2024-13-007 - DeepCas - Exploiting a cold-adapted CRISPR-Cas9 nuclease from the Deep Ocean

DeepCas

2024-13-007

A novel Cas nuclease in different model organisms, unique in the CRISPR-Cas market and exploit its commercial potential on different fields

Environmental: Development of new therapies that will be translated into savings from the health care system. Also, this finding can be applied in a wide variety of fields (healthcare, agricultural, biotechnology...) Social: Patients suffering a wide range of diseases would have new therapies available. Governance: The project is rised in a public institute with public governance.

Social impact: Identification of new nucleases would open new possibilities for treating genetic disorders and advancing personalized medicine. In fact, improved nucleases could also make already existing CRISPR-based therapies more efficient and accessible, leading to a broader societal benefit in healthcare and biotechnology. This will be directly translated into better patient's quality of life. Economic impact: In addition to clinical benefits, identification and validation of new caspases in order to develop new gene therapies would reduce indirect costs related with chronic diseases. In addition, several companies in the CRISPR-Cas market, which is expected to reach $80 billion by 2029, are constantly under development of new strategies, so our asset will be valuable for them.

Problem: Most of the current genome editing tools are based solely on systems from a limited number of isolated bacteria. As a result, there is a biased and incomplete view of the diversity and potential of CRISPR-Cas systems which limit CRISPR-Cas applications such as targeting small sequences or introducing specific mutations through homologus recombinations. It is crucial to explore alternative Cas effector nucleases to expand its applications. Solution: The identification and validation of a novel Cas9 nuclease from uncultured microorganisms at the deep ocean will provide researchers and society an expanded catalog of nucleases which could make CRISPR-based therapies more efficient and accessible

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Licensing to a specialized company within the biotechnology or pharmaceutical sector that seeks to expand its Cas9 catalog for the development of new therapeutic solutions for treating various diseases.

Improve and increase the catalog of gene therapy strategies available in the market

Patients with diferent disorders that can be treated using gene therapy (hematological disorders, metabolic disorders, immunological disorders or oncology) Public Health System

Optimization of genome editing properties Evaluation of toxicity, specificity and efficiency Intellectual property rights (PCT application) Market research and commercialization plan through a specialized company

Funding for development, co-development and licensing opportunities

Novel Cas9 optimization Toxicity, specificity and efficiency evaluation PCT submission Market study and commercialization plan

New Cas9 nuclease with psychrophilic propertiEs (good activity at low temperatures) and easily denatured by moderate application of heat will help to increase the catalog of Cas9 nucleases and broadening their applications.

Economic & Development Barriers: as a public institution, at some point in the development of this technology, we will need external assistance to develop and commercialize our product.

1. Scientific development - Optimization of genome editing properties - Evaluation of toxicity, specificity and efficiency 2. Intellectual property and marketing strategy - Patent maintanance and PCT submission - Market analysis and commercialization plan

European patent submitted (EP24383089; priority date 08/10/2024) Pending EESR

This proposal involves two research group from two different institutions: IDIBELL and CSIC. This synergy ensures a multidisciplinary team who shares the ultimate goal of identifying and exploiting commercially a new Cas9 nuclease.

The DeepCas project aims to identify and validate a novel Cas nuclease in different model organisms, assess its uniqueness in the CRISPR-Cas market and exploit its commercial potential on different fields. After identifying the required sequences of a novel Cas9 system and testing its activity in vitro and on plasmids, its nuclease activity was validated in prokaryotes, invertebrates (C. elegans) and vertebrates (zebrafish, D. rerio). Our next objective is to evaluate toxicity, specificity and efficacy in C.elegans.