Next meeting ACTIP:

NEXT MEETING:
20th Anniversary meeting ACTIP (Alicante Spain)
May 26-27-28, 2010

hosted by the ACTIP Steering Committee, Executive Secretary and ACTIP Secretariat.

This meeting, the 20th anniversary of ACTIP, will review science, technology and business aspects in animal cell technology both in the past and in the future.
It will also have a session on product and process quality.

For ACTIP member companies and invited guests only.

In this issue:
European News
Vaccine News
Business News
Collaborations and mergers
Biomanufacturing News
Research News
Stem cell news
Clinical trials and results
Regulatory News
EDQM and EMEA News
Agenda

European News

EU chief scientist tipped for 'TV role'

In September, European Commission President José Manuel Barroso announced plans to appoint a Chief Scientific Advisor to assist the incoming EU executive in making tough decisions on everything from GMO authorizations to addressing climate change (EurActiv 25/09/09). The new job, to be created after the new-look EU executive is sworn in, forms part of an ongoing period of major reflection within the Commission, which is re-organizing its directorates in charge of science, research and innovation policy. 
Numerous scientific committees are already providing the European Commission with advice on policy decisions or proposals related mainly to consumer safety, public health and the environment. In addition, specific EU agencies, such as the European Food Safety Authority (EFSA) or the European Centre for Disease Prevention and Control (ECDC), have been set up to handle particular technical and scientific tasks. Further advice for EU policymaking is provided by the Commission's in-house research facilities, the Joint Research Centre (JRC), the Bureau of Economic Policy Advisers (BEPA) and the European Group on Ethics in Science and New Technologies (EGE). The European Research Area Board (ERAB), a high-level, independent advisory committee created by the Commission, also provides advice on the design and implementation of EU research policy. The EU executive also receives ad hoc advice from a number of 'comitology' committees regarding the implementation of EU legislation in national administrations. 
John Wood, who chairs the European Research Area Board (ERAB) — which proposed the creation of a Chief Scientific Advisor — sees the role as a public advocate on scientific issues. He says the advisor should be independent-minded and feel free to give evidence-based advice. It should be a person who can go on television during times of crisis, such as a pandemic, and help communicate on scientific matters in a way that engages the public and allays fears. "The Chief Scientist for Europe should be someone who speaks for science — someone who goes on TV during a crisis," he said. 
This follows the model of UK  and US scientific advisors. However, there appears to be a major gulf between this Anglo-Saxon model and what might be envisaged by Brussels. High-level science policymakers in the EU institutions view the role less as an independent voice of science, and more as a scientifically-savvy confidante of European Commission President José Manuel Barroso. Wood said the job would be less attractive to top scientists if it were merely a backroom policy position rather than a high-profile advocacy role. 
Source: Euractiv News, December 9, 2009

Barroso names Europe's first innovation commissioner

Irish Máire Geoghegan Quinn has been appointed as the first Research and Innovation Commissioner. The job is an expanded version of the old science & research post, and comes just months before the first European Innovation Act is due to be published. Outgoing Science and Research Commissioner Janez Potocnik will take up the environment portfolio. The education portfolio has been handed to outgoing Health Commissioner Androulla Vassiliou, who will now be responsible for the Marie Curie Programme unit, which previously rested with DG Research.
The creation of an innovation commissioner has been well flagged, with European Commission President José Manuel Barroso having repeatedly pledged to streamline innovation policy (EurActiv 24/09/09). One element of the new portfolio which remains unclear is precisely how the move affects the Commission's DG Enterprise — now called 'Industry & Entrepreneurship' — which is currently charged with drafting the Innovation Act. 
The Commission has also revealed that the new European Institute for Innovation & Technology (EIT) will be run by the education commissioner. This too is an initiative likely to be of interest to Geoghegan Quinn. The EIT is seen as a pet project of President Barroso and has recently moved into its new headquarters in Budapest. The first of its flagship 'Knowledge and Innovation Communities' (KICs) have been announced on 16 December.
Source: Euractiv News, November 30, 2010

EIT launches first 'Knowledge and Innovation Communities’ 

The European Institute of Innovation and Technology (EIT) has unveiled three major new innovation clusters focusing on climate, energy and information technology. Each of the initiatives will bring together academia and industry at several locations across Europe. The Knowledge and Innovation Communities (KICs) will receive start-up funding of €3 million and will report to the EU's commissioner for education, culture, multilingualism and youth. Contracts have been signed for 7 years.
The EIT headquarters in Budapest is expected to be fully up and running by April 2010, but the KICs will be autonomous and will be run "like a business", according to the Institute's chairman, Martin Schuurmans. He said the new clusters would be results-driven and their impact will be measured back on concrete outputs. Schuurmans said the winning bids were chosen based on excellence and the promise of strong independent leadership. Entrepreneurship will be key to their success, he added. "The KICs will be like small companies, they'll do their own thing. We will give help and advice but they have their own leadership and they'll stand on their own two feet," Schuurmans told a media briefing in Budapest. 
The KICs will receive 25% funding from the EIT and are expected to be self-sustaining in the medium term. They will be free to attract financial support from private sources, national funding agencies and EU research programmes. 
The Climate-KIC will look at water and land use, as well as greening Europe's cities and improving climate forecasting systems. Its aim is to become "the natural place" for companies to locate climate R&D centres and to become a magnet for top students of climate change. It will have centres in London, Zürich, Berlin, Paris and Randstad (the Netherlands). 
The so-called 'InnoEnergy' consortium is expected to focus on sustainable energy and will have bases in Karlsruhe, Grenoble, Eindhoven/Leuven, Barcelona, Stockholm and Krakow. The Polish centre is the only one to be located in Eastern Europe. 
The final KIC will focus on information and communications technology and links together existing clusters in Berlin, Eindhoven, Helsinki, Paris and Stockholm. 
Next steps:
Jan. 2010: KICs will be awarded start-up grants of €3 million. 
April 2010: The EIT's offices in Budapest will be fully operational. 
Source: EurActiv News, December 18, 2009

