alleles each of three polymorphic HLA class I molecules (A,B and C) that must be matched to recipients, as well as the non-polymorphic HLA-E molecule. Universal Donor Cells do not express free B2M and lack surface expression of HLA-A, B and C. The B2M-HLA-E single chain fusion protein is expressed from the B2M locus to inhibit lysis by host NK cells, with or without a covalently attached antigenic peptide in the trimer and dimer forms respectively.