ACTIP bulletin no. 70

ACTIP bulletin no. 70, November 2014

In this issue

Biomanufacturing News
Vaccine News
Business News
News from the Commission
Regulatory News
Clinical Trials News
Research News
Stem Cell News


Most innovative EU SMEs 2014

Both the French healthcare biotech SME “Erytech Pharma” and Italian industrial biotech SME “Bio-on” were awarded the EuropaBio’s Most Innovative Biotech SME award 2014. The three runner-ups included: Biosyntia (Denmark), Autifony (UK) and to-BBB (the Netherlands). The top 5 SME candidates were selected from a total of 35 applicants.
Erytech Pharma was awarded for developing an innovative concept to starve cancer cells in specific essential nutrients and quickly induce their death without impairing any healthy cells. Its novel breakthrough technology consists in the use of red blood cells loaded with therapeutic enzyme to develop safe targeted therapies to treat cancer.
Bio-on was awarded for designing and patenting the first fully biobased Polyhydroxyalkanoates (PHA) plastic obtained from agricultural waste, co- and by-products through natural bacterial fermentation processes excluding chemical solvents. The resulting bioplastic is 100% naturally biodegradable in both water and soil and suitable for use in particularly demanding application areas such as: biomedical devices, automotive, food packaging and others.
Source: EuropaBio newsletter, Oct 6-10, 2014.

EFPIA promotes pharma’s contribution to Europe

EFPIA has launched a new online initiative to promote pharma’s contribution to Europe. The European trade body’s new website details industry investment in R&D, levels of employment it brings and its contribution to the trade balance in 32 European countries.
Source: EuropaBio newsletter, Oct 27-31, 2014.


Successful implementation of automation in single use bioprocessing

Webinar presents a case study where the final filtration and dispensing of biologics in a closed single-use system was automated at FUJIFILM Diosynth Biotechnologies.
Key Learning Objectives:

  • Understand the potential benefits of automating single-use bioprocessing steps.
  • Understand how to evaluate the level of automation that is appropriate for your bioprocessing step.
  • Understand critical success factors in implementing a single-use automation project.
  • Understand the capabilities of the automated solution developed by Parker domnick hunter.

Available online upon free registration at:

Biomanufacturing News

Biopharma wary of disposable systems for large-scale production

Janssen mirrored Pfizer’s concerns about the scale, price and robustness of using single-use technology for high-volume production, but says industry collaboration could help bring the required standards in place.
Source:, May 28, 2014.

SAFC to make virus-resistant cell lines

BioReliance and its parent company Sigma-Aldrich Fine Chemicals (SAFC) are working on viral-resistant cell lines that will prevent contamination in bioreactors. The company is working to prevent the propagation of viruses, as opposed to the issue of viral clearance, occurring later in the manufacturing process.
Source:, June 24, 2014.

SAFC analyses cell culture media

Sigma-Aldrich Fine Chemicals (SAFC) is characterising raw materials to reduce delays and cell death in biologics manufacturing. SAFC´s R&D department aims to discover unnecessary materials in dry powder manufacture, which have no bearing on the cell culture process, to eliminate the steps that are just habit. Some of the media currently used in biologics manufacturing are a hangover from the days of microbial work and necessary from growing bacterial, but not for human or Chinese hamster ovary (CHO) cells.
Source:, July 2, 2014.

Production of human stem cells in single-use vessels

Although successful expansion of mesenchymal stem cells (MSCs) in vitro has been well established, the large clinical-scale production of MSCs remains a bottleneck, potentially limiting clinical applications. A recent study by Khandaker Siddiquee and Ma Sha demonstrated the large clinical-scale culture of human adipose-derived MScs in an industrial single-use vessel at 3.75 L working volume. The vessel offered a precision controlled environment for the ideal growth of stem cells under simulated physiological conditions.
Source:, Aug 11, 2014.

