10 BIOTECHNOLOGY


artery SMCs, optimal transfection was obtained with an exponential waveform at 450V, 350µF, and 1000 Ω, whereas bronchial SMCs showed greatest transfection at 350V. Te optimal voltage to apply will be dependent on the application of the cells posttransfection. Te capacitance was varied from 200µF to 1000µF with voltage held constant at 350V. Te different cell types varied both in cell survival and in electroporation efficiency; however, the higher capacitance led to a higher transfection rate and had little effect on cell survival in both the pulmonary artery SMCs and aortic intimal SMCs.


Higher capacitance settings resulted in poor cell survival in bronchial SMCs; however, high transfection efficiencies could be reached by using a greater number of initial cells.


Both exponential and square waveforms were tested for the three different SMCs. Although square-wave electroporation was capable of transfecting SMCs, the efficiency was far lower than that of the exponential waveforms, so only exponential results are presented.


Te Opt mini 96-well/Sqr, Exp preset protocols on the Gene Pulser MXcell electroporation system were used in these experiments to begin to optimise electroporation-mediated transfection into three different primary human SMC types. Te ease of the system is such that multiple cell types can be simultaneously tested in one single experiment, limiting time, reagents, and effort. Te differential response of these SMCs of different origin to varying voltages and capacitance, in terms of viability and gene expression, highlights the fact that every cell is unique and that electroporation parameters cannot be simply applied from one cell to another..


Holly Reynolds and David A Dean are with University of Rochester Medical Center, Department of Pediatrics, Rochester, NY, USA. Bio-Rad Laboratories Inc is headquartered in Hercules, California, USA. www.bio-rad.com


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Orphan drugs to ‘create paradigm shift’


The economic recovery process has proved difficult for the pharmaceutical industry. Factors such as patent expiry, dry pipeline, and strict approval guidelines, have slowed down the attractive drug discovery and industry growth. Coupled with an investment risk in new therapy or molecules, this creates a challenge for the industry to overcome. However, in the midst of the current status, there seems to be a bend in the road – marked by the evolution of a paradigm shift in the industry: Orphan drugs. “While the pharmaceutical industries


“The Gene Pulser MXcell system was used to simultaneously test several parameters. Cell viability and transfectiion efficiency were recorded for each sample.”


have been focusing on ‘blockbuster’ small molecules (chemical drugs) for high revenue generation in the past, it is expected that in five years, around $90.0billion worth of branded drugs will lose their exclusivity,” finds Frost & Sullivan Healthcare consultant Shabeer Hussain. “The current economic situation plus the huge generic competition shifted the focus of pharmaceutical companies and they are moving to a new business model – ‘niche busters’, also called Orphan drugs.” This new business model will


provide an approach to an integrated healthcare solution, thereby enabling pharmaceutical companies to develop newer areas of therapeutics, diagnosis, treatment, monitoring and patient support. Orphan drugs provide attractive opportunities to reduce the impact of revenue loss due to the patent expiry for blockbuster drugs. Clinical trials for orphan drugs


are run efficiently with smaller patient groups, thereby reducing costs significantly. Incentives for drug development provided by governments, the FDA and the EU commission support in special protocols are a further boost for companies developing orphan drugs. Pharmaceutical and biopharmaceutical companies are also joining in partnerships by licensing products to maintain revenue. Such collaborations reduce the cost of developing and marketing orphan drugs. In numerous ways, a balanced, strong alliance will achieve mutual benefit to both pharma and biotech companies. While the focus on ‘niche busters’


grows, the safety regulations and approval processes will become stricter, putting larger companies in a better position to cope with the increasing demands. However, though smaller pharma-


biotech companies will struggle to compete with the more powerful competitors in the industry, Hussain believes there will still be assurance for niche players with specialist therapies, technologies, unique capabilities and expertise. Acquisitions at a global level, aimed


at specific niche capabilities with technologies, are likely to be the most effective way of achieving a better partnership and collaboration, as well as a more diverse client space.


For more information, visit www.frost.com


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