Vaccine News

Vaccine makers did not hype H1N1, says EVM

The EFPIA’s European Vaccine Manufacturers (EVM) group has denied allegations that its members were involved in hyping up the threat of the H1N1 pandemic to boost vaccine sales.
The EVM group, representing Baxter, Crucell, GlaxoSmithKline, MedImmune, Novartis, Sanofi, MSD, Solvay and Wyeth, rejected the allegations made in the “Faked pandemics – a threat for health” motion put before the Council of Europe (CoE).
It said that: “The assertions in this motion are unfounded and result from a misunderstanding of the pandemic planning process and collaboration between public and private health partner.”
The group, which is part of the European Federation of Pharmaceutical Industry Associations (EFPIA), also confirmed it will attend a CoE hearing on January 26 to address the matter further.
Source: www.In-pharmatechnologist.com, January 19, 2010

Insect cells could cut vaccine production to 10 weeks

In a paper published online on 29 December in Biotechnology Journal  Austrian researchers detail why they believe insect cell derived influenza virus-like particles (VLPs) “are a very fast, safe and effective alternative vaccine approach”. Using two insect cell lines, namely Sf9 and BTI-TN5B1-4, the team was able to produce the first batch of VLPs 51 days after cloning work began. Accounting for an additional two weeks of gene synthesis, vaccine production of a new influenza subtype could start less than 10 weeks after isolating the RNA sequence. In earlier work published in The New England Journal of Medicine Baxter claimed it could produce a H5N1 vaccine within 12 weeks of an outbreak. This used African green monkey kidney cells and the Austrian researchers draw parallels between mammalian and insect cell culture.
In tests on mice the Austrian researchers claim the VLPs they produced from both insect cell lines induced high titers of neutralising antibodies, leading them to believe that the vaccine could be effective. Although both cell lines quickly produced effective VLPs the researchers believe BTI-TN5B1-4 has advantages over Sf9, in part because it decreases protein and baculovirus background. This reduces DNA contamination in BTI-TN5B1-4. Furthermore, cells derived from BTI-TN5B1-4 are used in the production of GlaxoSmithKline’s Cervarix [human papillomavirus (HPV) bivalent (types 16 and 18) vaccine, recombinant]. Cervarix has been approved by the European Medicines Agency (EMA) and US Food and Drug Administration (FDA), establishing a regulatory precedent for therapeutics made using BTI-TN5B1-4.
Source: www.In-pharmatechnologist.com, January 11, 2010

Vaccine market worth $52bn in 2016

Global vaccine revenues will be $52bn (€36.2bn) in 2016, more than double the total for 2009, driven by new products and indication expansions of established therapies, according to a report.
GBI Research reports that companies in the vaccine sector will be competing for a slice of a $52bn market by 2016. The report valued the market in 2009 at $24bn and predicts it will have a compound annual growth rate (CAGR) of 11.5 per cent until 2016.
Underpinning growth is increased investment in R&D following the success of premium priced products, such as Gardasil (human papillomavirus (HPV)), Cervarix (human papillomavirus) and Prevnar (pneumococcal conjugate), which became the first blockbuster vaccine.
Financial support of R&D is predicted to lead to the launch of novel vaccines. GBI states that companies are focusing on product differentiation to ensure the success of their innovative vaccines. A number of vaccines in development fit this model.
In disease sectors served by multiple vaccines companies are using new technologies to increase prices and boost profits. Pharma is also looking to maximise return from other vaccines through indication expansion.
Away from the big pharma players there are a number of smaller companies with novel vaccine technologies and promising candidates. Many of these firms lack the financial strength to bring a product to market and consequently are expected to enter into licensing and alliance agreements.
Source: www.In-pharmatechnologist.com, January 18, 2010

Rotavirus vaccine could save millions of lives

Two studies in Africa and Latin America published 28 January in the New England Journal of Medicine (NEJM) verified the effectiveness of rotavirus vaccines, which have so far been trialled only in developed nations and richer developing countries (see Double vaccine victory over rotavirus).The NEJM studies — in Malawi, Mexico and South Africa — are the first to show that the vaccines work in the world's most impoverished areas too. If included in vaccination programmes worldwide, such vaccines could save millions of children in the developing world, say the authors.
In the first study researchers vaccinated almost 5,000 infants in Malawi and South Africa with GlaxoSmithKline's Rotarix vaccine. Overall, it was 60 per cent effective at preventing cases of rotavirus diarrhoea and 30 per cent effective at preventing all kinds of diarrhoea.
The second study was carried out in Mexico, which introduced a rotavirus vaccine — again Rotarix — in 2007. Using public health data provided by the Ministry of Health, the researchers found that deaths from diarrhoea dropped after 2007, reaching a reduction of two-thirds by 2009.
In June last year the WHO Special Advisory group recommended that rotavirus vaccines be included in all national immunisation programmes. Developing countries rolling out the vaccine will face many challenges, including procuring additional infrastructure, such as refrigerators; sorting out supply chain logistics and training nurses.
Link to full African study in the New England Journal of Medicine
Link to full Mexican study in the New England Journal of Medicine
Source: SciDevNet.com, February 1, 2010