The scalable solution to consistent cell culture

From research through to full-scale bio-production, requires a system that delivers reliable results, makes effective use of space and is easy to scale up across a range of applications. A new case study at Thermo Fisher Scientific uses practical examples backed up by experimental data to:

  • Carry out consistent culture expansion
  • Minimize footprint & maximize efficiency
  • Ensure sterility

Examples include the scale-up of viral vaccine production, the efficient production of viral gene transfer vectors and the expansion and differentiation of hMSCs for clinical, research and bio-production use.
For the study see:
Source:, Sept 1, 2014.

Genzyme looks to integrate up and downstream for continuous bio-manufacturing

Genzyme filed a patent application for a technology platform that will bridge the gap between upstream and downstream processing in continuous biomanufacturing. The company claims the platform feeds a liquid culture medium containing the biologic manufactured through a perfusion into a first multi-column chromatography system, capturing the therapeutic protein and then continuously fed into a second chromatography system where it is purified and polished.
Source:, Sept 19, 2014.

Compatible plastics to cut costs and up safety in single-use bioreactors

Sabic (Saudi Arabia) manufacturers industrial polymers for pharmaceutical packaging and drug delivery devices, but the company is now using its plastic for single-use systems in response to the shift from small to large molecular drugs. According to Sabic, biomanufacturing costs and the risk of extractables and leachables can be reduced by the development of more compatible plastics.
Source:, Oct 10, 2014.

Vaccine News

Takeda receives $70M from Japan government for pandemic flu vaccine

Takeda has received a ¥7.2 B ($70M) subsidy from the Japanese government to expand production capacity of its cell-culture pandemic influenza vaccine. The vaccine is manufactured using Baxter´s Vero cell technology rather than traditional chicken eggs, which the company says will allow fast scaling up for swift production in case of an emergency outbreak.
Source:, Apr 30, 2014.

Promising Phase II results for Angenus brain cancer vaccine

The Phase II results for Agenus´ autologous cancer vaccine “Prophage” showed promising results. The single-arm, multi-institutional, open-label study showed that patients with newly diagnosed glioblastoma multiforme who received Prophage in addition to the standard of care treatment lived nearly twice as long as expected.
Patients treated with the vaccine also had a median progression-free survival of nearly 18 months, which is around 2-3 times longer than patients treated with radiation and temozolomide alone. Also, 22% of patients were alive and without progression at 24 months. Each patient received a vaccine tailor-made for them from their own surgically resected tumour, as such, the vaccine works by stimulating the patient’s immune system to attack the tumour-based on the spectrum of mutant proteins it expresses.
Source:, July 1, 2014.

Sanofi Pasteur licenses temp-stable mRNA vaccine from CureVac

Sanofi Pasteur, has signed an agreement with German biopharma firm CureVac to develop a mRNA-based vaccine. CureVac uses an RNA-based platform to develop cancer immunotherapies and prophylactic vaccines as well as adjuvants based on non-coding RNAs for enhancing the immune response of other vaccines. Under the licensing deal Sanofi will fund all R&D manufacturing and commercialization and will have exclusive worldwide marketing rights for the vaccine, whose target is undisclosed.
Source:, July 8, 2014.

FDA approves Protein Sciences cell-based flu vaccines for 2014

The FDA approved Protein Sciences’ strain change amendment for its cell-based seasonal influenza vaccine known as Flublok. The difference between egg-based vaccines produced by other manufacturers and Protein Sciences’ vaccine is that they only need the genetic information to make the exact match to the existing stain vaccine.
Source:, July 11, 2014.

GSK files world´s first Malaria Vaccine

GSK has submitted an application for the world’s first malaria vaccine to the European Medicines Agency (EMA). The vaccine candidate is produced in yeast cells and targets the Plasmodium falciparum malaria parasite, most common in sub-Sahara Africa. The vaccine is designed to prevent P falciparum from infecting, maturing and multiplying in the liver, by triggering the body´s immune system.
Source:, July 25, 2014.

EMA revises guidance on developing flu vaccines

The European Medicines Agency (EMA) has released a second module of a new guideline on influenza vaccines for a six-month public consultation, which covers the non-clinical and clinical requirements for the development of new flu vaccines.
Source:, Aug 1 2014.

