ACTIP bulletin no. 64 January 2012
The next ACTIP meeting will take place
and will be hosted by the ACTIP Secretariat. The meeting will focus on "The Cell Environment" and on "Challenges in upstream and downstream processing". Only for ACTIP members and individually invited guests.
In this issue:
News from the Commission
Stem Cell News
Clinical trials News
Top 10 women in biotech
News from the Commission
On November 30, 2011, the European Commission presented its €80-billion research funding programme for the decade - Horizon 2020 - with the aim of boosting research, stimulating innovation and simplifying the way scientists and smaller businesses can get funding for EU-backed projects.
The Horizon 2020 programme brings together all EU research and innovation funding under a single scheme running from 2014 to 2020. It replaces the Seventh Framework Programme for research (FP7), which expires in 2013. The programme is exopected to be a driver for Europe's economic growth.
Horizon 2020 is divided into three tranches designed to boost specific causes.
I. The first aims to establish the EU as a leader of cutting-edge projects using a €24.5-billion budget, including funding for the European Research Council (ERC). The body, whose support for individual academic studies has garnered praise, sees its budget increase by 77%.
II. The second allocation is targeted at industrial innovation with a budget of €17.9 billion, including €13.7 billion for key technologies.
III. The third and biggest tranche, worth €31.7 billion, goes to so-called "societal challenges" divided into six themes:
Health, demographic change and well-being;
Food security, sustainable agriculture, marine and maritime research and the bio-economy;
Secure, clean and efficient energy;
Smart, green and integrated transport;
Climate action, resource efficiency and raw materials; and
Inclusive, innovative and secure societies.
The remaining €5.9 billion is set aside for the Joint Research Centre – the Commission’s in-house research institution – which works on issues that include the environment, nanotechnology and nuclear safety.
Friendly to small businesses .The Commission has sought to considerably simplify funding applications under the new research programme, replacing several existing funding schemes with two. Four existing methods for calculating costs under the various schemes will be replaced with one calculator.
The programme represents a breakthrough for small and medium enterprises under EU projects. A clause within Horizon stipulates that 15% of the second and third tranches described above (worth €8.6 billion) must be allotted to smaller companies. Under the new programme, smaller firms will be able to apply for funding for new proposals, fund the research and technological development of such ideas, and receive help bringing them to market.
Controversial: The new programme will operate in tandem with the EU regional - or 'structural' - funds. Under the proposals, Horizon 2020 will award seals of excellence to universities and research centres once they have demonstrated a level of proficiency up to the highest EU standards. Such regions, usually found in the newer member states of eastern Europe, will be encouraged to use structural funds to bring their research infrastructure up to scratch to win such seals, which will enable them to attract more funds from Horizon and private investors. Some eastern member states resist the move, since they believe it could reduce their ability to use the structural funds for infrastructure projects, and also limit their access to Horizon 2020.
Source: www.Euractiv.com, December 1, 2011
Under the European Commission’s new proposal for funding the eighth framework programme (FP8) – Horizon 2020 – the European Research Council (ERC) is set to see a 77% boost in funds to €13.2 billion. ERC President Helga Nowotny (a leading Austrian social scientist) told EurActiv the following in a recent interview.
Over the last five years, the ERC has proven to make a real difference. Its impact on the integration of the European Research Area is beyond any doubt and beyond what we had expected. The ERC is a game-changer and its success has been underlined by the scientific community, member states and other stakeholders.
The budget rise would allow more top researchers, of which [there are] many younger ones, to pursue their pioneering research in Europe. Above all, it's in these difficult times that funding science and innovation in general, and excellence and frontier research in particular, is crucial for economic recovery. Therefore it will be likely that the ERC will receive a substantial increase in its budget under the Horizon 2020 framework.
However, the increased budget or the Horizon 2020 themes selected will not change the ERC's emphasis: the ERC is committed entirely to a bottom-up approach of frontier research in all fields of science and scholarship, the evaluation of which is based on the sole criterion of excellence. We have a very well defined, widely acknowledged evaluation procedure. Except for simplification, the worst thing to do would be to alter this process. Everyone in the European Commission understands that.
Source: www.EuractivNews.com, January 2, 2011
The European Research Council (ERC) was established to fund more fundamental and frontier research within the framework of FP7. As it states on its website, it funds research of the very highest quality at the frontiers of knowledge, thus feeding into the innovation chain and supporting the EU's Europe 2020 strategy for smart, sustainable and inclusive growth and the EU's flagship Innovation Union initiative.
The ERC complements existing funding schemes at the national and European levels. The ERC grants are open to researchers from any country in the world. However, the ERC-funded research should be carried out in one of the 27 EU Member States or in one of the associated countries. The ERC grants are open to researchers from both public and
private institutions and of any age but normally between two and twelve years after an PhD award. Scientific excellence is the sole criterion on the basis of which ERC grants are awarded
Applications can be made in any field of research with particular emphasis on the frontiers of science, scholarship and engineering. In 2012, grants will be available for Starting Independent Researchers, Advanced Investigator Grants and Synergy Grants (for small groups).