Gates promises US$10 billion for vaccine push

The Bill and Melinda Gates Foundation has pledged to spend more than US$10 billion on vaccine development and deployment in the next decade.
The funding, more than double the amount donated to vaccines by the foundation over the previous decade, was announced at the World Economic Forum in Davos, Switzerland,  on 29 January. It will go towards providing 90 per cent vaccine coverage in target countries and aims to prevent the deaths of over eight million children in the next nine years, said Melinda Gates. Together with the Global Alliance for Vaccines and Immunisation (GAVI) — which will receive a significant proportion of the money — the foundation aims to take a "comprehensive approach, from the research and development end right down to the delivery end", said Julian Lob-Levyt, chief executive officer of GAVI.
The money will be spent on the development of new vaccines for infections such as HIV, malaria, tuberculosis, pneumococcal pneumonia and rotavirus — which causes severe infant diarrhoea — and on scaling up the delivery of vaccines such as diphtheria, pertussis and polio (DPT) in the developing world.
Previous funding from the foundation has mainly gone towards basic research and development of vaccines, and the news that this time deployment has been taken seriously is very good news to public health communities and the general public. However, it is widely believed that the funding should be complemented by support from development agencies and governments for research into health systems. This would strengthen the health systems which will deliver the vaccines.
Source: SciDevNet.com, February 1, 2010

Business News

Market for Cell Culture products to expand further

The more than 400 biopharmaceutical products in clinical trials are driving growth in the cell culture products market. The current industry focus on three product categories—vaccines, stem cell therapeutics, and biosimilars — all of which will rely on cell culture products, mean that the prospects for growth in cell culture sales are good.
There are three categories of cell culture products on the market — media, sera and reagents. Media represents the largest portion, about 40% of the marketplace. Serum-based media is widely used to grow a broad range of animal cell types and cell lines such as Chinese hamster ovaries or murine myeloma cells. The most common media used for microorganisms, primarily used for the growth of bacteria, is Lysogeny broth, a nutritionally rich medium.
Most current animal cell-based cultures require serum supplementation, which is commonly used as a supplement to growth medium. As a result of the shift from traditional serum-supplemented media to serum-free and animal product-free media, sera sales are decreasing. Sales in this segment once enjoyed increases of about 10% annually, but are now slowing to annual increases of only about 3%. As cell culture suppliers continue to make the transition to serum-free products, the number of effective serum-free and specialty cell cultures on the market is rapidly increasing. New products introduced under the major brands are all serum-free formulations.
Reagents, or cell culture media components, represent about 38% of product sales in the sector. The reagents segment of the cell culture market is poised to experience big gains. Companies and research laboratories that work with cell culture want to grow cells cost efficiently, at the same levels as are possible with sera products but in serum-free environments. Typically, this requires the addition of growth factors, lipids, substitutes for transferrin to replace its iron-carrying function, alternatives to albumin that can replace its carrier properties, compounds that can replace the detoxification properties of serum, and specific attachment factors.
Suppliers are encouraged to pay closer attention to traceability of media components, which is being achieved through more frequent audits of vendors to determine the source materials and processes used to develop the product.
The world cell culture market is expanding at double-digit rates and is expected to maintain this momentum through 2013. Worldwide sales are estimated to have reached $1.87 billion in 2008, up 12% from 2007. Cell culture sales are expected to increase at a compound annual rate of 13% over the forecast period, 2003–2013, to reach $3.4 billion in 2013.
The cell culture market will continue to be strengthened by the development of culturing protocols and technologies for stem cells and by biomedical research.
An additional driver of the cell culture market will be generic biopharmaceuticals, also referred to as biosimilars, follow-on proteins, biogenerics, comparable biologics, follow-on biologics, or generic biologics.
A possible threat to the future of the cell culture market is the production of biopharmaceuticals in transgenic animals and plants.
Finally, a greater call for a faster vaccine-production method is predicted. The distribution complications associated with H1N1 vaccines, which are still egg-based and labor-intensive, made it abundantly clear that alternative methods are necessary. Vaccines produced using cell culture will be an important driver of the market in the near future.
Source: GEN News, Jan 15, 2010, review by Bruce Carlson

Pharma industry prepares for end of 'blockbuster medicines'

There has been much angst in the pharmaceutical sector in recent years as the pipeline of new groundbreaking medicines for common illnesses appears to be drying up. This comes at a time when advances in gene therapy and personalized medicine suggest the days of finding a mass-market drug to sell to large volumes of patients may be coming to an end. 
Dhavalkumar Patel, of the Novartis Institutes for Biomedical Research, said the future of innovation in healthcare lies in finding new medicines that treat several rare illnesses, but this takes longer than simply developing a single drug for a major disease. 
Patel told a healthcare conference in Brussels on 3 December 2009, hosted by the Friends of Europe think-tank, that US regulators grant longer "data exclusivity" periods than the EU, allowing pharma companies to search for new applications for their products. In the US, the period of data exclusivity is ten years; in the EU, the maximum period is just five years, he said. It's just too expensive to develop a drug for a condition that affects 1,000 people worldwide – that wouldn't be sustainable. But many conditions are influenced by the same biological pathway so if one molecule could treat several rare conditions, it becomes sustainable," he said. 
Source: EurActiv News, December 3, 2009