Biopharma companies race to develop Ebola vaccine

As the death toll from the world’s most expansive Ebola outbreak nears 1,000, multiple companies are stepping up efforts to bring antibodies and other vaccines to human trials, though none seem likely to be ready until 2015 at the earliest.
Source:, Aug 12 2014.

US FDA approves first needle-free flu vaccine delivery system

For the first time ever, the FDA has approved a needle-free injection system to deliver the seasonal flu vaccine. The approval allows for the administration of bioCSL´s flu vaccine Afluria with a needle-free injection device, known as the PharmaJet Stratis injection system, for intramuscular injection in adults.
Source:, Aug 19, 2014.

Boehringer Ingelheim licences CureVac mRNA vaccine

Boehringer Ingelheim will pay mRNA drug developer CureVac up to €465m for an exclusive global license and development collaboration on CureVac’s potential mRNA vaccine in early clinical development for the treatment of lung cancer. The agreement is part of Boehringer Ingelheim´s commitment to oncology.
Source:, Sept 23, 2014.

Oral influenza vaccine one step closer, says Vaxart

A seasonal flu vaccine (H1N1 influenza) taken in the form of an oral solid dose has been found comparable to currently available jabs, and could be manufactured and distributed faster than injectables, according to Phase I data presented by Vaxart during the 15th Annual World Vaccine Congress held in Brussels in October 2014.
Vaxarta’s solid-dose product contains an adenovirus vector encoded with an avian influenza A hemagglutinin and a toll-like receptor-3 (TLR-3) ligand used as an adjuvant. According to Vaxart, vaccines are delivered to the epithelium of the small intestine where they activate the local immune system of the gut, which in turn generates a broad local and systemic immune response.
The firm is also tackling H5N1 (pre-pandemic influenza) with a vaccine also in Phase I trials, while oral vaccines for the herpes simplex virus and the Ebola virus are in the pre-clinical stage.
Source:, Sept 23, 2014.

CSL pays $275m to acquire Novartis’ flu vaccines and three manufacturing sites

CSL Limited has agreed to acquire Novartis’ flu vaccine business which includes manufacturing facilities in the US (Holly Springs), UK (Liverpool) and Germany (Marburg). This is the second time since April that Novartis has sold off parts of its vaccine business, with the previous sale going to GlaxoSmithKline.
The resulting company, bioCSL, will combine Novartis and CSL’s existing vaccines and pharmaceutical subsidiary, and will create the second largest vaccine company in the $ 4bn global flu industry. The combined business will continue to in-license and distribute a broad range vaccines and specialty pharmaceuticals, and produce blood typing reagents for local use.
Source:, Oct 28, 2014.

Swissmedic OKs trial of GSK’s adenovirus-based Ebola vaccine

Swiss approval for a trial of an Ebola vaccine developed by GSK has been welcomed by the World Health Organisation (WHO). They represent dosing and safety trials held in advance of Phases II and III trials currently scheduled for late 2014 – early 2015. GSK vaccine, which is a modified non replicating version of a Chimpanzee adenovirus that carries a version of a protein present on the Ebola virus, is one of two candidate anti-infective being tested. The second vaccine which will be studied concurrently is rVSV-ZEBOV, a version of a weakened vesicular stomatitis virus (VSV) developed by the Public Health Agency of Canada and licensed by NewLink Genetics. If shown to be safe and effective, either of the vaccines could be scaled up for production during the first quarter of next year, with millions of doses produced for wide distribution in high-risk countries, says WHO.
Source:, Oct 28, 2014.

Business News

Abbott grows branded generics with $3.3B CFR acquisition

Abbott Laboratories will acquire CFR Pharmaceuticals, a Latin American developer of branded generic drugs, for up to about $2.9B, plus assumption of about $430M in debt.
The potentially up-to-$3.3B deal more than doubles Abbott’s presence in branded generics, an area where Abbott remained active after spinning off its branded pharmaceuticals into AbbVie.
The deal also grows Abbott’s presence in emerging markets, especially Latin America, where the diagnostics-and-devices giant becomes one of the continent’s top 10 generic pharma companies.
Source:, May 16, 2014.

