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Reducing the cost, time, and ethical burden of preclinical research in the pharmaceutical industry

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New genetic tools streamline the drug development process

Drug discovery can take years, with huge costs for both pharmaceutical companies and patients. The GENBIO project created two new tools which could revolutionise this critical process.

Health

Centuries of medical revolution have provided humans with strong pharmaceutical defences against diseases. Recent technological advances also mean scientists can more easily identify and define them. Yet the drug discovery process, to create and test new treatments, remains expensive, inefficient and time-consuming. Drug development can take 10-14 years, and the average cost runs into billions of euros. The use of animals adds to ethical burdens, raising development costs further. “In the end, the inefficiencies in drug discovery and validation don’t only impact the bottom line of pharmaceutical and biotechnological companies, but also translate into higher prices of medicines for patients,” says Jaya Krishnan, co-founder and CTO at Genome Biologics, a biotechnology company in Germany. The EU-funded project GENBIO targeted the key bottleneck in drug discovery, the period from early success to final validated drug candidate ready for preclinical trials. The new system streamlines this process, making it both more efficient and ethical. “We have developed a completely new patented drug discovery and validation workflow,” explains Krishnan, GENBIO project coordinator. “Most importantly, we have discovered more than 10 new heart disease therapies, some of which we now want to develop further through clinical studies,” he adds.

Two innovative genetic tools working in tandem

GENISYST is a genetic tool that allows for the creation of complex human disease models to test new drugs. GENISYST can be used in lab-grown human heart organoids, or with animals both small and large at a fraction of the cost and time. It uses viruses loaded with DNA instructions to alter the RNA expression of animal cells, mimicking disease without the need to change the underlying genome. Multiple genes can be expressed or suppressed in one animal, rather than breeding many with altered individual genes. “Complex disease models can be created in ‘one go’ in a single animal and in a fraction of the time,” notes Krishnan. The tool can reduce animal numbers by a factor of over 100, preclinical study costs by a factor of 10 and the time needed for studies by a factor of 6-8. Coupled with this is GENIMAPS, software created in the GENBIO project. GENIMAPS combines big data and artificial intelligence to rapidly match the right compound needed to treat heart disease. The AI algorithms allow the team to identify new drugs at speed, which are then tested and validated through GENISYST models. New targets for heart disease have already been found, and published in several leading scientific journals: ‘Nature Reviews Cardiology’, ‘Cell’, ‘Science Translational Medicine’ and ‘Circulation’.

Gaining interest in the pharmaceutical industry

The technologies created in GENBIO have drawn interest from pharmaceutical companies. A new research collaboration is under way with AstraZeneca, exploring the underlying drivers of heart regeneration and recovery following heart attacks, heart failure and injury. The GENBIO team has filed nine patent applications and won several awards recognising their innovation in the biotech space. They are now seeking additional support from big pharma, aiming to create further jobs and opportunities within the EU. “We believe that our technologies and drug pipeline have the potential to disrupt and revolutionise treatments for heart disease and tackle the larger burden of cardiovascular disease in the EU,” says Jonathan Ward, co-founder and CEO at Genome Biologics.

Keywords

GENBIO, drug, discovery, heart, disease, genetic, tools, innovative

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