Biotech drug database goes free and public 

The UK's Wellcome Trust has announced that an online database of drugs that it bought in 2008 is now officially launched as a free, open-access concern.
ChEMBLdb has information on the properties and activities of over 500,000 small molecules, their quantitative properties and bioactivities (binding constants, pharmacology and ADMET etc). The data is abstracted and curated from the primary scientific literature. It was transferred for £4.7 million from biotech firm Galapagos NV in July 2008 - since then, the paywall has been taken off and around 100,000 more compounds added by the European Molecular Biology Laboratory's European Bioinformatics Institute (EBI), which hosts the database.
At the moment there are several ways to search the data, the most important of these are:
* Searching target data via keyword, BLAST, and soon to be by protein family-based search.
* Searching compound data via keyword, substructure, and similarity search.
The hosts positively encourage feedback on the interface and search capabilities, the identification and reporting of errors in the data, and general queries. Keep up to date with ChEMBL news and data releases by subscribing to the chembl-announce mailing list. Details for those interested can be found at the database, and at team leader John Overington's blog.
Source: Nature Blog: The Great Beyond, January 9, 2010, posted by Richard van Noorden

Another free database of potential malaria drugs by Glaxo

Pharmaceutical company GlaxoSmithKline (GSK) has set an example to other drug companies by making its database of potential malaria drugs public, says a Nature editorial. The move last month (January), provides access to data on 13,500 malaria drug candidates and marks an unprecedented open attitude to data sharing from big pharma (see Glaxo to share malaria drug data).
The editorial applauds GSK's chief executive, Andrew Witty, for showing leadership in this attempt to re-engage with the neglected diseases that primarily affect developing countries. It suggests that more drug companies should embrace a similar attitude towards these diseases, as they present a "low risk area" for experimenting with open data.
Other institutions also have a role to play, argues the editorial. Academic organizations must provide the infrastructure for archiving open data, and universities should support research that can develop drug leads such as those presented by GSK. And both should be more open. The editorial accuses academics and their institutions of being among the "worst offenders" in terms of "hogging intellectual property", and concludes by suggesting they follow GSK's lead and allow royalty-free use of technologies for good causes.
Link to full article in Nature
Source: SciDevNet.com, February 2, 2010

Biotech start-ups maximise the value of R&D outsourcing

Biotechnology start-ups are starting to outsource the early stages of research and development, such as drug discovery, according to a new study by the Cass Business School in London. Traditionally, it was thought to be too risky to outsource this type of research, but the study of 68 UK biotechs shows new managerial techniques are overcoming these difficulties. The approach will become increasingly important as more scientific procedures become standardised or industrialised. Outsourcing requires managers with a lot of experience to be able to manage projects at a distance and consider the risks that are involved. It relies very much on their project management skills.
Being able to outsource the early stages of research and development allows companies to save money and provides access to resources and knowledge that were previously unavailable or unaffordable. Small biotechs are also able to take advantage of high quality public science labs.According to the report, another crucial benefit of outsourcing is the opportunity to work internationally. Companies are now able to capitalise on knowledge and, in many cases, more cost-effective resources abroad, in countries in Eastern Europe, China, Russia and India, for example.
Source: Science Business, December 17, 2009

Collaborations and Mergers

BioInvent and ThromboGenics Win “Licensing Deal of the Year”

BioInvent International AB and ThromboGenics NV announced  they have won “Licensing Deal of the Year” at the Scrip Awards 2009, presented on Nov 18. The award recognises the major partnership deal that BioInvent and ThromboGenics signed with Roche for their novel anti-cancer monoclonal antibody TB-403 (anti-PIGF). It acknowledges the achievement of both companies in crafting a licensing deal that has both monetary and strategic benefits to all parties.
The Scrip Awards are one of the biotech and pharmaceutical industry’s most prestigious and highly contested international awards, and they are chosen by a panel of senior executives from the biotech and pharma industry.

Under the terms of the deal with Roche, BioInvent and its co-development partner ThromboGenics received an upfront payment of €50 million, with an additional €450 million in potential milestones, as well as double digit royalties on future product sales. The working relationship with Roche is continuing to progress well and recently BioInvent and ThromboGenics announced positive results in a Phase Ib trial of TB-403 in patients with advanced solid tumours. The results showed positive safety and tolerability data. ThromboGenics, who discovered TB-403, receives 60% and BioInvent 40% of all revenue from the deal.
Source: BioInvent Press Release, Nov 19, 2009

Merck Serono to invest more than €150M in R&D Center in China

Merck Serono will strengthen its global research and development capabilities by establishing a global R&D center in Beijing. Merck Serono is planning to invest more than €150 million ($225 million) and create more than 200 new qualified jobs over the next four years to set up the China R&D center and conduct R&D activities in China.
The China R&D organization will become one of the key R&D hubs for the company. The China team will lead drug development for China and other Asian countries, for local clinical trials as well as for the participation in global clinical trials. The team also will ensure the management of collaborations with research institutions in China and continue to look for partnerships with local academic institutions and companies. Research activities conducted in the China R&D center will mainly focus on biomarker research including pharmacogenomics and bioanalytics activities.
Merck Serono already has some research collaborations in China and plans to further develop its collaboration network and build its R&D strategy on more innovation opportunities by tapping into the Chinese scientific expertise.
Source: GEN News Highlights, Nov 23, 2009

Roche establishes new medical research hub in Singapore

Roche announced that it will enter into a strategic alliance with Singapore’s scientific and medical institutions to set up a major new translational research hub in Singapore.
This “Hub for Translational Medicine” aims to enhance the understanding of how scientific advances from preclinical research can be transferred in practice to patients. Bringing together world-class expertise from Singapore’s scientific and medical research institutions with Roche’s significant capability in translational medicine and clinical development, this new centre will focus on expanding knowledge of disease biology to develop new personalised treatment approaches. more
Source: Press Release Roche Group Media Relations, January 28, 2010