Pfizer strikes out with AstraZeneca

Pfizer’s so-called final £69 B ($116 B) offer for AstraZeneca fared no better than several past recent attempts, with the British-based pharma turning down the offer flat, and leaving uncertain what if anything its eager U.S. suitor will do next.
AstraZeneca’s board quickly rejected Pfizer’s £55 ($92.56) per share offer £24.76 ($41.67) per share in cash (45%) and 1.747 Pfizer shares (55%) per AstraZeneca share. The board contended that Pfizer’s offer significantly undervalued AstraZeneca at a time when it was making progress in rebuilding its patent cliff-depleted pipeline and returning to revenue growth by 2017.
Source:, May 19, 2014.

Roche invests 120 M CHF in new production facilities at Basel

Roche announced plans to invest a total of 120M Swiss francs in the construction of new production facilities at its Basel site. 85M CHF will go into a new facility for chemically manufactured medicines, while the remaining 35M CHF will be invested in the expansion and refitting of an existing multipurpose unit for the production of investigational drugs and medicines that are already on the market.
Source: Roche Group Media Relations, May 19, 2014

BioInvent sells back rights to drug development candidate ADC-1013 to Alligator Biosciences AB

BioInvent International AB announced that it has sold back all its rights to drug development candidate ADC-1013 to former partner Alligator Bioscience for an undisclosed sum. BioInvent has successfully manufactured ADC-1013 (a FIND® optimized n-CoDeR® antibody) and will fulfil the remaining CMC activities required to supply drug product to the First-in-Human study, expected to commence by the end of 2014.
Source: BioInvent, Lund, Sweden, May 27, 2014.

Merck & Co. acquires Idenix for $3.85B

Merck & Co. will acquire Idenix Pharmaceuticals for about $3.85 B cash, in a transaction that boosts the buyer’s hepatitis C virus (HCV) pipeline and could set off yet another wave of portfolio-reshaping megadeals. Idenix brings to Merck three clinical-phase HCV drugs that the buyer is evaluating for potential inclusion in future combination therapies IDX21437 and IDX21459, both nucleotide prodrugs, and samatasvir, a NS5A inhibitor.
Source:, June 9, 2014.

Genentech acquires Seragon for up to $1.725B

Genentech agreed to acquire Seragon Pharmaceuticals for up to $1.725B, in a deal that complement the Roche subsidiary’s breast cancer products and R&D with a portfolio led by an early-stage clinical candidate for hormone receptor-positive breast cancer.
Seragon lead candidate ARN-810 is in Phase I clinical trials for patients who have hormone receptor-positive breast cancer and have failed current hormonal agents. ARN-810 is an oral selective estrogen receptor degrader (SERD), a new class of medicines designed to block estradiol action at the estrogen receptor, then change the receptor’s shape in a manner that targets it for elimination by the cell.
Source:, July 2, 2014.

Roche acquires Genia for up to $350M

Roche acquired Genia Technologies, the developer of a single-molecule, semiconductor-based, DNA sequencing platform using nanopore technology. The deal could net Genia shareholders up to $350M.
Genia’s sequencing technology is expected to reduce the price of sequencing while increasing speed and sensitivity.
Source:, June 2, 2014.

Salix & Cosmo in $2.7B merger

Salix and Cosmo Pharmaceuticals agreed to a merger. Salix will become a subsidiary of an Irish-domiciled unit of Cosmo that will change its name to Salix. That will enable Salix to reduce its taxes by re-domiciling in Ireland.
Salix will pay Cosmo about $2.7B in stock, in return for owning Cosmo’s U.S. patents for rifamycin MMX®, for conditions of the colon that include diverticulitis; methylene blue MMX®, designed to aid in the detection of colon cancer; and the ulcerative colitis treatment Uceris®.
Source:, July 9, 2014.

