A new approach for safe and effective human therapeutic gene editing

Advancements in the development of genome editing technologies have significantly improved our ability to make precise changes to the DNA of eukaryotic cells, with the potential to truly transform healthcare interventions from the targeting of faulty genomes of cancer cells through to correcting genetic errors of inherited disorders. Despite this potential, current genome editing techniques such as CRISPR - which have originated as research tools - have to date failed to fully translate into therapeutic application due to unresolved challenges in accuracy (high risk in mis-editing DNA and off target effects), tolerance (potential for an immunological reaction) and ultimately safety and while these problems are acceptable in research or _ex vivo_ applications, they represent critical barriers preventing the full potential for human therapeutic genome editing to be realised.With the potential to truly disrupt the global genome editing market (forecast to reach $8.1 billion by 2025), Mote have developed a solution with significantly higher precision and safety than current genome editing technologies, and better suited to therapeutic use. Having proven the ability to effectively target a defined DNA sequence in cultured human cells and in collaboration with the Belfast based pHion who offer a unique platform drug delivery system suited for therapeutic applications, the proposed 18 month industrial research project will seek to advance the concept to Phase 1 trial based around two initial targets -- EBV and Cystic fibrosis (CF) with R&D activity around alternate targeting approaches, the design of a delivery system for integrase and therapeutic payload and animal modelling.As a fundamental technology the approach offers the potential to be translated into therapeutic agents for a range of genetic diseases, retroviral diseases and cancers, with the promise to replace costly and lengthy drug treatments with improved efficacy and patient outcomes. At a time when significant investment is being made in the genome editing market -- investment that centres largely around the use of CRISPR which has known limitations (it is a great tool but wrong for the job), the development also offers a significant commercial opportunity for both partners also helping strengthen the UK's dominant position in the cell and gene therapy market with global exploitation potentialAwaiting Public Project Summary