In the Life Sciences, applications can still be submitted for Advanced Grants (deadline March 14, 2012) in the following fields:
* LS1 Molecular and Structural Biology and Biochemistry
* LS2 Genetics, Genomics, Bioinformatics and Systems Biology
* LS3 Cellular and Developmental Biology:
* LS4 Physiology, Pathophysiology and Endocrinology
* LS5 Neurosciences and neural disorders
* LS6 Immunity and infection
* LS7 Diagnostic tools, therapies and public health
* LS8 Evolutionary, population and environmental biology
* LS9 Applied life sciences and biotechnology
Please consult the ERC website for full details: http://erc.europa.eu/funding-and-grants
In December, the European Commission organized its first ever Innovation Convention launched by President Barroso and convened by Commissioner Geoghegan-Quinn with the aim of “bridging the gap between good ideas and bringing products to market”.
One of the highlights of the Convention was the EU Women Innovators Prize, which saw first and second place awarded to founders of biotechnology companies. Dr Fabienne Hermitte, co-founder of IPSOGEN received second prize for her work as a pioneer in personalised healthcare diagnostics, which allow more individualises treatments for cancer patients. Dr Gitte Neubauer, co-founder of CELLZOME was awarded the first prize for her ground-breaking research into chemo-proteomics and targeted medicine against inflammatory diseases and cancer.
During the event, the former Chief Scientific Advisor to the Scottish Government, Professor Anne Glover, was named as the first ever European Chief Scientific Advisor. She will provide independent scientific advice to President Barroso throughout all stages of policy development in Europe. This important appointment is additional proof of the Commission's commitment to ensure that responsible science and innovation dominate Europe's quest to kick start growth and respond to our most pressing societal challenges. Life science and biotechnology will continue to play a central role in this.
Moreover, the opening of the Convention was marked by the significant agreement between the European Commission and the European Investment Bank (EIB) to launch a new guarantee facility for innovative SMEs, building on the success of the Risk-Sharing Finance Facility (RSFF) launched in 2007. In signing the agreement, Commissioner Geoghegan-Quinn stated: “Investing in research and innovation carried out by SMEs means that we will have more growth, sustainability and competitiveness in Europe”.
Source: EuropaBioNews, December 7, 2011
On December 5, 2011, EU ministers reached agreement on outstanding issues surrounding a unified EU patent which would pave the way for the Polish presidency to claim success in the venture at a signing ceremony in Warsaw on 20 December (the Warsaw Accord) amid mounting optimism that the new regimes in Spain and Italy could drop their opposition to the idea.
In May, Italy and Spain complained to the European Court of Justice against the use of the so-called 'enhanced cooperation' procedure for the patent, which allows a group of countries to go ahead without the approval of all 27 EU member states. They claimed the move went against the spirit of the EU single market. The proposals recognise English, French and German as the patent's official filing languages but Rome and Madrid feared this would give an unfair advantage to companies from the 'big three' jurisdictions.
Issues still at stake are:
* Where to locate the central patent court, with London, Munich and Paris the candidate cities;
* Where to locate an appellate body, for which Luxembourg is highly tipped;
*The location of an arbitration centre, for which Ireland, Slovenia and Portugal are vying;
* The relative powers and financing of all these bodies and the system itself; and
* Transition procedures to govern the introduction of the new system.
Source: www.EuractivNews.com, December 5, 2011
It is essential that cell therapies can be made efficiently and effectively on a large scale for them to reach the market. Yet it can be a challenge to achieve GMP standards during large scale production. ATMI offers a potential solution with its new Integrity Xpansion bioreactors, which feature a 2-D multiplate design that offers the same cell environment as standard multitray set-ups but has been developed to support large-scale production.
The new reactors can house 180 plates with a surface area of 11 sqm spaced 1.6 mm apart in a unit 60 cm high and 35 cm in diameter which represents a large reduction in the factory floor space required compared with other bioreactor systems with the same capacity. Because critical cell culture parameters do not change using Xpansion systems the risk of failure during process scale up is mitigated.
The company claims that the combined increase in scale up speed and reduction in upstream and downstream processing steps cuts the number of operations required during manufacture and hence the cost of goods can successfully be reduced by up to 40 percent.
Source: www.In-pharmatechnologist.com, Dec 1, 2011
DSM Biologiocs has signed an MoU with the Australian Institute for BioEngineering and Nanotechnology (AIBN) on the commercialization of development-stage biologics projects.The MoU follows on an agreement published in May 2011 between DSM and the Australian government to build and operate a biopharmaceutical manufacturing facility in Brisbane. The 50 mEuro facility, already under construction in Brisbane, will be owned by Biopharmaceuticals Australia, a venture set up by the State Government, but day to day operations will be led by DSM. There are up till now no biomanufacturing facilities in Australia. The deal will give DSM access to a number of research projects that are ready to move to commercialization stage.
Source: www.In-pharmatechnologist.com, December 20, 2011
US company Biologics Modular is leasing its mobile, modular production facilities and clean rooms to help clients make FDA regulated drugs without fixed plants. The company thinks the model will appeal to research park innovator biopharm that need access to flexible and affordable GMP production capacity. The company offers the model as an alternative to outsourcing to CMOs. The facilities are manufactured by Biologics Modular at its site in Indiana before being qualified and validated. Clients also receive support with process validation, GMP quality control and regulatory strategies. By taking this modular, pre-constructed, pre-tested approach to facility design the company claims it can deliver a plant within 20 weeks. The facilities are housed in steel shipping containers. The modules inside the container are like giant Lego pieces. When the plant is no longer needed it can be decommissioned and in most cases the company will also handle the removal.