Mylan buys Abbott´s generics business for $5.3B

Mylan will buy the outside-U.S. branded generic drug business of Abbott for about $5.3B. Abbott will in return take a short-term, roughly 21% stake in a new company that will combine the business with Mylan’s existing operations. The new company will re-domicile in the Netherlands, where it takes advantage of the nation’s lower corporate tax rate.
Source:, July 15, 2014.

Top 10 Pharma Firms of 2014

Drug companies ranked according to their market capital so far in 2014.
#1. Johnson & Johnson: $282.48B
#2. Roche: $245.97B (CHF 223.64B)
#3. Novartis: $197.06B (CHF 179.19B)
#4. Pfizer: $183.39B
#5. Merck & Co: $166.75B
#6. Sanofi: $140.33B (€105.00B)
#7. GlaxoSmithKline: $123.25B( £73.02B)
#8. Bayer: $107.02 B (€80.03 B)
#9. AstraZeneca: $92.62 B (£54.87B)
#10. AbbVie: $84.73 B (AbbVie acquired Shire for approx. $54 B in cash and stock)
Source:, Aug 11, 2014.

Roche to acquire Santaris Pharma

Roche agreed to acquire Santaris Pharma, a privately held biopharmaceutical company based near Copenhagen, Denmark. Santaris Pharma has pioneered its proprietary Locked Nucleic Acid (LNA) platform that has contributed to an emerging era of RNA-targeting therapeutics. This new class of medicines has the potential to address difficult to treat diseases in a range of therapeutic areas.
Source:, Aug 4, 2014.

Novartis invests $35M in stem cell tech-company with option to acquire

Novartis has invested $35M for a 15% stake in Israel-based Gamida Cell, which offers stem cell expansion technologies and therapeutics. Novartis will have the option to acquire Gamida Cell for $165M in cash, depending on development milestones connected to Gamida´s NiCord, which is currently being studied in a Phase bI/II study as in investigational therapeutic for leukemia and lymphoma.
Source:, Aug 20, 2014.

Roche to acquire InterMune for $8.3B

Roche plans to acquire InterMune for $8.3B cash, in a deal that expands the buyer’s respiratory product portfolio with the seller’s lead product anticipated for launch later this year, pirfenidone for idiopathic pulmonary fibrosis.
Under the deal Roche will launch a tender offer no later than August 29, to acquire all outstanding shares of InterMune common stock. In return, InterMune agreed to file a statement containing the unanimous recommendation of its board that the company’s shareholders tender their shares to Roche.
Source:, Aug 25, 2014.

Merck KGaA plans to acquire Sigma-Aldrich for $17B

Merck KGaA plans to acquire Sigma-Aldrich for $17B, in a deal that will more than double the acquirer’s presence in life sciences tools and technologies by expanding upon and complementing the operations of the German giant’s Merck Millipore tools subsidiary.
The deal expands Merck Millipore by multiplying its 60,000 products and solutions with the more than 230,000 chemicals, biochemicals, and other essential products Sigma-Aldrich manufactures and distributes to more than 1.4 million customers globally in research and applied labs, as well as in industrial and commercial markets.
Source:, Sept 22, 2014.

Over 700 biosimilars in development worldwide

Over 700 follow-on biologic therapies are now in development, and they are expected to account for around a quarter of the $100 B worth of sales stemming from off-patent biologic drugs by the end of this decade.
It is rare to see a new business segment emerge in any market, but this is what is happening within the biopharmaceutical development industry, with 245 biopharma companies and institutes now developing or marketing biosimilars throughout the world, according to a study by T. Reuters BioWorld.
Biosimilars promise to produce big savings, typically offering 20%-30% discounts on innovator biologics. The report expects them deliver savings of as much as $33 B by 2020 across the EU alone, even though uptake in the region is being held back by EU member states generally not permitting inter-changeability.
The US will permit interchangeability, and this is expected to speed uptake of biosimilars there, leading to lower prices. It also suggests that take-up of the recent WHO proposal for a voluntary global naming scheme could level the playing field for biosimilars and their reference biologics.
Biosimilars offer new hope to patients who previously have had no access to expensive biologic drugs, bringing their power for the first time of markets in Africa, Asia, Eastern Europe and Latin America.
Source:, Sept 30, 2014.

