Antibody-mediated DNA/RNA delivery
3E10 is a cellpenetrating mAb that transports a donor DNA for gene editing into cells and tissues in vivo
- Advantages:
- No need for DNA cutting or binding agent
- Patient treatment by simple IV administration of simple mixture of geMab and donor DNA
- Established manufacturing processes for both components
- The approach has no sequence limitations to reagent design
- Therapeutic applications. Gene editing to correct mutations causing genetic disorders: sickle cell disease, thalassemia, cystic fibrosis, lysosome storage diseases
- Lead Innovator: Peter Glazer, Ph.D.
- IP status: pending "COMPOSITIONS AND METHODS FOR ENHANCING DONOR OLIGONUCLEOTIDE BASED GENE EDITING"