Source: www.In-pharmatechnologist.com, Dec 1, 2011
VIIV, a joint venture of GSK and Pfizer addressing products to treat HIV-positive patients, has partnered with Russia's JSC Binnopharm to boost manufacturing capacity in Russia. The latter will act as a local secondary producer and packager of drugs for ViiV.
The deal builds on an earlier agreement between GSK and Binnopharm whereby the Russian firm agreed to produce GSK's major range of cervical cancer, pneumococcal and rotavirus vaccines for the local market. Under that agreement, GSK supplied Binnopharm with bulk supplies of the vaccines and transferred some production technology and expertise to the firm's plant. Binnopharm was responsible to gain regulatory approvals.
Pfizer, too, has expanded its production capacity in Russia. In March it formed a partnership with Petrovax Pharma to produce Pfizer's pneumococcal vaccine at Petrovax' facility in Moscow.
Together, all these deals mean that 70% of Russia's HIV-positive patients could ultimately be treated with locally manufactured products.
Source: www.In-pharmatechnology.com, November 3, 2011
To contribute to an informed debate, EuropaBio is launching a new brochure acknowledging the complexity of biotechnology-derived medicines (which are also referred to as biological medicines) and the emergence of biosimilars.
Recently adopted EU legislation on pharmacovigilance, which is currently being implemented in Member States, should play a vital role in maintaining safety standards for all biotech medicines, including biosimilars. This can be achieved by ensuring a correct and precise identification of the product that is prescribed and dispensed as well as ensuring the assessment of the immunogenicity as a key component in the Risk Management Plan of any biological medicine. With biotechnology derived medicines, it is important that in case of adverse events, products can be identified by the product batch. EuropaBio advocates the use of the products’ brand name when prescribing medication. The implementation of the new pharmacovigilance legislation offers a timely opportunity to put these best practices in place across the European Union.
As the discussions continue in Europe to further develop the regulatory pathway for biosimilars, new initiatives to foster multi-stakeholder dialogue, such as the Process on Corporate Responsibility in the Field of Pharmaceuticals initiated by Vice President Tajani, provide the opportunity to extend the debate to other relevant aspects of biosimilars including market access. This brochure is EuropaBio’s contribution to these forums.
Source: EuropaBio News, October 26, 2011
US company Amgen has partnered with non-branded drugmaker Watson to develop biosimilar cancer drugs. Amgen will be responsible for developing, manufacturing and commercializing biosimilar versions of antibody-based cancer treatments, while Watson will contribute $400m in development costs and will take charge of marketing and life cycle management. The collaboration will not pursue biosimilars of Amgen's proprietary products. In 2010, Watson already signaled its interest in biosimilars by acquiring UK-based CMO Eden Biodesign.
Source: www.In-pharmatechnologist.com, December 22, 2011
US based company iBio is expanding use of its non-transgenic green plant technology to biosimilar monoclonal antibodies after producing rituximab using the system. Previously, green plants were shown to be able to produce therapeutic proteins and viral antigens for vaccines.
iBio developed a vector system for multi-chain proteins, such as antibodies, to be expressed correctly. The plants produced a balance of heavy and light chains which self-assembled into the mab-structure. They then purified the mab from the plant tissue. Plant-produced rituximab underwent clinical tests to evaluate efficacy and showed similar cytotoxicity and antigen recognition to rituximab produced in mammalian cells.
Source: In-pharmatechnologist.com, July 10, 2011
GE Healthcare is planning to invest 1 bn $ of its R&D budget on the development of new bioreactors, purification platforms and characterization systems as well as on the further expansion of its diagnostics, molecular pathology, biomarker and molecular imaging businesses.
Life sciences and new technologies for the manufacture of biopharmaceuticals such as the new cancer therapeutics is a key focus for GE Healthcare. Further witness of this strategy is the acquisition of cell culture/media manufacturer PAA laboratories. In addition, GE Healthcare recently signed a deal with Sartorius Stedium Biotechnology for the exchange of licenses for various patents key to their respective biomanufacturing technology offerings.
Source: www.In-pharmatechnologist.com, September 19, 2011
Contract manufacturer CMC Biologics has ramped up bioreactor capacity to meet demand for Phase III clinical trials and beyond. The company added a 2,000 L Thermo Scientific Hyclone single use bioreactor (SUB) at its manufacturing facility in Copenhagen, which it aims to have up and running by December 2011. Already CMC expanded it manufacturing capacity in Seattle, Washington last year. It also follows soon after its deal with Oxford Biotherapeutics, which saw CMC to grant the UK developer access to its CHEF1 cell line based expression system for drug research and development applications.
Source: www.In-pharmatechnologist.com, September 13 2001
BioInvent International AB and Les Laboratoires Servier have entered into an antibody collaboration on an oncology target involved in tumour cell metabolism provided by Servier. BioInvent will receive a licensing fee, research support and potential milestone payments of more than EUR 11m, as well as royalty on future sales of the product.
Under the terms of the agreement Servier will engage BioInvent to screen its proprietary n-CoDeR® library for antibodies specific to the undisclosed target. Servier will also have access to BioInvent's in-house pre-clinical capacities in selecting an antibody candidate for development.
The Bioinvent n-CoDeR® library will be used in combination with a functional assay set up at Servier to select antibody candidates. This is expected to enable Servier to develop one of the first monoclonal antibodies targeting tumour cell metabolism, an approach anticipated to have major therapeutic potential.
