Developing the Next Generation of Gene Editing Tools
CRISPR is one of the greatest discoveries of this century, and Cas9 is the most widely used Cas protein for genome editing. But that’s just the tip of the iceberg – one of many possible new technologies for gene-editing. Initial successes have inspired researchers to discover new CRISPR systems for targeting DNA that naturally occur in bacteria and have the potential to advance therapeutic approaches.
While many researchers and companies are now leveraging the CRISPR-Cas9 system for wide therapeutic use, the next scientific breakthrough will be in advancing the use of CRISPR-based tools and finding other variants that can cut and edit genes to expand the range of treatable diseases. Leaps by Bayer is supporting this mission by investing in companies like Metagenomi with the goal of finding more efficient and safer enzymes.
“Investments in companies such as Metagenomi allow us to support innovative approaches that will ultimately help us achieve our Leaps goals: advancing from treating to curing genetic diseases,” says Dr. Juergen Eckhardt, Head of Leaps by Bayer. At Metagenomi, researchers are applying a powerful big data approach that could lead to the discovery of new classes of next generation gene editing systems, in order to accelerate finding cures for genetic diseases and other therapeutic areas. The financial investment from Leaps by Bayer and other visionary investors could help to unlock full potential of these systems and translate them into new therapies for patients.
CRISPR in the fight against COVID-19
CRISPR is also being leveraged in the fight against COVID-19. The FDA has issued an emergency-use authorization for a CRISPR-based COVID-19 test that takes less than an hour. Scientists are also working to create a version that could be used at home.
In addition, Stanford researchers have developed a CRISPR-based treatment, which destroyed 90 percent of the virus in tests. But like all other potential COVID-19 treatments, there are many regulatory steps to go through before it is approved and can be used by patients.
Mining for new gene editing tools and applications
Metagenomi is applying machine learning algorithms to DNA library databases to rapidly find and enhance natural enzyme systems derived from bacteria that can, in turn, facilitate disease applications. This meta-genomic approach enables identification of novel gene editing systems – or new scissors to cut genes – with potential for enhanced capabilities that leapfrog first generation gene editing approaches. For instance, smaller and more compact Cas proteins would become easier to deliver into cells for editing the genome, since they can be packed into small delivery vehicles and are easier to manufacture. Metagenomi’s big data approach has already identified more than 10,000 CRISPR systems, and its gene editing systems may target new sections of DNA that are inaccessible to current Cas9 and similar enzymes.
The novel gene editing systems may also overcome current usage limitations related to intellectual property restrictions and give more opportunities to treat diseases in several therapeutic areas.
When will the next generation of gene editing tools be available to patients?
“While CRISPR is showing already some promising results in early clinical trials, it’s difficult to predict the future, but the hope is that in the next decade some patients will be cured of diseases that currently have limited or no treatment options,” says Juergen Eckhardt. However, research is still in its early stages, and there is a high risk of failure. “In order to push beyond what is considered possible, we need to go beyond industry conventions and explore radically new approaches,” emphasizes Eckhardt.
Gene editing has an unimaginable potential to change the world for the better in various areas of human life and to address societal needs.
Focus on cell and gene therapy
At Leaps by Bayer, we are investing in the development of CRISPR-based drugs and technologies to move from treatment to cure. One example beyond Metagenomi and one area of engagement and investment for Leaps is advancing sustainable organ replacement (through gene edited animal organs). Worldwide, an estimated 1.5 to 2 million people are on organ transplant waitlists. eGenesis, for instance, is a biotechnology company that is utilizing gene-editing technologies for the development of human-compatible organs to address the global organ shortage. This could save lives in ways that were not thought possible just a few years ago.
About Leaps by Bayer
Leaps by Bayer utilizes impact investing to find solutions for some of the greatest challenges of our time in the fields of healthcare and agriculture.
The ten leaps
- Cure genetic diseases
- Provide sustainable organ replacement
- Reverse autoimmune diseases
- Regenerate lost tissue function
- Prevent and cure cancer
- Reduce the environmental impact of agriculture
- Cure through microbiome health
- Develop a sustainable protein supply
- Eradicate insect-borne infections
- Drive transformational digital business models
Related links:
Gene editing: The promise CRISPR holds to revolutionize our world