Pall, Thermo, Fujifilm, Merck Millipore show new up & downstream offerings

The products, unveiled at BioProcess International in Boston, will help industry with their upstream and downstream processing, including a new Dip and Read anti-CHO host cell protein (HCP) detection kit for quantitation of residual HCPs (Pall), the first single-use system specifically engineered for microbial fermentation (Thermo Scientific), a new CHO DG 44-derived mammalian expression platform (Fujifilm), three new chromatography resins for Chromabolt pre-packed columns and a new film in Mobius single-use process containers (Merck Millipore).
Source:, Oct 14, 2014.

Amgen chases $ 3bn with the biosimilar market

Despite plans to lay off more than 2,000 employees and decrease 23% of manufacturing facilities, Amgen is preparing for a biosimilar onslaught and expanding its portfolio on biosimilars. In addition to the biosimilars adilimumab, transtuzumab, bevacizumab, infliximab, rituximab and cetuximab programs, Amgen has initiated three additional biosimilar programs. The first Amgen biosimilar is expected to launch in 2017, followed by four others through 2019.
Amgen is also looking to ramp up its biomanufacturing capabilities, which the company believes will significantly cut costs. With a new next-generation biomanu-facturing facility in Singapore to begin commercial production in 2017, the company expects an estimated cost reduction of 60% or more per gram of protein. The $ 200m facility will initially focus on the manufacturing of monoclonal antibodies with new technology and innovation, and once completed, the facility will be fully reconfigurable.
Source:, Oct 29, 2014

B-MS extends biologics manufacturing contract with Lonza

Bristol-Myers Squibb (B-MS) and Lonza announced a multi-year expansion of their existing biologics manufacturing agreement. The contract, which began in 2003, will be expanded to include the production of commercial quantities of a second unnamed B-MS biologic medicine at Lonza’s mammalian manufacturing facility in Portsmouth, New Hampshire.
Lonza currently manufactures the active drug ingredient for rheumatoid arthritis treatment Orencia (abatacept) in Portsmouth, as well as clinical supplies of a investigational biologics medicine for B-MS.
Source:, Oct 31, 2014

News from the Commission

EU ombudsman takes issue with clinical trial transparency moves by EMA

In a letter to the European Medicines Agency, the European Ombudsman Emily O’Reilly has expressed concern that a significant change of policy on trial data transparency could limit the amount of data that becomes available. According to the letter the EMA seems to be planning to limit access to clinical trial data by imposing strict confidentiality requirements and by allowing data only to be seen using an interface provided by the EMA.
Source:, May 20, 2014.

EC adopts new rules facilitating public support for research, development and innovation

The EC has adopted new rules that will facilitate the granting of aid measures by Member States in support of research, development and innovation (R&D&I) activities. The new R&D&I state aid Framework sets out the conditions under which Member States can grant state aid to companies to carry out R&D&I activities. Moreover, the scope of measures that no longer need to be notified to the EC for prior approval has been widened under the new General Block Exemption Regulation (GBER). These new rules will help Member States reach the targets of the Europe 2020 Strategy for smart, sustainable and inclusive growth, while at the same time limiting distortions in the Single Market.
Source: EC press release, May 21, 2014.