Source: BioInvent Press Release, January 4, 2012
Roche announced that it has been awarded an exclusive contract by Karolinska University Hospital in Stockholm, Sweden, to provide a diagnostic tool for primary screening of Human Papillomavirus (HPV) in Swedish women. This is the first major public tender in Europe to screen women using Roche’s HPV test for high risk virus types which potentially can cause cervical cancer.
The screening program is expected to serve as a pilot for implementation of HPV primary screening nationwide in Sweden and will start in November 2011.
In 2008, European Union guidelines for cervical screening introduced the alternative possibility to use HPV testing as the primary diagnostic test, provided that implementation was piloted in an organized fashion with stringent evaluation. The Swedish program is one of the first to follow these guidelines.
Source: Roche Group Media Relations, September 30, 2011
Thanks to advances in chemistry and software, researchers can soon sequence a human genome for $1,000 in a day.
Back in July, Jonathan Rothberg, CEO of the, predicted that by 2013 his company would develop a chip that could sequence an entire human genome.
Connecticut-based biotech company Ion Torrent has developed a chip that can sequence an entire human genome and packed it into a new tabletop sequencer called the Ion Proton. The company recently introduced the device at the Consumer Electronics Show in Las Vegas, although the sequencer is only available to researchers at this point.
At $149,000, the new machine is about three times the price of the Personal Genome Machine, the sequencer that the company debuted about a year ago. But the DNA-reading chip inside it is 1,000 times more powerful, allowing the device to sequence an entire human genome in a day for $1,000—a price the biotech industry has been working toward for years because it would bring the cost down to the level of a medical test. Currently, other sequencing machines, which are optics based, sell for approximately $500,000.
Source: MIT Technology Review, January 12, 2012
The world’s biggest drug makers are racing to market the first medicine to tap into a gene mutation that drops heart-attack risk by as much as 88 percent. Normally, the PCSK9 gene creates a protein that disrupts the ability of liver cells to remove bad cholesterol from blood, enabling it to accumulate. A mutated form of the DNA found in 3 percent of people lowers levels of the protein, allowing more of the artery-clogging hormones to be swept away.
Just five years after the effects were discovered, more than a half-dozen companies led by Amgen Inc., Sanofi and Pfizer Inc. are developing a new family of treatments based on the science. Amgen and Sanofi partner Regeneron Pharmaceuticals Inc. will present data from early human trials at the American Heart Association meeting mid November. At stake is entry into a market that generated $36.4 billion in worldwide sales last year, according to IMS Health.
It is the biggest opportunity for lowering cardiovascular risk for the entire pharmaceutical industry, with millions of people who could potentially benefit.”
Current treatments are led by Lipitor, a statin known to block an enzyme the body needs to produce bad cholesterol, or LDL, in the liver. The new family of treatments are mostly based on antibodies that need to be injected once or twice a month.
Amgen, the world’s largest biotechnology company, is testing its drug, AMG 145, in several trials in high-cholesterol patients, including those who can’t tolerate existing treatments such as Lipitor. A single dose of AMG 145 lowered bad cholesterol as much as 70 percent in volunteers, Amgen told analysts in April. Amgen’s data from a clinical trial of 54 people will be reported Nov. 14 at the heart meeting in Orlando, Florida.
Regeneron, jointly developing its treatment with Paris- based Sanofi, also on Nov. 14 will present early results from a 62-patient study. Second-stage trials showed that adding their PCSK9 drug to high doses of Lipitor lowered cholesterol by 65 percent versus a 17 percent reduction for high-dose Lipitor alone, Sanofi and Tarrytown, New York-based Regeneron said in a statement. Sanofi will start final-stage trials of the drug next year, the company has said. Three phases of clinical trials generally are required for U.S. regulatory approval.
Other companies pursuing research on this front are Merck & Co., Isis Pharmaceuticals Inc. with partner Bristol-Myers Squibb Co., and Alnylam Pharmaceuticals Inc. In July, Pfizer, the world’s biggest drugmaker, began a second-stage testing of its anti-PCSK9 antibody, while Merck is working in the lab on an injected antibody and a pill that could block PCSK9’s effects.
Source: Bloomberg Business Week, November 11, 2011
An early clinical trial of a hepatitis C vaccine has shown "promising" results in a trial with 41 patients, according to researchers at Oxford University. Designing a vaccine has been difficult as the virus changes its appearance, making it hard to find something to target. In the UK, up to 500,000 people may be infected with the virus. The World Health Organization believes the global figure could be as high as 170 million people. It spreads through blood-to-blood contact such as sharing needles. While infection can be controlled with antiviral drugs, the Oxford University researchers say a vaccine "would be a major step forward".
Cold viruses were modified with genetic material from the hepatitis C virus in order to prime the immune system to attack the hepatitis C virus. The aim of the Phase I trial was to determine whether the treatment was safe and to help plan future trials.Forty-one healthy patients were given the vaccine. Scientists said it produced a "very strong" immune response which lasted for at least a year and had no major side-effects. The next step will be to give the vaccine to people at-risk of hepatitis C infection to see whether it protects against the virus.
Source: www.BBC.com/news, January 4, 2012
A malaria vaccine has shown promising results in a clinical trial in Africa. Infants given the prototype vaccine had about half the risk of getting malaria compared with those who did not receive the jab, say researchers.
