Biotech award for gene transport technology

For the development and commercialisation of its proprietary gene transport unit (GTU) technology and GTU product applications in DNA vaccination and immune therapy, Finnish company FIT Biotech has won Frost & Sullivan's 2004 European Excellence in Technology Award.

Among criteria considered for this award are the number of new technologies developed, their significance, ability to bestow competitive advantage, ease of adoption, potential to become an industry standard and overall impact in terms of shifting R&D focus.

"Significantly, GTU technology is applicable to DNA vaccination as well as to immuno and gene therapies for the modulation of the immune system via antigens or cytokines for use in the treatment of infectious diseases, cancer and allergies,“ explained F&S analyst Rajaram Sankaran. "The technology could also be used in the expression of specific genes in order to complement a genetic defect. This is useful in the therapy of diseases caused by mutations in a single gene such as Parkinson's disease or many dermatological disorders,“ he added.

Strong product line

According to F&S, by leveraging its GTU technology platform FIT Biotech has built a strong product pipeline that comprises a therapeutic HIV vaccine, a preventive HIV vaccine, as well as vaccines for melanoma, diabetes and hepatitis C. FIT Biotech's HIV vaccine programmes have already progressed to the Phase I/II stage.

FIT Biotech has also built an impressive intellectual property portfolio and has filed numerous international patents for its technology and the applications based on it, including novel vectors (GTU), medical devices (device and gene therapy), self-replicating vectors for DNA immunisation against HIV, AIRE (immunological mechanisms) and DNMT3L (immune diseases, new genes).

In conjunction with BD Technologies (a unit of Becton, Dickinson and Company), FIT Biotech is working on the development and delivery of HIV vaccines. A joint Phase I/II multi-centre clinical trial combines FIT Biotech's GTU-MultiHIV (investigational) vaccine with BD's disposable intradermal delivery device.

As part of a current public-private research partnership with the non-profit organisation International AIDS Vaccine Initiative (IAVI), FIT Biotech is testing the GTU-MultiHIV vaccine in human trials in Finland.

"Such early trials, should they prove successful, could set the stage for more large-scale tests and focus attention on a vaccine that has the potential to protect uninfected people from contracting the HIV virus,“ notes F&S.

Meanwhile, German company Girindus has won Frost & Sullivan's Product Differentiation Award for outstanding advances in the industrial production of therapeutic oligonucleotides.

Every year the consulting firm confers this award on a company whose corporate strategy and innovation give it a decisive edge over its competitors and whose differentiation strategies have had an impact on the maturation of the individual markets.

F&S selected Girindus after conducting an extensive industry-wide survey of oligonucleotide manufacturers. The award citation states that Girindus, a worldwide market leader in the manufacture of therapeutic oligonucleotides, holds a leading position on the basis of both its conventional solid-phase production methods and its recently-developed one-of-a-kind solution-phase process.

"For the first time, our innovative solution-phase process has gained full market recognition in addition to our solid-phase production,“ commented Fritz Link, Girindus ceo.

In the recently published analysis of its market research study, F&S said that Girindus now has enormous sales potential as a result of this product differentiation strategy.

Throughput screening

Pharmaceuticals and biotechnology companies are under constant pressure to discover and develop new therapeutics at a lesser cost and within shorter timeframes. However, with investments exceeding US$ 500 million and nine out of ten candidates failing to get drug approval, pharmaceuticals and biotechnology companies face a challenging task ahead.

High throughput screening (HTS) is emerging as a viable option as pharmaceuticals and biotechnology companies strive to become the first to market a new drug. HTS has the potential to provide mechanistic insights and new drug leads with unparalleled efficiency.

"By shortening the drug discovery cycle, HTS offers the opportunity to capture maximum market share and optimise revenue generation within the first year of entry,“ explains Research Analyst Dr Amarpreet Dhiman from Frost & Sullivan. "It is also likely to increase the chances of clearing clinical trials by addressing issues related to pharmacokinetics, animal toxicity and side-effects.“

The European HTS market was worth US$483 million in 2003 and is projected to be valued at

US$925 million in 2010. Germany is the largest market for HTS in Europe, with revenues greater than US$120 million of the market value.

For multinational pharmaceuticals and biotechnology companies that need to compress time and costs in drug development, outsourcing HTS has emerged as a cost-effective option that also provides access to additional drug entities and innovative technology. In fact, outsourcing is expected to form 57percent of total drug development expenditure in 2010.

The introduction of new technologies over the past decade has also proved instrumental in improving the chances of discovering innovative drugs and shortening the discovery cycle. For instance, integration of robotics with HTS has played a key role in achieving higher screening rates and avoiding expensive manual interventions.

Bioinformatics also speeds up the drug discovery process by predicting the biological properties of a particular lead compound and its viability as a potential drug candidate. When used in conjunction with bioinformatics, HTS can rely on quality lead compounds to generate considerable time and cost savings.

High Content Screening ­ an advanced form of cell-based assays ­ offers the potential to address and ease the many bottlenecks currently encountered in the drug discovery process.

"Insights from information-rich assays are likely to assist in the discovery of effective drugs and could also reduce the need for secondary screens in the future,“ says Dr Dhiman.

The role of HTS in heightening the probability of success in drug discovery is largely due to the support of several complementary technologies. For instance, combinatorial chemistry has increased the availability of targets and drug candidates through the acquisition of compound libraries.

"An increase in the quantity of compound libraries is essential to improve the quality of HTS,“ explains Dr Dhiman.

Advances in molecular and cellular biology, genetics, genomics and proteomics have also been a major driving force for HTS. New classes of therapeutic molecules have been developed due to the success of genomics and proteomics in providing an expanding list of cellular targets.

Although HTS has succeeded in generating a vast amount of data for propelling drug discovery, a significant void remains in terms of data storage, interpretation and analysis that are crucial for evaluating future drug candidates.

"The lack of validated statistical methods to ensure the integrity of the data obtained during the screening becomes challenging for most companies involved in drug discovery,“ notes Dr Dhiman. "In this regard, it is absolutely critical to improve statistical procedures for efficiently analysing the data generated during the HTS process.“

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