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Tailor-made miracle molecules from Radebeul

by Anke Wilde

Company start-ups require a generous dose of courage. Few scientists set up their own company after having discovered a future-oriented technology or molecule with long sought-after properties. However, Jacques Rohayem decided to make the leap three years ago and founded Riboxx. The French virologist discovered a way to create medically highly effective and at once stable ribonucleic acids (RNA) while working at the TU Dresden. Here you can read about an example of a successful start-up.

Tailor-made miracle molecules from RadebeulJacques Rohayem dared to start a company and to set himself up as a supplier for research institutes
At Riboxx, almost everything revolves around a type of miracle enzyme that can both iron out the creases and also stick molecules together to form a revolutionary product. Jacques Rohayem discovered the beneficial properties of this enzyme several years ago whilst researching full-time at the TU Dresden. His company has been supplying both research institutes and major pharmaceutical companies for three years now. The company employs a workforce of 17. The company is based in Radebeul, a small town in the state of Saxony, close to the city of Dresden, otherwise famous for being the birth town of adventure author, Karl May.

The Riboxx product range includes two groups of applications for the ribonucleic acids: the first group are long RNA, which are used as a vaccine amplifier, a so-called adjuvant. They change the body's immune response, thus improving the effectiveness of vaccines. Their fields of application are extremely diverse. "Their uses range from treating virus infections such as HIV and hepatitis C through to the prevention of bacterial diseases such as tuberculosis and even cancer," explains Jacques Rohayem.

The second group comprises short ribonucleic acids - molecular chains such as the so-called small interfering RNA (siRNA) to be precise. Tailored specifically to a disease, they can regulate cell processes and silence specific genes. Although only a few drugs have fully completed the approval process, molecular scientists and pharmacists have had high hopes of these short snippets of RNA for numerous years now. "In specific cancers, the appropriate siRNA molecules can attack the gene responsible for the formation of tumours and prevent it from producing tumour proteins," tells Rohayem. Should these proteins be absent, then the tumour cells cannot divide continually and escape their natural cell death either. The mechanism that stimulates the malignant growth in the first place is silenced with the help of these molecules.

The body defends itself against RNA molecules

The problem is, however, that ribonucleic acids are rapidly degraded by the body, Jacques Rohayem explains. "They only exist in the cell as so-called messenger RNA. As such, they serve the transcription of the genetic codes set by the DNA at the cell's core that manages the cell activities." They are degraded as soon as they have triggered all cell functions. Outside of the cell - so in the bloodstream or lymphatic system, for example - they are recognised as a poison extremely rapidly. Certain enzymes, namely the so-called RNases, take care of their disposal. It is a protective reaction by the body, which has a genuine purpose: viruses are also made up of RNA. With their cell-altering properties, viruses are, to a certain extent, the role model for the medical use of RNA.

It is difficult to introduce the active substances into directly the cells and tissues affected by the disease. Those wishing to take advantage of the healing properties of RNA snippets need more stable molecules. This is where the expertise of Riboxx comes in. With the help of its ironing and sticking enzymes, the biotechnologists give the tiny RNA molecules a new profile. Regardless of the exact chemical composition of the RNA, it only needs the one enzyme. Most single-stranded, spiral molecular chains are untwisted by the enzyme's special biochemical properties and fused with a symmetrical molecular chain. The result is a double-stranded RNA sequence that is not broken down by the body's protective system as quickly meaning active substances can be administered in lower doses.

Large pharmaceutical companies promise higher returns

From manufacture of the enzyme itself through to production of the molecules needed - everything takes place at Riboxx. Jacques Rohayem is extremely proud of his enzymatic processes. Without them, it would be difficult to control the rolled and knotted molecules, and to fuse them one to one, he says. The RNA snippets can now even be produced on an industrial scale. One gram of RNA is currently produced per litre, but according to Rohayem, this can be scaled up significantly more.

This naturally also increases the potential client base, which includes both research institutes, who only test the effect of the RNA molecules in the cell culture or mouse subjects and require accordingly small amounts, and the pharmaceutical industry, which manufactures tablets and vaccines. The number of clients has risen steadily since Rohayem obtained the patent for his TENPORA process and Riboxx came onto the market three years ago. More than 150 institutes and companies are now supplied from Radebeul. "It would be nice to win one or two contracts with the large pharmaceutical companies," confides Rohayem. Such major contracts would secure the turnover.

Generous start-up help for company start ups

Founding a company in the biotech industry is an extremely costly undertaking. Both the equipment as well as the highly-qualified staff are extremely expensive. Without financial support, Rohayem would not have been able to enter the market with its procedure. Hence the Frenchman only has good words to speak of the German research environment. He obtained the start capital for his start-up from the biotechnology start-up offensive (Gründungsoffensive Biotechnologie - GO-Bio) of the German Federal Ministry for Education and Research (BMBF). The European structural funds also provided financial means. Moreover, Rohayem obtained additional venture capital from Saxony's technology start-up fund, which supports future-oriented company start-ups in the free state with up to four million euros over a period of several years.

Rohayem sees the company start-up as a positive development for him personally. "The greatest challenge was making science into technology, technology into a product, and a work group into a company," he muses. It is for this reason that he has also returned to school in parallel to developing the company, and obtained a Master of Business Administration to also be able to operate a company and manage staff.

Company manager and scientist in one

All in all, a great many similarities exist between the work of a company manager and that of a scientist. In science, you must sell your research ideas and write proposals. "And in business, it is a product that should be successful on the market." "It's a great deal of fun," he enthuses. However, the business planning requires a comparatively lots of preparation. A good ten years can pass before a new drug is developed and released onto the market. Most medicines that rely on the small RNA molecular chains are still in the test phase, and it will only be clear after the clinical studies are concluded whether they will be actually approved. Yet only when such a drug is approved and Riboxx is commissioned with its production can the Radebeul-based company manufacture the required RNA molecules on a large scale. Rohayem remains hopeful and patient though. "You simply need to have staying power."

academics :: December 2012