New portal helps SMEs go international

To help EU-based SMEs extend their business to markets beyond the EU, a SME Internationalisation Portal was launched. The database is free, open to the public and contains some 300 service providers that cover approximately 1200 support services. The database includes a number of search categories, including:

  • SME’s country of origin
  • Services available in other Member States
  • Services available from the EU
  • Services available in the target market

Public service providers for SMEs looking to expand beyond the EU are invited to register via and add their service offer to the database.
Source: EuropaBio Newsletter, June 16-20, 2014

Founding partner EuropaBio celebrates launch of new public-private partnership on Biobased industries

EuropaBio celebrated the launch of a new Public Private Partnership (PPP) between Biobased Industries enabled by Industrial Biotechnology and the EC for €3.7B. The Bio-based Industries PPP was initiated by EC President Barroso, Vice-President Kroes, Research and Innovation Commissioner Geoghegan-Quinn and Transport Commissioner Kallas as part of the EU’s €20B “Innovation Investment Package”.
The Bio-based Industries PPP is a 7 year initiative with the aim of creating a society and economy which increasingly makes everyday products, such as food, feed, textiles, chemicals and fuels, from locally sourced biomass and wastes, rather than from fossil-based sources.
Source: Brussels, July 9, 2014.

EC outlines priorities for pharma sector in new report

In a report on the industry as a whole, the European Commission says that with an annual output of €220B, and nearly 800,000 employees, the pharma industry is vital for the EU economy, though more needs to be done to meet its full potential. The report addresses main concerns from antibody resistance to a growing elderly population to the rising costs of development and price schemes, which seems to flux across countries. The reports also identifies policy areas where actions have been undertaken and which require further exploring, including setting priorities to develop new therapies, ensuring transparency and ethical behaviour and reinforcing the presence of EU Pharma in the global market.
Source:, July 7, 2014.

EU SME instrument: first 155 winners of grants announced

The EC published a list of 155 small and medium-sized enterprises that will be first to benefit from its new €3B SME Instrument. The 155 SMEs from 21 countries will each receive €50,000 to finance feasibility studies for their projects, and they can also benefit from up to 3 days of business coaching. After that, projects may be considered for further financial support worth up to €2.5M.
The SME instrument was launched under Horizon 2020, the EU’s new €80B research funding programme, to help innovative small firms get innovative projects from the lab to the market.
Source: European Commission, Press release, Sept 18, 2014.

A new EU consortium focuses on antibiotic “husbandry”

Driving Reinvestment in R&D and Responsible Antibiotic Use (DRIVE-AB) is a public-private partnership aiming to tackle the growing threat of antibiotic resistance, helped by EUR 9.4m in funding from the EU Innovative Medicines Initiative (IMI).
Oct 28, 2014.

Regulatory News

US FDA unveils long-awaited biosimilar guidance & industry reacts

The FDA released its biosimilar draft guidance for industry, which details how it will evaluate comparative analytical characterization of the biosimilar in relation to the reference product concerning products for which pharmacokinetic and pharmacodynamic data are required to help demonstrate biosimilarity. The draft guidance is designed to help companies design and use clinical pharmacology studies to help prove that a developing biosimilar is similar to its reference product.
Industry groups BIO and PhRMA, as well as biotech company Genentech, took issue with the US FDA draft guidance. The possible outcome of the analytical comparison of the biosimliar with its reference product includes 4 categories, incl. not similar, similar, highly similar, and highly similar with fingerprint-like similarity. Industry seeks more clarity from the draft on the type or scope of information needed to differentiate similar molecules from highly similar or highly similar with fingerprint-like similarity.
Source:, May 13 & Aug 15, 2014.

100 year-old rules on API impurities to change in 2015

Active pharmaceutical ingredients manufacturers are gearing up for changes to regulations on elemental impurities made by the US Pharmaceopeia (USP). The two chapters will be introduced next year in the USP, laying out new tests for impurities to replace the 100 year old “heavy metals tests” currently in use.
Source:, May 22, 2014.

US FDA offers new supply chain security guidance

As manufacturers will have to identify illegitimate product for trading partners beginning next year, the FDA is now offering pragmatic advice in the form of draft guidance for drugmakers regarding situations where a suspected product might enter the supply chain. The draft guidance comes as the agency is still debating how to create a track-and-trace system that will help to rid the supply chain of fraudulent or falsified medicines.
Source:, June 12, 2014.