Novartis plans full takeover of Alcon for $39.3B

Novartis is proposing a complete purchase of eyecare company Alcon at some $39.3 billion. This excludes the $10.4 billion Novartis spent in April 2008 to purchase a 25% stake in Alcon from Nestlé.
Novartis claims the proposed acquisition of Alcon would strengthen its ophthalmology portfolio and provide greater access to the global eyecare sector. If the takeover is completed, Alcon would be established as a new Novartis eyecare division. 
Alcon made total sales of some $6.3 billion in 2008, of which $2.6 billion was recorded by its ophthalmology pharmaceuticals division. The company’s ophthalmology surgical division made sales of $2.9 billion. It’s consumer division, which encompasses contact lens care products, OTC eye drops, and ocular vitamins, achieved sales of $0.8 million.
Novartis reported $0.5 billion in net sales of selected ophthalmic pharmaceuticals in 2008 and claims it provides a range of complementary medicines used to treat eye diseases that are not addressed by Alcon’s portfolio. The company’s Ciba Vision eyecare and contact lens business generated net sales of $1.7 billion in 2008.
Source: GEN News Highlights, January 4, 2010

Sinovac forms vaccine manufacturing joint venture

Sinovac Biotech has formed a joint venture with Dalian which will give it cost effective manufacturing capacity for 20m does of live attenuated vaccines and 20m doses of vero cell cultured vaccines a year.
Vaccines for rabies, mumps, varicella and rubella are in Sinovac’s pipeline and by striking the deal with Dalian, which will provide land use rights, manufacturing facilities and established operating infrastructure, it believes it can support their production.
The joint venture is called Sinovac (Dalian) Vaccine Technology and will be headquartered in Dalian, Liaoning Province in North East China, occupying a 1m sq ft site and a 200,000 sq ft building. Housed in the headquarters is a 60,000 sq foot manufacturing and R&D facility with two vaccine production lines, one for live attenuated vaccines and another for vero cell cultured vaccines. The site has the capacity to house approximately six different production lines and has quality assurance and control facilities, a research laboratory, office building and warehouse.
The day-to-day operations of the joint venture will be led by Sinovac management, with the positions of chairman, general manager, head of research and development and financial director being held by the biotech’s representatives.
Source: In-pharmatechnologist.com, Nov 26, 2009

Six Pfizer-Wyeth R&D sites to close post merger

Pfizer will close six R&D facilities in the US and the UK, reducing its global capacity by over a third following completion of its acquisition of Wyeth. The US drug giant said that units in Princeton, New Jersey; Rouses Point and Plattsburgh, New York; Sanford and Research Triangle Park, North Carolina, as will as two in Gosport and Slough in the UK, would cease operations.
Pfizer did not specify how many employees the move would affect, However when the multi-billion dollar Wyeth acquisition was completed earlier this month the firm said that as many as 20,000 of the combined 130,000-strong workforce would be cut.
Drug, biologics and vaccine R&D will be focused at five main sites: Cambridge, Massachusetts; Groton, Connecticut; Pearl River, New York; La Jolla, California; and Sandwich in the UK. Other facilities in San Francisco, Cambridge, UK and Shanghai, China will conduct the development of monoclonal antibodies in a move that biopharmaceutical R&D head Mikael Dolston said would transform the firm into the world's leading biopharma manufacturer.
Source: www.In-Pharmatechnologist.com, Nov 10, 2009

(Bio)manufacturing News

New U.K. Center for Regenerative Medicine

A regenerative medicine manufacturing center is to be established at the University of Loughborough with a portion of a £70 million (about $111.49 million) U.K. Government investment. The Engineering and Physical Sciences Research Council (EPSRC) Centre for Innovative Manufacturing in Regenerative Medicine will carry out research and test new ideas in clinical and industrial settings.
The center aims to create next-generation platforms for manufacturing regenerative medicines and inform business models, policy, and public debate. Investment from the EPSRC will total £5.3 million (roughly $8.44 million) over five years, with 28 industrial and government partners contributing an additional £3 million (approximately $4.78 million).
The £70 million government funding also includes the established of a Centre for Innovative Manufacturing in Photonics at the University of Southampton and a Centre for Innovative Manufacturing in Liquid Metal Engineering at Brunel University in London.
Source: GEN News Highlights, January 7, 2010

Merck to buy Avecia’s contract manufacturing biologics business

Merck is to acquire Avecia’s contract manufacturing biologics business, Avecia Biologics. The transaction, which is still subject to regulatory clearance, does not include Avecia’s U.S.-based oligomedicines business. Financial details have not yet been disclosed.
If ratified, the deal will be effected through Merck’s affiliate, Merck Sharp & Dohme. Merck will acquire all of the assets of Avecia Biologics, including its process development and scale-up, manufacturing, quality, and business support operations in the U.K. The company said it plans to honor all Avecia Biologics’ contractual obligations and will talk with its customers to determine ongoing and future biological process development and manufacturing requirements after the deal has been completed.
Source: GENNews Highlights, Dec 17, 2009

