Trucode is pioneering a simple systemic drug delivery approach for gene editing, with potential advantages for patients in terms of safety and convenience:
Intravenous drug administration
Virus-free drug delivery
No bone marrow transplant or immunosuppression required
Our scientific collaborators, Peter Glazer, MD, PhD; Mark Saltzman, PhD; and Marie Egan, MD at Yale, have demonstrated the potential utility of these PNAs for gene editing in human cells and animal models by intravenous administration.
Intravenous injection of PNA and DNA correction sequence-containing nanoparticles was shown to correct a hemoglobin defect in ß-thalassemic mice, durably reversing the disease phenotype by achieving gene correction in bone marrow derived hematopoietic stem cells.
We are further enhancing nanoparticle delivery by examining alternate routes of delivery, altering cargo release patterns, optimizing preferential uptake by particular cell types and thereby further broadening our delivery capabilities to address multiple cell types in multiple organ systems.
Trucode Gene Repair: In vivo delivery
Others: Ex vivo modification plus transplant