Purification of drugs key to their safety, success
Ajdovščina, 24 January - The Ajdovščina-based company Bia Separations is a world leader in the development of purification processes for biologics, in particular for gene therapy. According to chief scientific officer at Bia Separations, Pete Gagnon, purification is key to the safety and consequently to biologics' commercial success.
Having started to work for Bia on an occasional basis back in 2006, Gagnon became its chief scientific officer two years ago and left the US to move to Ajdovščina.
He is very enthusiastic about biology, saying it is a field both wierd and entertaining, giving an inquiring mind a great deal of satisfaction. Gagnon says that even after 40 years in this business, "it is still new everyday".
"If you are frustrated by not knowing what's going on or how something work, this is not the field for you. But if you like being in a situation when you're always discovering and leaning new things, things nobody has ever done before, then this is perfect. And I'm one of those people, after 40 years in this business I still can't believe people pay me to have such a good time," says Gagnon.
Developing technology to separate desired and non-desired components
The high-tech company Bia Separations has developed its own technology and tools for chromatography, a lab technique for separating various biological components which enables pharmaceutical companies to separate the desired components from the non-desired ones, thus purifying the drug. Bia Separations is the world's leading producer of CIM (Convective Interaction Media) monoliths for the pharmaceutical and chemical industries.
As Gagnon says, the "chromatography media have enabled us from the start to do things no other media could do before. This enables us to solve problems that could not be solved before. That's why this is so exciting"
Purified virus key to drug's success
Virus purification, a process in which medicinal compounds are separated from the harmful ones, which can often be very poisonous or even carcinogenic, is key to the safety of drugs.
The process represents a very high percent of a drug's production costs, according to the company. Bia Separations' smart media have become a part of registered drugs, which means its products will be used for the next several years.
One of the key challenges in purification technology is a rather high degree of loss of the product with which one starts. In recombination proteins, there are many products where there is only a 20% recovery of the product one starts with, or even less, whereas the wish is to maximise the recovery to 80-90%. "In purification of drugs there is always a battle between recovery and purity. You want both, but that's a major challenge."
Working on the medicine for the toddler Kris
The tools developed by Bia Separations are according to Gagnon particularly suited for purifying components for gene therapy, which is applied to correct flawed gene sequences in the DNA to cure genetic disease. Unlike classic medicines, these drugs do not use simple chemical compounds, but viruses which serve as a carrier of healthy genes.
Bia Separations has recently been in the spotlight for having cooperated on the development of Zolgensma, a drug for infants with spinal muscular atrophy. The currently most expensive drug on the planet - a treatment for one patient costs 2.1 million US dollars - is produced by US company Avexis under the wing of Novartis. The public has become aware of it because of a major humanitarian campaign for Slovenian toddler Kris which in just a few days raised the 2.3 million dollars for his treatment in the US.
Until Zolgensma was put on the market, spinal muscular atrophy was mostly treated with nusinersen, which is sold under the brand name Spinraza. The patient had to receive it every few months after the initial therapy, whereas with Zolgensma, a single dose should suffice.
Achievement which opens the door for everyone
Although the work on Zolgensma is one of Bia Separations' achievements that has recently achieved the most attention in the media and the public, Gagnon says "it is the fruit of a long chain of important technology achievments going back for many years".
The recognition of this genetic drug is, however, important for gene therapy, which has been trying to make a breakthrough for several decades. "With the new drug Zolgensma, which is coming to the market, everything will change, the doors will open for everyone," believes Gagnon.
Gene therapy also has the potential to replace the entire industry of monoclonal antibodies - biological medicines used for treatment of some types of cancers and autoimmune diseases, and to "enable things which the industry of antibodies could never achieve".
Cooperation with National Institute of Biology
Slovenia's National Institute of Biology, which which Bia Separations has worked ever since its establishment, has also worked on the development of Zolgensma with specific analytical techniques which are needed for precise characterisation of the drug.
The Institute of Biology, a pioneer in methods of defining and quantifying genetically modified organisms and microorganisms, including viruses, applies its specific analytical methods to check for Bia Separations the quality of drug development as well as the development and formation of virus processing procedures.