Novartis Inaugurates $1B U.S. cell culture flu vaccine facility

Novartis officially opened what it says is the first large-scale cell culture flu vaccine and adjuvant manufacturing facility in the U.S. The $1 billion facility in Holly Springs, NC, results from a partnership between Novartis and the U.S. Department of Health and Human Services. The deal also requires Novartis to provide two commercial-scale annual lots of prepandemic vaccine for a minimum of three years. The HHS retains options to purchase additional flu vaccines over 17 years.
Novartis reports that although the FDA has yet to approve a cell culture-based flu vaccine, part of the HHS contract support for the Holly Springs facility includes funding for development of a cell culture flu vaccine. If such a vaccine is licensed for use in an emergency, the facility will be ready to respond to a pandemic by 2011 and is expected to be up to full commercial-scale production in 2013.
The plant will also start manufacturing Novartis’ MF59® adjuvant by the end of this year. Although not yet licensed in the U.S., the vaccine adjuvant is currently undergoing trials. It is already a component of the EU-sanctioned seasonal flu vaccine Fluad® and the AH1N1 vaccines Focetria®, which received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use in September, and Celtura®, which was licensed in Germany and Switzerland earlier this month.
Novartis’ existing cell culture-based flu vaccine plant in Marburg, Germany, is licensed to produce the seasonal cell culture-based vaccine Optaflu® and also the H1N1 pandemic vaccine Celtura.
Source: GEN News Highlights, Nov 24, 2009

Sartorius launches single-use biomanufacturing range

Sartorius Stedim Biotech has introduced FlexAct BP, the first product in a range which will be an integrated single-use system for entire biomanufacturing steps in upstream and downstream processing.  In July Sartorius entered into an agreement with SAFC to move closer to creating a “totally disposable factory” and the launch of FlexAct is another aspect of its drive towards integrated, single-use biomanufacturing platforms. The platform’s design is focused on integration and standardisation to make it easy for users of stainless steel equipment or individual disposable components to fully convert to a single-use system, according to Sartorius.
The equipment is suitable for production capacity needs from 50 to 1,000 litres and has integrated monitoring and control for pH, temperature, pump speed and fluid level. This removes the need to supervise the system, freeing the user to perform other tasks, and Sartorius believes it represents an advance in implementing process-relevant analytical tools.
In the future users will be able to use the equipment in conjunction with other systems in the FlexAct range which cover media preparation, cell harvesting, ultrafiltration/diafiltration crossflow, virus removal, inactivation and adsorption, polishing, form/fill and form/transfer.
Sartorius has designed the whole system to ensure full scalability of all components, such as bags, filter capsules or tubing, to simplify the installation process. The components are installed on the central operating module and require minimal set-up.
Source: In-pharmatechnologist.com, Dec 2, 2009

Research News

Method found to suppress breast cancer stem cells

A team of researchers from the University of Michigan Comprehensive Cancer Center, Inserm, and Institut Paoli-Calmettes has identified a strategy to target human breast cancer stem cells. They found that inhibiting the cell-surface protein CXCR1 decreased tumor growth and metastasis in mice xenotransplanted with human breast cancer cells. The study is published in The Journal of Clinical Investigation.
The scientists used either an antibody or a small molecule known as repertaxin to suppress CXCR1. This selectively depleted the cancer stem cell population in two human breast cancer cell lines in vitro. Loss of the cancer stem cells was followed by extensive death of many of the remaining tumor cells. Importantly, treatment with repertaxin had similar effects in mice xenotransplanted with human breast cancer cells.
The authors therefore suggest that strategies that target CXCR1, the soluble protein that binds to it, and the signaling pathways downstream of it might provide a good approach to deplete breast cancer stem cells.
Source: GEN News Highlights, January 14, 2010

Cancer maps for skin and lung cancer and others in the making

Scientists have unlocked the entire genetic code of two of the most common cancers - skin and lung. Not only will the cancer maps pave the way for blood tests to spot tumours far earlier, they will also yield new drug targets, says the Wellcome Trust team. The scientists found the DNA code for a skin cancer called melanoma contained more than 30,000 errors almost entirely caused by too much sun exposure.The lung cancer DNA code had more than 23,000 errors largely triggered by cigarette smoke exposure. From this, the experts estimate a typical smoker acquires one new mutation for every 15 cigarettes they smoke. Although many of these mutations will be harmless, some will trigger cancer.
Scientists around the globe are now working to catalogue all the genes that go wrong in many types of human cancer. The UK is looking at breast cancer, Japan at liver and India at mouth. China is studying stomach cancer, and the US is looking at cancers of the brain, ovary and pancreas. The International Cancer Genome Consortium scientists from the 10 countries involved say it will take them at least five years and many hundreds of thousands of dollars to complete this mammoth task.
By identifying all the cancer genes we will be able to develop new drugs that target the specific mutated genes and work out which patients will benefit from these novel treatments. It could even be possible to develop MoT-style blood tests for healthy adults that can check for tell-tale DNA patterns suggestive of cancer. By studying the cancer catalogues in detail, the scientists say it should be possible to find exactly which lifestyle and environmental factors trigger different tumours.
Source: BBC News, December 16, 2009

Synthetics stop bleeding

Nanoparticles designed to mimic the clotting capability of blood platelets have been shown to quickly reduce bleeding in rodents with severed arteries. The synthetic particles, which stick to the body's own platelets, stanch bleeding more effectively than a clotting drug currently used to stem uncontrolled blood loss. "We're helping to form the clot," says Erin Lavik, a bioengineer at Case Western University in Cleveland, who led the research.
If successful in further tests, researchers hope the nanoparticles could one day be injected soon after a traumatic injury by paramedics, or in the battlefield. Early safety tests are promising, but developing safe blood-clotting treatments has been a challenge. "There's a balance between the two edges of the sword--bleeding too much and clotting too much," says Mortimer Poncz, a physician at the University of Pennsylvania Medical School, in Philadelphia, who was not involved in the research. "You don't want to stop bleeding in the leg but die of a heart attack or have stroke."
Lavik and collaborator James Bertram, a graduate student at Yale, have now developed a nanoparticle small enough to flow through capillaries unfettered. It also has a platelet's specific stickiness. The particle is about a third of the size of a normal platelet. Each particle has a polymer core that's coated with polyethylene glycol (PEG)--a water-soluble molecule that keeps them from sticking to each other or to the blood vessels. The PEG molecules are also topped with a peptide sequence that binds to activated platelets. "People had previously shown that activated platelets bind to this sequence, so we optimized the chemistry to expose the molecule, presenting them to activated platelets," says Lavik, who was recognized by Technology Review as a TR35 Young Innovator in 2003.
Source: MIT Technology Review, December 18, 2009

