Defining Unmet Needs in Gene Therapy
Viral vector is currently the most common vehicle for gene therapy due to its high gene delivery efficiency. However, use of viral vectors are associated with several disadvantages including: (1) virus-associated safety risks, (2) high manufacturing costs, and (3) limited payload capacity of viral vectors, which severely restrict the repertoire of genes that can be encoded.
A virus-free vector obviates these shortcomings. It is safer than viral vectors due to lower immunogenicity and lower risk of genome toxicity. Moreover, virus-free vector has the capacity to carry larger fragments of genetic material compared to conventional viral vectors. The manufacturing costs of virus-free vectors are also markedly lower than those required for viral vector production.
Despite the advantages of virus-free vectors, it remains a challenge to efficiently modify, expand, and deliver persistent therapeutic cells with stable gene expression. Electroporation is the most efficient method for delivery of virus-free vectors into therapeutic cells, but following electroporation these cells become fragile with markedly reduced viability and expansion capacity.
Quantum Engine™ Addresses the Unmet Needs
GenomeFrontier Therapeutics, Inc. addresses these unmet needs of conventional gene therapy by integrating four platforms: Quantum Nufect™ , Quantum pBac™ , iCellar™ , G-Tailor™ , which improve vector delivery, therapeutic gene integration, cell expansion capacity, and gene design, respectively. Synergistically, these four platforms form the Quantum Engine™ for producing virus-free, gene-modified cells optimal for cell modified gene therapy.
TIR: Terminal Inverted Repeat, also known as ITR and TRD.