Many diseases are caused by mutations in our DNA resulting in the production of faulty proteins, genome instability or changes in expression patterns within our highly specialized cells.
These genomic changes are responsible for various serious diseases such as cancer. Addressing the root cause of these diseases has not previously been feasible with conventional small molecule-based drugs due to a lack of druggability.
The development of genome engineering approaches allows the targeted repair of such mutations or the introduction of recombinant genetic material into target cells. Discoveries such as the CRISPR/Cas system for gene editing have turbo charged the development of gene therapy approaches allowing the targeted alteration of DNA sequences within cells of patient. These powerful tools can help to effectively cure hereditary diseases by fixing the mutated genetic code in affected cells.
Genome engineering technology has been incepted several decades ago and new approaches are invented every year further improving the accuracy and efficacy of next generation drugs. Addressing accuracy of gene therapy in target cells remains critical as off-target effects can have undesired consequences in patients. However, recent developments allow to precisely excise single nucleotides of faulty DNA and repair lesions with the correct base.
The continuous improvements of target precision and DNA repair or alteration efficiency of the genome engineering toolkit illustrate the potential of the technology for the years to come.
The possible applications of genome engineering are plentiful. Alteration of the genome of microorganisms such as E. coli or P. pastoris, for instance, allows the introduction of foreign genetic material useful for the production of complex molecules with high yields, purity and represent an eco-friendly alternative to conventional methods such as chemical synthesis. Similarly, genetically engineered microorganisms have been utilized heavily in the detoxification of waste materials and leaching of valuable resources such as metals and are at the forefront of a new and sustainable economy.
However, while the technology continues to make tremendous leaps, aggressive IP strategies attempt to claim and block substantial access to the technology especially for one of the most widely utilised approaches such as the CRISPR/Cas system. This has dire consequences for biotech companies and early start-ups struggling to navigate the complex legal landscape of licensing agreements and freedom to operate. Legal uncertainty is poison for innovation and development of technology since investment returns are hard to predict for funding bodies. Our team is closely following recent legislation and developments especially in Europe and the USA in order to help our clients understand obstacles and opportunities in this competitive but highly lucrative field of research.
Our team consists of experts with extensive hand-on experience of the most widely utilized genome engineering technologies and closely work together with management and scientists to develop bespoke strategies to protect technologies with the broadest possible scope.
If you have any questions or suggestions do not hesitate to contact us.