Lentiviral vector gene therapy slows brain disease

The brain disease X-linked adrenoleukodystrophy (ALD) can be treated with a combination of gene therapy and blood stem cell therapy, new EU-funded research shows. The research is part of the X-ALD ('X-linked adrenoleukodystrophy: pathogenesis, animal models and therapy') project, which received EUR 1.8 million under the 'Life sciences, genomics and biotechnology for health' Thematic area of the EU's Sixth Framework Programme (FP6). The study's findings were published in the journal Science.

The international team of researchers from France, Germany, the Netherlands and the US evaluated two patients in a two-year pilot study. They successfully slowed the progression of ALD by using a lentiviral vector to introduce a therapeutic gene into the blood cells of the patients. While the researchers recognise the need for studies with larger groups of patients to be conducted, these results indicate that gene therapy with lentiviral vectors may play a central role in the treatment of human disorders.
 
By using a lentiviral vector in this study, the researchers successfully removed the patients' blood stem cells and genetically corrected them in the lab. They introduced a working copy of the ALD gene into the cells. The researchers then infused the altered cells back into the patients following treatment that damaged their bone marrow. After two years, the researchers detected ALD proteins in the patients' blood cells. Neurological improvement was noted, and the progression of the disease was similar to that found with bone marrow transplants. According to the team, the healthy ALD protein was expressed in about 15% of blood cells.

See also: http://www.sciencemag.org/
Source: CORDIS Europa, November 21, 2009

Stem Cell News

Roche and allies to develop stem cell-derived cell lines for drug discovery
Roche is teaming up with the Massachusetts General Hospital and Harvard University to develop new stem cell-based cell lines as disease models for early drug candidate testing. The 3–5 year partnership will initially focus on metabolic disorders and cardiovascular disease and will expand to cover a range of other diseases.
The collaboration aims to develop cell lines that can be used to evaluate the potential efficacy, safety, and toxicology profiles of new drugs pulled from Roche’s compound library. The company says that the cell lines will be derived from the tissues of both healthy volunteers and patients with a range of diseases. The ultimate goal is to use stem cells for discovering new treatment approaches and bridging the gap between the laboratory and the clinic.
Roche has forged a number of collaborations focused on evaluating stem cell-based approaches for drug discovery. In June 2009, the firm signed a €7.5 million (about $10.36 million), two-year collaboration with I-STEM (Institute for Stem Cell Therapy and Exploration of Monogenic Diseases) focused on the use of I-STEM’s neuronal stem cell proliferation technologies in the screening of Roche’s compounds for potential new candidates against neurodegenerative diseases.
In 2008, Roche partnered with U.K.-based stem cell consortium SC4SM (Stem Cells 4 Safer Medicines) to generate a repository of stem cells suitable for toxicology testing in high-throughput platforms. The initiative is being funded primarily by the U.K. Government, with Roche and two other pharmaceutical companies also contributing. During the same year the firm signed an agreement with Cellular Dynamics to test a number of its drug compounds for cardiotoxicity.
Source: GEN News Highlights, Feb 5, 2010

Clinical trials news

Positive results Phase I trial of TB-403 in patients with advanced solid tumours

BioInvent International AB and ThromboGenics NV announced positive results from a Phase I trial of their novel anti-cancer monoclonal antibody TB-403 in patients with advanced solid tumours. The results were presented at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston, U.S. TB-403 was well tolerated with no reported dose limiting toxicity. These positive data support progression of TB-403 and further development. Detailed results of an earlier Phase I trial in healthy volunteers, also presented at the meeting, showed that TB-403 demonstrated a good safety profile with no serious adverse effects reported.

The novel mechanism of action of TB-403 represents a potentially promising cancer therapy. It is a humanized monoclonal antibody directed towards placental growth factor (PlGF), expected to act by blocking the formation of the new blood vessels that are required for the tumour growth. Preclinical exploration of PlGF biology suggests a role in tumour angiogenesis and metastasis and a limited role in the maintenance of normal vasculature. This mode of action could result in therapeutic benefit with an acceptable side effect profile.
Source: BioInvent Press Release, Nov 16, 2009

Regulatory News

Herceptin approved in EU for patients with HER2-positive advanced stomach cancer

Roche announced tthat the European Commission has approved Herceptin (trastuzumab) in combination with chemotherapy for use in patients with HER2-positive metastatic stomach (gastric) cancer. The approval is based on the impressive results from the international ToGA trial, which showed that treatment with Herceptin significantly prolongs the lives of patients with this aggressive cancer. Overall survival for patients with high levels of HER2 in the ToGA study was 16 months versus 11.8 months (on average) for patients receiving chemotherapy alone.
Source: Roche Group Media Relations, January 28, 2010

FDA approves ACTEMRA for the treatment of moderately to severely active rheumatoid arthritis