The vaccine, known as RTS,S, is one of two experimental malaria vaccines being tested around the world. More than 15,000 children aged under 18 months took part in the year-long study, published in The New England Journal of Medicine . The trial was conducted in seven African countries on two groups of children - newborns aged six-12 weeks - and babies aged five-17 months.
One year on, there were about half the number of cases of malaria in the older group of children given the vaccine, compared with those in a control group who received vaccines against other illnesses.
A preliminary trial of another potential vaccine recently revealed promising results.
The trial was designed to test safety, but researchers found that 45 children given the MSP3 vaccine had high levels of protection.The results of the Burkina Faso trial were also published in The New England Journal of Medicine.
Source: BBC News Health, October 18, 2011
Stem cell news
A team from King's College London have submitted two clinical-grade stem cell lines to the UK Stem Cell Bank (UKSBC), which will test and validate them before offering them to researchers. The cells are the first to be grown completely free from animal-derived products, known as "xeno-free," and developed specifically to be of clinical grade and for public use.
The hope is that the cells will be grown and processed by the bank to feed cell stocks for human trials and, beyond that, patient treatments. The cells have the potential to become the "gold standard" lines for developing new stem cell based therapies for use in regenerative medicine trials in patients.
The UK Stem Cell bank already has more than 90 research grade stem cell lines for use in laboratory studies, but as yet has no clinical grade xeno-free lines for use in human trials. A few companies, such as Pfizer and Advanced Cell Technology, are already conducting or are about to start human trials using hES cells -- which are harvested from embryos -- to test their potential for repairing spinal cord injuries and eye disorders like macular degeneration. But the hES cell lines for these early trials were reclassified from "research grade" to "clinical grade" for specific short-term clinical studies in selected disease areas.
The now deposited cells were grown from frozen embryos donated by patients who had had in-vitro fertilization (IVF) treatment and no longer wanted to use their remaining stored embryos. The embryos would otherwise have been discarded.
Source: Reuters, December 6, 2011
Professor Frank Barry, one of Ireland’s leading scientists, is to receive a major award for his work dedicated to stem cell therapy On February 6, 2012. This is the first time that the 2012 Marshall R. Urist Award for Excellence in Tissue Regeneration Research, has been awarded to a scientist working outside of the US.
Professor Barry, Director of the National Centre for Biomedical Engineering Science (NCBES) at the National University of Ireland, Galway, is to be presented with the award by the Orthopaedic Research Society. Created in 1996, the award is sponsored by John Wiley & Sons, Inc and honours an investigator who has established him/herself as a cutting-edge researcher in tissue regeneration research.
Source: InsideIreland.ie, January 6, 2011
On October 18, 2011, the Court of Justice of the European Union (CJEU) published its decision in Brüstle v. Greenpeace. The Court ruled that processes that involve the derivation of stem cells from a human embryo at the blastocyst stage, entailing the destruction of that embryo, could not be patented.
The Court states that the exclusion from patentability concerning the use of human embryos for industrial or commercial purposes set out in Article 6(2)(c) of the Biotechnology Directive also covered the use of human embryos for purposes of scientific research. Also, the Court found that where an invention does not itself “use” human embryos but relates to a product whose production necessitates the prior destruction of a human embryo or a process that requires a base material obtained from such destruction, that invention would not be patentable because it would constitute use within the meaning of Article 6(2)(c) of the Directive.
USA: Unlike Article 6(2)(c) of the Biotechnology Directive, efforts affecting stem cell research in the United States have focused largely on government funding rather than the legal scope of patentability. Since 1996, United States appropriations bills have included the Dickey-Wicker Amendment as a rider that explicitly prohibits the use of government funds for the creation of human embryos or for research in which human embryos were destroyed or discarded.
Despite the ongoing developments for government funding of stem cell research, U.S. patent law has long recognized the patentability of stem cells and stem cell research tools. In the recent, well publicized Myriad decision, the Federal Circuit affirmed the principle that biologically pure compositions that do not occur in nature are patentable.The USPTO’s official policy under this principle has been that stem cells and methods of making or using stem cells are patentable subject matter
The conflict: Thus, under current law, hESCs and parthenogenetic stem cells and methods of making or using such cells are patentable in the U.S., but not in the EU. This difference may require research institutions and companies to re-examine their IP, regulatory, and commercial strategies on a jurisdictional basis.
In Europe, institutions may seek to protect hESC and parthenogenetic stem cell innovation through the nondisclosure mechanisms of confidentiality and trade secrets. These institutions will need to carefully evaluate the suitability of seeking patent protection in the U.S., where the disclosure requirements of the patent system stand in conflict to the nondisclosure principles of trade secrets and confidentiality.
In the U.S., institutions that seek patent protection for these same types of innovation will have to consider the absence of prohibition on others reproducing that work in Europe. Thus, these institutions will also need to consider the potential for global protection afforded by confidentiality and trade secrets.
Source: Genetic Engineering and Biotechnology News, December 1, 2011 (Vol 31, No 21)
The U.S. Food and Drug Administration recently approved the first cord blood therapy for use in stem cell transplantation, the first such approval of a stem cell product in the world.
The cord blood cell therapy HEMACORD (manufactured by the New York Blood Center NYBC) was approved for its use in patients with certain blood cancers and some inherited metabolic and immune system disorders, the FDA said.
