Periodic Reporting for period 1 - CCedit (Coiled-coil mediated exonuclease tethering technology for the enhancement of CRISPR gene editing)
Reporting period: 2020-02-01 to 2022-01-31
The ERC PoC CCEdit action investigated industrial and commercial opportunities of the CRISPR/Cas-coiled-coils-exonuclease construct. It was hosted by the National Institute of Chemistry, Department of Synthetic Biology and Immunology, led by Prof Dr Roman Jerala. In 2018, he obtained an ERC Advanced Grant for the project MaCChines, enabling development of the science and technology in the field of self-assembling biotech nanostructures. This innovative science produced results with applied and commercial potential such as enhanced CRISPR/Cas technology.
In 2019, the Department filed a patent application on the improvement of the CRISPR method. Specifically, the apparently biggest potential of this genome editing tool lies in gene inactivation via non-homologous end joining (NHEJ) cell repair mechanism, where double stranded breaks (DSB) are repaired in an error prone pathway leading to gene inactivation. Such advancements could be used in the most critical and perspective industries for the wellbeing of humankind. This particularly includes agriculture, healthcare with an emphasis on advanced therapeutics and various biotech research tools. According to some market insights, the commercial opportunity should reach over 6.3 billion EUR by 2026. The most recent successes, such as the Intellia Therapeutics and Regeneron first successful in vivo trials in 2021, just keep increasing the public expectation and thus also the market value. Yet the technology can be exploited only if the accompanying legal aspects are taken into consideration. The IP disputes, mainly between the Broad Institute and UC Berkeley, are still ongoing. While the IP landscape is well defined, the ownership over some of the core patents depends on the legal procedures.
The action searched for ‘proof-of-concepts’ not just in actual applications but also in business development and communication. The Slovenian biotech landscape falls behind the developed western Life Sciences ecosystems, so this PoC project was an opportunity to introduce various new practices. Starting in early 2020, when the world faced the SARS‑CoV‑2 pandemic, limitations in organising events and travel hindered the possibility of traditional business practices. The CCEdit leveraged the potential of digital communication and achieved two visible awards for the website and the overall strategy (1st place NETKO 2021 and 2nd place Websi 2021). Throughout the action we conducted a merged model of business analysis and training, which allowed researchers to obtain some skills in a biotech start-up field. Additionally, the action enabled to mentor a group of four young researchers (two female and two male), which worked on their own technology, motivating them for future commercial opportunities originating from the Department. The commercial potential of the innovation was tested through discussions with the leading companies and a key-opinion-leader, while the capacity building was achieved by linking with the UK’s gene and cell accelerators. The best confirmation that the action was successful may be the award for the Best Business Practice in 2021, awarded to our project by the American Chamber of Commerce in Slovenia.
The characteristics of the project, with emphasis an on the IP landscape and application itself, did not yet fulfil all the investment criteria of the venture capital organisation, however the EIC grants and established links present some good opportunities in the future. The CCEdit group and other collaborators is now also competing for a multi-mullion opportunity to build a gene & cell infrastructure and ecosystem for the CCEdit project outcomes and related technologies. Importantly, during the development of CCedit technology within the PoC we discovered that it could be also implemented for the targeted delivery of a genetic cargo with potential applications for cancer immunotherapy and gene therapy, which is likely to open much more impactful opportunities and we plan to develop this direction further either through the following PoC project, EIC project or other funding opportunities.
In 2019, the Department filed a patent application on the improvement of the CRISPR method. Specifically, the apparently biggest potential of this genome editing tool lies in gene inactivation via non-homologous end joining (NHEJ) cell repair mechanism, where double stranded breaks (DSB) are repaired in an error prone pathway leading to gene inactivation. Such advancements could be used in the most critical and perspective industries for the wellbeing of humankind. This particularly includes agriculture, healthcare with an emphasis on advanced therapeutics and various biotech research tools. According to some market insights, the commercial opportunity should reach over 6.3 billion EUR by 2026. The most recent successes, such as the Intellia Therapeutics and Regeneron first successful in vivo trials in 2021, just keep increasing the public expectation and thus also the market value. Yet the technology can be exploited only if the accompanying legal aspects are taken into consideration. The IP disputes, mainly between the Broad Institute and UC Berkeley, are still ongoing. While the IP landscape is well defined, the ownership over some of the core patents depends on the legal procedures.
The action searched for ‘proof-of-concepts’ not just in actual applications but also in business development and communication. The Slovenian biotech landscape falls behind the developed western Life Sciences ecosystems, so this PoC project was an opportunity to introduce various new practices. Starting in early 2020, when the world faced the SARS‑CoV‑2 pandemic, limitations in organising events and travel hindered the possibility of traditional business practices. The CCEdit leveraged the potential of digital communication and achieved two visible awards for the website and the overall strategy (1st place NETKO 2021 and 2nd place Websi 2021). Throughout the action we conducted a merged model of business analysis and training, which allowed researchers to obtain some skills in a biotech start-up field. Additionally, the action enabled to mentor a group of four young researchers (two female and two male), which worked on their own technology, motivating them for future commercial opportunities originating from the Department. The commercial potential of the innovation was tested through discussions with the leading companies and a key-opinion-leader, while the capacity building was achieved by linking with the UK’s gene and cell accelerators. The best confirmation that the action was successful may be the award for the Best Business Practice in 2021, awarded to our project by the American Chamber of Commerce in Slovenia.
The characteristics of the project, with emphasis an on the IP landscape and application itself, did not yet fulfil all the investment criteria of the venture capital organisation, however the EIC grants and established links present some good opportunities in the future. The CCEdit group and other collaborators is now also competing for a multi-mullion opportunity to build a gene & cell infrastructure and ecosystem for the CCEdit project outcomes and related technologies. Importantly, during the development of CCedit technology within the PoC we discovered that it could be also implemented for the targeted delivery of a genetic cargo with potential applications for cancer immunotherapy and gene therapy, which is likely to open much more impactful opportunities and we plan to develop this direction further either through the following PoC project, EIC project or other funding opportunities.