Roche announced that the United States (US) Food and Drug Administration (FDA) approved ACTEMRA (tocilizumab, RoACTEMRA in the European Union) for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response to one or more tumor necrosis factor (TNF) antagonist therapies. ACTEMRA, the result of a research & development collaboration with Chugai, is the first interleukin-6 (IL-6) receptor-inhibiting monoclonal antibody approved to treat RA, may be used alone or in combination with methotrexate or other disease modifying anti-rheumatic drugs (DMARDs).  more
Source: Roche Press Release, January 11, 2010

EDQM and EMEA News

EDQM: Training session on IVMP products

Training Session on the ‘Guide for the Elaboration and Use of monographs on
Immunological Veterinary Medicinal Products (IVMPs)’
18-19 March 2010, Strasbourg, France

The 'Guide for the Elaboration and Use of Monographs on Immunological
Veterinary Medicinal Products' was drafted in order to facilitate the
interpretation and use of European Pharmacopoeia monographs and general
chapters in the field of veterinary vaccines. The training session aims to
give participants the knowledge they need to use the Guide and provide
extensive clarification about the inter-relationship between the different
parts of the European Pharmacopoeia.

Source: Visit http://www.edqm.eu/en/Events-253.html for more information
EDQM NEWS: 13 new monographs and chapter on glycan mapping adopted

In its 135th session, the European Pharmacopoeia Commission adopted a new general chapter on glycan mapping (2.2.59) and 13 new monographs.
For more information chick here:
http://www.edqm.eu/en/News-General-Information-43.html

EMEA: Concept paper gene transfer medicinal products

The European Medicines Agency has published a Concept paper on the revision of the note for guidance on the quality, pre-clinical and clinical aspects of gene transfer medicinal products (EMA/CHMP/GTWP/BWP/234523/2009).

The document is available on the following webpage:
http://www.ema.europa.eu/pdfs/human/genetherapy/23452309en.pdf

This Concept Paper proposes a revision of the Note for Guidance on the Quality, Preclinical and Clinical Aspects of Gene Transfer Medicinal Products (CPMP/BWP/3088/99) that came into effect in 2001. This revision will address the issues identified from clinical experience and provision of Scientific Advice on gene therapy medicinal products and will lay down detailed and updated requirements for the quality, non-clinical and clinical aspects of gene therapy medicinal products.

Please provide any comments you may have on this document to GTWPsecretariat@ema.europa.eu , by using this template:
http://www.ema.europa.eu/pdfs/human/regaffair/submitcomment.doc
The deadline for submitting comments is 31st of March 2010.

EMEA: Concept paper advanced therapy medicinal products

The European Medicines Agency has published a draft Concept paper on the development of a guideline on the risk-based approach according to Annex I, Part IV of Dir. 2001/83/EC applied to advanced therapy medicinal products (EMA/CHMP/CPWP/708420/2009).

The document is available on the following webpage:
http://www.ema.europa.eu/pdfs/human/cpwp/70842009en.pdf

This concept paper is intended to provide the background and rationale of the guideline on the risk based approach and shall describe the approach and content of the future guideline.
Please provide any comments you may have on this document to veronika.jekerle@ema.europa.eu<mailto:veronika.jekerle@ema.europa.eu, by using this template:
http://www.ema.europa.eu/pdfs/human/regaffair/submitcomment.doc
Please note that the deadline for submitting comments is 31st of March 2010.

AGENDA

Open Forums on Regulatory Aspects during Biopharmaceutical Development
February 23, 2010, Vienna, Austria 

Posttranslational Modifications of Proteins - Consequences and Therapeutic Use
February 24-25, 2010, Vienna, Austria 

Cell Line Development and Engineering
1–5 March 2010, Prague, Czech Republic
www.informa-ls.com/celldev

EMBL Workshop on Visualizing Biological Data (VizBi)
March 3-5, 2010, Heidelberg, Germany

Bio-Europe Spring
March 8-10, 2010, Barcelona, Spain

5th Annual Pharmacovigilance Conference
Implementing best practices in drug safety and surveillance
17th-19th March 2010, BSG House, London, UK
Information: jana.jankova@vgpharma.com

Training Session on the
‘Guide for the Elaboration and Use of monographs on Immunological Veterinary Medicinal Products (IVMPs)’
18-19 March 2010, Strasbourg, France
Information: http://www.edqm.eu/en/Events-253.html

Biopharmaceutical change control
April 13-15, 2010, San Diego, CA, USA
Information: www.healthtech.com/Conferences_Overview

Cell Culture Engineering XII
April 25-30, 2010, Banff, Alberta, Canada
Information: info@engconfintl.org 

ERBI's 12th Annual BioPartnering Exchange
May 2-4, 2010, Cambridge, UK

20th Anniversary World Congress on Biosensors
May 26-28, 2010, Glasgow, UK

ACTIP 20th anniversary meeting
May 26-27-28, 2010, Pueblo Acantilado, El Campello (Alicante, Spain)
Only for ACTIP members and invited guests
Info: actip-secretariat@telefonica.net

BIOSPAIN
Sept 30-Oct 2, 2010, Pamplona, Spain
http://www.clinam.org/conferencehttp://www.georgiabiosciences.com/BioConvention.aspxhttp://www.erbiconference.co.uk/http://www.eurobiotechforum.com/http://www.eurasante.com/en/great-projects/eurobio-2009.htmlhttp://www.eurasante.com/en/great-projects/eurobio-2009.html

Quality of medicines in a globalised world: dreams and reality
October 14-15, 2010, Prague, Czech Republic
Information: European Directorate for the Quality of Medicines & HealthCare (EDQM), Council of Europe
francine.baumgarthen@edqm.eu