“Research is always a lot of failures and a few successes”
Av It was long considered a utopia – that one could cure severe hereditary diseases by repairing the faulty gene that caused them. But in recent years, a number of effective gene therapies have been developed, and today it is fully possible to cure, for example, small children suffering from spinal muscular atrophy or patients with severe hemophilia.
However, there is a catch: the cost. Gene therapies cost millions. For example, the price of the above mentioned therapy for spinal muscular atrophy is over 20 million kronor for a single treatment, and the one for hemophilia costs almost twice as much.
In the five-year project Genenova, which is funded by Sweden´s Innovation Agency, Vinnova, and several partner companies within the industry, Johan Rockberg and the other researchers are developing methods to both reduce production costs and make it possible to more quickly develop new therapies for patients with often rare genetic diseases.
“On one level, it is a very engineering-oriented challenge: How do we make a little more medicine a little cheaper? But on the other end, it is about how we gain a better understanding of the relationship between human cells and viruses”, says Johan Rockberg.
The technology used involves a harmless adeno-associated virus (AAV) that delivers DNA to replace missing or damaged genes in the patients' cells. But to produce the virus, human cells are needed. The problem is that these two components are hardly compatible. Or, as Johan Rockberg puts it, “human cells hate viruses.”
One of the major challenges for researchers is therefore to find suitable methods to turn off the immune system functions that cells normally have to avoid spreading viruses, while keeping the cells in a good enough mood to produce as much as possible.
In the project, researchers are looking for new, smart solutions in areas such as the production of starting material, i.e., DNA, production of vectors, separation of these vectors from the cells, and quality control.
This is not a simple task, and the project includes prominent researchers from various fields, from hardware, mathematics, and AI to cell biology, immunology, and transcriptomics – to name a few.
New Horizons Conference
On December 12, Johan Rockberg will be moderating the conference New Horizons in Biologics & Bioprocessing at Life City in Hagastaden, Stockholm. Read more at bioprocessing.se.
Several Swedish companies contribute to the project with their cutting-edge technologies. For example, the researchers utilize Uppsala-based Biotage's method for purifying plasmid DNA, Lund-based Ziccum's air-drying through laminar pace technology, Qutem's services in advanced electron microscopy, and Alfa Laval's separation technology.
KTH's own bioreactors and expertise in bioproduction will be used when scaling up production.
You are now just over halfway through the project. How is it going?
“It's going well, even though research is always a lot of failures and a few successes. When we put together all the pieces we've worked on, I would say that if we are halfway in terms of time, we are certainly halfway in terms of achievements as well. So I am hopeful“, says Johan Rockberg.
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