Welcome to our latest newsletter, which arrives at a time when gene therapy is high in profile with a number of positive press reports on gene therapies in the last few weeks.

This product class has been in the balance for the last few years, with the potential recognised but the risks deemed high. At Eden we have first hand experience of the strength and depth of the current research in this area, both in the UK and beyond. What’s currently lacking is commercial and investment interest. However, if positive press continues and a market approval is granted this could rapidly change.


Ark Therapeutics
Although Sibono, a private company based in China has received approval for the first gene-based medicine, Ark Therapeutics could become the first Western company to get a gene-based product approved by regulators.

Listed on the London Stock Exchange in March 2004, Ark is working on a gene-based medicine for Glioma, Cerepro(TM), which helps stop cancer returning to the brain after a tumour has been removed by surgery. The company recently announced that it has filed its response package to questions raised by the EMEA’s scientific committee as part of the marketing authorisation application (MAA) review process. The application for marketing approval was submitted by Ark in the second half of last year and, following validation of the submission by the EMEA, was accepted for review in October 2005.


US National Cancer Institute
The journal Science has reported positive gene therapy results by a team at the US National Cancer Institute led by Dr Steven Rosenberg, a pioneer in the field. This technique has eradicated cancer from two dying men using genetically modified versions of their own cells. Both were suffering from advanced melanoma but it could be customised to attack other common cancers.

Scientists remain cautious although the results show, in principle at least, that aggressive cancer can be treated this way, even after it has spread and the prognosis is bleak. However, of the 17 patients with advanced skin cancer who underwent gene therapy, the treatment only worked on two people.


Introgen Therapeutics
Based in Texas, Introgen Therapeutics also has a product waiting for regulatory approval. It plans to register its lead product, Advexin, in the US for head and neck cancer over the next few months. The company has already benefited from the interest of Colgate–Palmolive, the consumer giant, which bought a 9.7 per cent stake as part of its plan to develop a mouthwash to prevent oral cancer.



Behind every successful business there’s a strong team of women running the show – and the ladies at Eden are no exception! Stealing the biggest headline for this edition is without a doubt the successful completion by the eight strong team in the Hydro Active women’s challenge (5K) on 3rd September at Sefton Park (on behalf of MacMillan Cancer support).



Congratulations are due as together they raised over £1000, with some of this sponsorship coming from the link in the last newsletter – so thank you to all those who supported them.

In terms of other news; in the last few weeks we are delighted to have been able to announce a new contract with Aquapharm Biodiscovery to develop a biopharmaceutical protein from a novel marine organism. We have also begun work on two further client projects in the development labs – bringing the total to six.

Alistair Darling has confirmed that he will officially open the NBC facility on 1 November 2006. However we officially take over the cGMP facility at the beginning of October, with the last of the contractors leaving the facility following completion of validation. By the end of that month we will employ approximately 50 people! – which is some growth from 11 in December last year!



European Biopharm Scale-Up Conference
Geneva, 12-14th September
The times have changed at scale-up conferences. In the 1980’s, the meeting would be dominated by engineers talking about novel fermenter designs and process scientists like myself struggling with mg/Litre antibody titers. Today it seems the focus is largely biology, despite advocates for perfusion fermenters, airlift fermenters and the like, the basic stirred tank fermenter design remains the work horse of the Industry. According to Professor Florian Wurm of ExCellGene, Switzerland metabolic engineering of CHO cells and physiological optimisation rather than novel vectors was seen as giving us assurance of 25 g/L fermenter yields (Yes, I have not missed out a decimal point) routinely by the end of the decade. This was supported by an excellent presentation by Professor Rainer Fischer, the Department Head of the Fraunhofer Institute for Molecular Biology and Applied Ecology. Professor Fisher is also a strong advocate of the advantages of transgenic plants. Although his enthusiasm was tempered by his recent experience in discussion with the EMEA who want a “comparable level of scientific predictability to that of CHO cells”.

The second major focus was purification. If we obtain g/L fermenter harvests how do we improve purification yields? A common worry expressed by several speakers was how to cope with the volume of buffer needed and the practicality of holding tanks and WFI requirements. The future holds better chromatography resins but, according to GE Healthcare Biosciences, even the possibility of employing a dual chromatographic step procedure using their novel Protein A resin combined with novel anion exchange resin. The reduced steps offer major advantages in time and cost of goods. But the highlight for me, with a fondness for multivariable process optimisation experimental design, was from Ache Stockerman, Principle Process Scientist at Amgen. This combined Bioprocessor’s SimCell system, a sophisticated combination of robotics with microfluidic cultures, along with Tecan automated Elisa plus Guava system for cell counts and viability measurements. The result, process design where ‘n’, the number of experimental conditions, exceeded a thousand resulted in a complete map of the interaction of experimental conditions on the likes of product yield. Not cheap but if the promise is borne out this could fundamentally change cell line selection to establish a cell bank. Or for that matter perhaps provide the data to support major process change.

Crawford Brown