Genetic scissors CRISPR/Cas*: prospects of curing many diseases

The new annual report of the Commission of Experts on Research and Innovation (EFI), presented to the chancellor virtually on account of the pandemic, deals with the key future issue of the genetic scissors CRISPR/Cas in medicine. In a video podcast, Professor Carolin Häussler, holder of the Chair of Organisation, Technology Management and Entrepreneurship at the University of Passau, describes the outstanding significance of this still young technology to the EFI Commission: "CRISPR/Cas is a gene-editing tool which is giving basic medical research fresh momentum and enabling new therapeutic approaches to be developed for many diseases. The target-specific modification of genetic information will make it possible to eliminate the cause of hereditary diseases directly." 

Professor Häussler went on to say that there was great potential in somatic gene therapy, i.e. in applications in which the genetic changes brought about relate only to the individual under treatment. A high level of benefit for the patient is accompanied here by economic added value potential. She said that realising that potential would require considerable further progress, both in research and in the transfer of results from research to application. The genetic scissors CRISPR/Cas, she said, could be used not only for medical purposes, but also deployed in agriculture and industrial applications; the EFI, she said, had so far concentrated on medicine alone.

"With the aid of CRISPR/Cas, genes can be modified or switched on or off. This creates new possibilities for treating hereditary diseases that affect millions of people. The researchers ascribe great potential to CRISPR/Cas, because it simplifies gene editing and thus greatly widens the circle of researchers and applications", emphasises Professor Irene Bertschek of the Leibniz Centre for European Economic Research in Mannheim (ZEW). She added that this was leading to a marked increase in research and development activities relating to CRISPR/Cas, and that most of the current development work being done on medical applications of CRISPR/Cas were regarded as giving no cause for concern in ethical terms.

Transparency and the building of public awareness are of enormous importance. People should be able to face these developments in an informed and open-minded manner. I'm sure that in this way, people's fascination with science in society can be given a great boost."

Professor Carolin Häussler, University of Passau

The EFI has recommended the following measures to the federal government:

• Speed up authorisation procedures:  the authorisation procedures relating to all aspects of CRISPR/Cas and the clinical studies conducted on it ought – "always under the precept of maintaining safety and ethical acceptability" – to be organised in such a way that the administrative burden on researchers is reduced. Related applications and authorisation procedures should be able to be combined. Furthermore, efforts should be made to harmonise authorisation procedures beyond the borders of the respective federal states, and "also to adjust the number of staff in the approval authorities as appropriate at an early stage", says Professor Häussler.
   
• Reinforce top-level research on CRISPR/Cas:  In order to reinforce top-level research on CRISPR/Cas, a number of lighthouse projects at internationally competitive German locations should be expanded or created. In these lighthouse projects in particular, the transfer of scientific insights to medical application should be given high priority.
   
• Improve the transfer of scientific insights to praxis: According to the EFI, it is true that internationally Germany is up among the front runners in the research being done on CRISPR/Cas, but it "falls behind badly when it comes to the transfer to application and commercialisation", says Professor Bertschek. Cooperations and working groups should definitely be initiated and funded, and via early interaction between research and clinical praxis they should facilitate the transfer to application and generate innovations. To provide researchers with consultancy facilities and an appropriate network, the EFI recommends a discussion on the establishment of a German gene therapy centre, which could adopt the role of a competence centre for the transfer from basic and pre-clinical research to clinical application.
   
• Improve framework conditions for the provision of venture capital:  The prolonged research and development cycles in medical biotechnology are accompanied by tremendous financing requirements and a high level of risk. Once again, the EFI emphasises that the framework conditions for the provision of private venture and growth capital need to be improved. In that context, it welcomes the establishment of the "future fund", which is intended to support both pioneering technologies – especially in the field of biotechnology – and major financing rounds for start-ups and their scaling. "We call for this to be implemented as soon as possible", says Professor Uwe Cantner of the University of Jena, who is also chairman of the EFI.
   
• Keep society informed:  The EFI considers it very important to inform society at regular intervals about the potentials and risks associated with CRISPR/Cas, and to pursue public discussion on the subject. In conclusion, Professor Häussler said: "Transparency and building public awareness are of tremendous importance. People should be able to face these developments in an informed and open-minded manner. I'm sure that in this way, people's fascination with science in society can be given an enormous boost."

Source: EFI administrative office

The Commission of Experts for Research and Innovation (EFI), headquartered in Berlin, has been giving the federal government scientific policy advice since 2008, and submits an annual report on research, innovation and technological performance capability in Germany. The main tasks of the EFI are to analyse the strengths and weaknesses of the German innovation system in international and time-scale comparison, and assess the perspectives of Germany as a research and innovation location. On this basis, the EFI elaborates proposals for national research and innovation policy.