Cord blood is one of three sources of stem cells used in transplants, the other two are bone marrow and peripheral blood. Once these stem cells are infused into patients, the cells migrate to the bone marrow where they divide and mature. When the mature cells move into the bloodstream they can partially or fully restore the number and function of many blood cells, including immune function.
Source: www.medicaldaily.com, November 11, 2011
Scientists of the Sloan-Kettering Institute in New York have converted human embryonic stem cells into nerve cells that produce the brain chemical dopamine, which is known to play a role in the development of Parkinson's. When these nerve cells were transplanted into the brains of mice, any symptoms of the illness in the animals were eliminated over a three to five-month period. The researchers hope a similar solution can be developed for humans and trials are already planned.
Until now a similar approach in mice using human embryonic cells hasn't worked well, triggering the formation of tumor-like structures. But the New York team developed a new method for creating cells, closer to the way they naturally form. Their findings have now been published in the journal Nature.
The team now plans to create the new cells on a larger scale over a 12-month period, providing enough to graft 100 human patients after extensive safety testing.
Source: Daily Mail Online, November 8, 2011
The UK government has announced the creation of a £30m centre to turn scientists' stem cell research into a new multimillion-pound industry to help drive Britain's recovery.
The cell therapy centre, which will open in April 2012, is part of a £220m programme to turn British skills into manufacturing success. Stem cell research has been selected for the country's second technology and innovation centre as British scientists are world leaders in the development of treatments for conditions including Parkinson's and prostate cancer, and in skin regeneration for burns victims.
The new stem cell centre, which is likely to be located in south-east England, will receive £10m of funding from the government, £10m from other awarding bodies such as the EU, and £10m from companies using the site.
Pharmaceutical firms Pfizer, GlaxoSmithKline and AstraZeneca have committed to the project. But the site is aimed primarily at small British companies, which will use it to exchange ideas and test the production of new products. It will have a test manufacturing facility to help labs convert a couple of grams of potentially life-saving cells into kilos of commercially available product.
The global cell therapy industry is expected to be worth more than £3.1bn a year, according to Professor Chris Mason, professor of regenerative medicine bioprocessing at UCL.
BioInvent International announced that new preclinical data has been presented on its BI-505 programme that demonstrates anti-cancer activity in multiple myeloma and beneficial effect on bone density. The results of an animal study were presented at the American Society of Hematology (ASH) Annual Meeting, San Diego, USA on Sunday 11 December 2011.
Compared to untreated controls, BI-505 treated animals showed a significant reduction of tumour burden induced by primary human multiple myeloma cells as measured by human IgG levels. The active control, bortezomid (Velcade®*, Takeda/ Millennium) also showed significant effect over the placebo control.
A second outcome investigated in the study was bone resorption. This is very important to multiple myeloma patients because, as the disease progresses, lysis, or breakdown, of bone causes increasing pain. This lysis is caused by the shift in balance in favour of osteoclasts, the bone cells in charge of skeletal breakdown, over osteoblasts, bone cells promoting build-up. The study demonstrated that both BI-505 and Velcade had a favourable effect on bone mineral density.
Source: Press Release BioInvent, December 12, 2011
A group led by the National Cancer Institute's Hisataka Kobayashi has developed a fluorescent spray that can label cancer cells within a minute. The hope is that surgeons could apply it during or after a procedure to catch any cancer cells they might have missed.
Several research teams have been working on fluorescent labels for cancer cells that could serve as a visual guide for surgeons, but other methods typically take much longer to work.
The researchers demonstrated the spray's ability to label cancer cells in mice in a study published in Science Translational Medicine. The fluorescence is activated by an enzyme called y-glutamyl transpeptidase that is abundant in tumor cells but not in normal cells. The probe that Kobayashi and his team designed contains a chemical target of the tumor enzyme. The enzyme cleaves the chemical on contact, and this activates the fluorescence signal.
Source: www.Technologyreview.com, November 29, 2011
BioInvent is running a number of projects in the research phase i.e. the stage prior to selection of a Candidate Drug. The Company‟s research portfolio currently includes projects mainly within the areas of cancer and inflammation. In the area of cancer, the research is focused on programmed cell death inducing antibodies with a strong ability to kill tumour cells, as well as activation of the body‟s own immune defence cells. BioInvent is also working in cooperation with a leading academic team in the UK on the possibility of using new therapeutic antibodies to strengthen these mechanisms of action and the effect of already approved and clinically well-tolerated therapeutic antibodies.
With BioInvent‟s F.I.R.S.T. platform, where antibodies are identified directly based on their powerful ability to kill primary cancer cells through differentially expressed, cancer cell-associated surface receptors, the Company is looking for new drug candidates for the treatment of various haematological cancers. The cooperation with leading Swedish and international academic teams was initiated with the objective to develop antibodies for the treatment of serious haematological and solid cancers through new pharmaceutical concepts based, for example, on the role of cancer-associated fibroblasts in tumour growth.
The Company‟s inflammation research is being enhanced by a partnership entered into in March 2010 with the US company Human Genome Sciences. Under this partnership the companies will work together to develop and commercialise antibody-based drugs based on target proteins from Human Genome Sciences‟ research and BioInvent‟s antibody technology. The Company‟s initiatives in oncology and inflammation have in common the development of therapies that impede the functions and activity of myeloid cells.
