Our approach is designed to utilize Homology’s AAVHSC vectors to deliver a functional gene between lengthy DNA guide sequences, or homology arms. The homology arms are designed to align the functional gene to the specific location in the genome. Once aligned, through homologous recombination, correction of the variant gene in the genome is replaced with the functional copy, which may lead to therapeutic protein expression. With gene editing, a person’s DNA is permanently corrected, so this approach can potentially be curative, including in rapidly dividing cells (e.g., pediatric liver cells).
Our gene editing approach does not require a nuclease, or enzyme that cuts the DNA, and has shown high levels of precision with no off-target effects.
Jason Wright, Ph.D., Sr. Director, Molecular Biology & Genomics discusses the journey of our gene editing approach from concept to clinic here.
Our gene editing approach offers several potential advantages:
- Enables single component in vivo gene editing
- Leverages the natural gene repair process of homologous recombination & does not require a nuclease for gene cutting like CRISPR/Cas9 or zinc finger nuclease
- Precise on-target editing and lack of unwanted off-target or on-target DNA modifications confirmed via unbiased, genome-wide measurements
- Editing efficiencies that we believe can achieve the requisite therapeutic threshold