GENE EDITING & XENOTRANSPLANTATION
CRISPR is a genome-editing tool that can selectively delete, modify or correct a disease-causing abnormality in a specific DNA segment. CRISPR refers to Clustered Regularly Interspaced Short Palindromic Repeats occurring in the genome of certain bacteria. CRISPR technology uses a protein-RNA complex composed of Cas-9, which binds to a guide RNA (gRNA) molecule that has been designed to recognize a particular DNA sequence.
The concept of cross-species transplantation, known as xenotransplantation, is the transfer of living cells, tissues or organs from one species to another. Due to the shortage of human organs, xenotransplantation emerged as an alternative potential option and its clinical potential is being explored with new technologies such as CRISPR Cas-9.
We believe that CRISPR technology is a highly disruptive and sophisticated technology that will allow us to address hurdles in xenotransplantation that have previously been insurmountable – and we have the world’s leaders in the field working on doing it safely and effectively.
The eGenesis team has been able to produce PERV-free activity through the use of our CRISPR Cas-9 technology.
The risk of cross-species transmission of PERVs, among other issues, has impeded the use of pig organs in humans until now.
We’ve built a stringent detection system using CRISPR-Cas9 and found that porcine endogenous retrovirus, also known as PERV, exhibit pig-to- human and human-to- human infectivity in cell cultures – a key advancement in the evolution of the xenotransplantation field.
Using our CRISPR-Cas9 technology and gene editing tools, we’ve successfully produced viable PERV-free porcine embryos. This research represents a critical and essential advancement in addressing concerns around cross-special viral transmission for clinical xenotransplantation.
The company’s clinical development plan covers four key areas:
- Cellular genome engineering — to address porcine endogenous retrovirus (PERV) and immunology separately and combine both features after optimization;
- Pig production – to assess PERV infectivity of engineered pigs and tumorigenesis testing of animal and animal cells;
- Additional preclinical testing – to assess human usable immunosuppressant and the viability and functionality of tissue transplants (xenografts); and
- Investigational New Drug (IND) enabling study – to be conducted at a designated pathogen free facility with a separate herd of pigs.