The Company is also conducting research and development on antibody-based drugs in cooperation with other external partners. Such partners include Bayer HealthCare, Daiichi Sankyo and Mitsubishi
Tanabe. All in all BioInvent has entered into agreements of this kind with the possible development of up to 30 antibody-based products. As well as undisclosed license fees and research funding, BioInvent will receive milestone payments and royalties on sales of any products commercialized.
Source: BioInvent Press Release, October 13, 2011
Clinical trial news
ThromboGenics NV and co-development partner BioInvent International announced that they have completed the enrolment of a 632-patient Phase IIb trial with their novel long-acting anticoagulant TB-402 (factor VIII inhibitor) for the prophylaxis of venous thromboembolism (VTE) after total hip surgery. The trial has recruited patients from 36 centers across Europe.
It is anticipated that the swift inclusion of patients in the final recruitment phase of the study will allow the Companies to report the outcome in the second quarter of 2012. This is ahead of previous guidance, which was the second half of 2012.
This double blind, randomized controlled trial is comparing two doses of TB-402 (25mg and 50mg), given as a single intravenous infusion after total hip replacement, with the recently approved factor Xa inhibitor rivaroxaban, which is given orally (10mg) once a day for 35 days.
TB-402 is a recombinant human monoclonal antibody that has a novel mode of action. It partially inhibits factor VIII, a key component of the coagulation cascade. An important potential benefit of TB-402 is that a single injection provides safe, stable, long-term anticoagulation for approximately one month, depending on the dose. This is expected to lead to reduced nursing time and improved patient compliance.
In 2010, a 316-patient Phase IIa trial comparing TB-402 with enoxaparin for VTE prophylaxis after total knee replacement reported positive results as well.
Source: BioInvent Press Release, December 16, 2011
During 2011, FDA approved 35 new medicines. This was, according to the agency, among the highest number of approvals in the past decade, surpassed only by 37 approvals in 2009.
Of the 35 drugs given the go-ahead, two were approved with a molecular test, making them the first companion drug-diagnostic sanctions. Such tests will be key drivers of drug development in the future. Seven newly sanctioned medications provided advances in cancer treatment, and 10 were for orphan diseases. Notably, the first new therapy for lupus in 50 years was green-lighted this past year.
In terms of how efficiently the FDA was able to do its job, three cancer drugs were given the go-ahead in less than six months. Two-thirds of all the drugs sanctioned last year were completed in a single review cycle and 34 were approved on or before the agreed-upon review time targets.
New cancer drugs: On August 26, FDA green-lighted Pfizer’s Xalkori to treat locally advanced and metastatic non-small-cell lung cancers that express an abnormal anaplastic lymphoma kinase (ALK) gene. The agency approved the drug along with a diagnostic test for the ALK gene abnormality, Abbott Molecular’s Vysis ALK Break Apart FISH Probe Kit. Up to 7% of those with NSCLC, typically patients without a history of smoking, have the gene abnormality.
The other drug-diagnostic approval was Roche’s Zelboraf to treat metastatic or unresectable melanoma and the Cobas 4800 BRAF V600 Mutation Test. Zelboraf, an oral kinase inhibitor, acts by blocking the function of the V600E-mutated BRAF protein. The mutation is present in about half the patients with late-stage melanomas.
In 2011, FDA sanctioned another drug for metastatic melanoma, Bristol-Myers Squibb’s Yervoy. This mAb therapeutic acts by binding to and blocking the actions of the cytotoxic T-lymphocyte antigen 4 (CTLA-4). However, the antibody can cause significant side effects, and severe to fatal autoimmune reactions were seen in 12.9% of patients treated with Yervoy.
Hepatitis C: two drugs for the treatment of hepatitis C won approval in May, 2011—the first new drugs approved for the viral disease in 20 years: Vertex’ Incivek (telaprevir) and Roche’s Victrelis. Both drugs interfere with the ability of HCV to replicate by inhibiting a key viral enzyme (NS3/4A serine protease). The FDA said that it may be possible to shorten Incivek treatment from 48 weeks to 24 weeks in most patients. Given the toxicity of hepatitis C therapies, a shortened treatment period represents a significant advance.
New Lupus drug: Last year also saw approval of the first new drug developed for lupus in 50 years, Human Genome Sciences (HGS) and GlaxoSmithKline’s Benlysta. The mAb therapy was green-lighted on March 9, 2011, for adult patients with active, autoantibody systemic lupus erythematosis who are receiving standard therapy.
Source: Genetic Engineering News, January 9, 2012
Roche announced that the European Commission has approved Avastin® (bevacizumab) in combination with standard chemotherapy (carboplatin and paclitaxel) as a front-line (first-line following surgery) treatment for women with advanced ovarian cancer.
Ovarian cancer is the most deadly of the gynaecological cancers, with approximately 220,000 women diagnosed and 140,000 women dying from the disease each year globally. The approval of Avastin marks a major advance in the treatment of women with ovarian cancer for whom treatment has been limited to surgery and chemotherapy. It represents the first major treatment advance for women with ovarian cancer in 15 years.
Source: Roche Group Media Relations, December 23, 2011
Roche announced that the Committee for Medicinal Products for Human Use (CHMP) has recommended that Zelboraf be granted full marketing authorization as a monotherapy for the treatment of adult patients with BRAF V600 mutation-positive unresectable or metastatic melanoma. Zelboraf represents a personalized medicine.
“The CHMP recommendation to approve Zelboraf represents an important milestone for people with metastatic melanoma who until recently had limited treatment options,” said Hal Barron, M.D., chief medical officer and head, Global Product Development. “We are working closely with health authorities worldwide to bring Zelboraf to people with this deadly disease as soon as possible.” more
Source: Roche Group Media Relations, December 16, 2011
Roche announced today that the European Commission has approved Tarceva® (erlotinib) for use in patients with a genetically distinct type of non-small cell lung cancer (NSCLC) in Europe. This approval will enable the use of Tarceva as a first-line monotherapy in people with locally advanced or metastatic NSCLC with EGFR (epidermal growth factor receptor) activating mutations. Tumours with these mutations are responsive to Tarceva and treatment with this medicine has been shown to more than triple the number of patients whose tumours shrink (response rate). Tarceva has also shown to nearly double the time patients live without their disease progressing (progression free survival – PFS) compared to chemotherapy.
Source: Roche Group Media Relations, September 1, 2011
FDA approval for novel test strips for self-monitoring of blood glucose
Roche announced that it has received clearance from the U.S. FDA for its Accu-Chek Aviva Plus test system. The new maltose-independent test strip is designed to prevent the interference of maltose on blood sugar readings which can occur in rare cases when drugs containing or metabolizing to maltose are parenterally administered. The Accu-Chek Aviva Plus test strip is cleared for patient self-monitoring of blood glucose and can be used with the proven Accu-Chek Aviva system.
Source: Roche Group Media Relations, September 29, 2011
Environmental campaigners are urging developing countries to include the rapidly advancing science of synthetic biology — the building of new organisms using genes as biological 'bricks' — in their biosafety legislation for genetically modified (GM) crops. They are concerned that synthetic biology products based on novel organisms could be developed, and commercialised, before there is regulation and understanding of their environmental and societal impacts.
With many countries in the midst of legislating for the arrival of GM crops, now is the time to include frameworks for dealing with synthetic organisms as well, according to Eric Hoffman, a biotechnology expert at Friends of the Earth (FoE), the environmental campaigning organisation. Hoffman said that, as with GM organisms, legislation is lagging far behind the technology.
Synthetic biology offers the prospect to increase the rate and decrease the costs of discovery and production of biological products, vaccines and medicines, perhaps even to the point that the pharmaceutical industry finds it more economical, or even profitable, to produce medications to address some of the neglected diseases," he said.
Source: SciDevNet, January 17, 2012
It's a well-worn cliché that men dominate the top ranks of the biotech industry. But over the past decade a group of extraordinary women has put that cliché to the test. Perhaps one of the most interesting aspects of our selection of 10 women who have excelled in the industry is that there were so many outstanding women in biotechnology to choose from.
One of this year's group runs a large cancer drug business. One not only founds biotech companies, she also places some savvy venture bets on future stars. Several are CEOs pursuing excellent science and top clinical trial prospects. Some came up through the scientific ranks, others through the business development side of the business.
In an entrepreneurial business like biotech, results are all that matter. So in some respects it's the ideal climate for women who want to reach the top ranks, where the old boy network helps but certainly doesn't rule. And as more women break into these ranks in the years to come, as is certain to happen, this group will become even harder to narrow down to a top 10.
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Phacilitate's North American Cell & Gene Therapy Forum 2012
30 January - 01 February 2012, Washington, DC, USA
More information: www.technologynetworks.com/Stemcells/index.aspx?Login=2&redirect=/Stemcells/eventsid.aspx?ID=126785
Cell Culture 2012
February 2-3, San Diego, USA
Developing Chemical Processes for Active Pharmaceutical Ingredients (APIs)
February 6-7, 2012, Mumbai, India
More information: www.technologynetworks.com
Applications of Synthetic Biology within Biotechnology
February 6, 2012, Barcelona, Spain
Information: www.efb-central.org or to the meeting website
Global ManuCHEM Strategies 2012
13 - 14 February 2012, Berlin, Germany
The 2012 London Regenerative Medicine Event
16 February 2012, London, UK
Cell Line Development & Engineering Asia 2012
20 - 23 February 2012, Shanghai, China
ICNBS Egypt 2012 International Conference
01 - 03 March 2012, Cairo, Egypt
8th World Meeting on Pharmaceutics, Biopharmaceutics and Pharmaceutical Technology
19-22 March 2012, Istanbul, Turkey
More information: www.efb-central.org
CERTIFICATION PROCEDURE , 1992-2012: 20 Years of experience
March 22-23, 2012, Larnaca, Cyprus
5th Proteins Congress 2012
02 - 03 April 2012, Copthorne Tara Hotel, London, UK
Strategies for Commercial Success of Biosimilars in an Increasingly Crowded Market Place
April 19th 2012, LOndon, UK
May 24-25, 2012, Rotterdam, the Netherlands
ISSCR 10th Annual Meeting
13 - 16 June 2012, Yokohama, Japan
ACHEMA 2012 - International Exhibition-Congress on Chemical Engineering
18 - 22 June 2012, Frankfurt am Main, Germany
Bioprocessing and Stem Cells Europe
June 27-28, 2012, London UK
The Scale-Up of Chemical Processes
09 - 11 July 2012, Lake Maggiore, Italy
15th ECB hosted by Turkish Biotechnology Association
23-26 September, 2012, Istanbul, Turkey