Too transparent or too opaque: EMA clinical trial plans criticized

The EMA is being criticized on two fronts with some suggesting its trial data publication plans will drive away drug R&D while others argue the agency is backtracking in a way that will hinder public health research. Industry critics suggest that the draft European laws are a disincentive to innovation that will drive RD away from the EU and endanger international intellectual property agreements. Other critics say the plan does not go far enough and the proposal to only allow researchers to view data online and not take copies will limit the ability to carry out re-analysis of medicines post approval.
Source:, June 12, 2014.

NIH: preclinical research needs more female animals

The US National Institutes of Health (NIH) has unveiled a policy that will balance testing on male and female animals and cells in preclinical tests. Research is skewed towards using male animals and cell, obscuring sex differences that could be vital to clinical studies NIH directors J. Clayton and F. Collins wrote in Nature.
Source:, June 18, 2014.

EMA revises what it classifies as advanced therapy treatments

The EMA Committee for Advanced Therapies (CAT) has revised a reflection paper on the classification of advanced-therapy medicinal products (ATMPs). ATMPs can be classified as either gene therapies, somatic cell therapies, tissue engineered products or combined ATMPs, depending on a whole host of information submitted to the CAT, including active substance and key elements of manufacturing. Manufacturers are encouraged to get their ATMPs classified for free of charge to gain access to incentives, such as fee reductions for scientific advice and the certification of quality and non-clinical data.
Source:, July 2, 2014.

Biologics process prompt WHO call for affordable biosimilars

The resolution made at the 67th annual Geneva session of the World Health Assembly (WHA), the decision-making body of the World Health Organisation (WHO), urges WHO member nations to:

  • Strengthen national regulation to meet public health needs for biosimilars
  • Encourage scientific expertise and develop solid, scientifically based regulatory frameworks for access to affordable, safe, medicines, and
  • Ensure new regulations do not prevent access to quality, affordable biologics.

Source:, June 6, 2014.

EMA concept paper focuses on quality control with animal tests

The EMA has issued a concept paper on transferring quality control methods validated in collaborative trials that is intended to encourage the uptake of better tests in terms of the replacement, reduction and refinement of the use of animals in tests. The need for laboratory/product specific validation is perceived as an obstacle to the implementation of methods that have the potential to replace, reduce and refine routine in vivo tests. The paper proposes a guideline to clarify criteria to be met to achieve product-specific validation and describe the level of validation required for labs and medicinal products that participated in large collaborative studies. The release of a draft guideline is expected towards the end of 2015.
Source:, Aug 5, 2014.

EDQM sets new policy for the testing of bacterial endotoxins

The European Directorate for the Quality of Medicines & Health Care (EDQM), under the European Pharmacopeia, has set a new policy for the testing of bacterial endotoxins (BET), the most common cause of pyrogenicity in pharmaceutical products. The new policy establishes that it’s up to the user to determine whether they come into compliance with the set limits. It states “Where a test is included in the monograph with no specific limit, it is up to the user to set the limit for the substance, based on the following considerations: use of the substance (route of administration, patient population); calculation according to the formula given in general chapter 5.1.10; process capability; or any other considerations raised by the competent authority.” These consequence for users come as a test for bacterial endotoxins is not included in new monographs for substances for pharmaceutical use. However, the requirements of the general monograph Substances for pharmaceutical use (2034) apply.
EDQM said it ran into issues during the elaboration of a monograph, as it is not always known at the level of the manufacturing of the substance whether the substance is to be used for the production of a parenteral preparation and therefore it is not known whether compliance with the BET is needed or not.
Source:, Oct 29, 2014.

EMA rejigs biosimilar guidelines to help developers avoid repeating trials

Reference drugs in biosimilar trials now only need to be ‘representative’ of a product cleared in the EEA not approved themselves under guidelines issued by the EMA. The ruling, detailed in the guidance issued by EMA, is designed to help developers avoid repeating costly early phase research and clinical trials.
The main caveat is that it will be the Applicant’s responsibility to demonstrate that the comparator authorised outside the EEA is representative of the reference product authorised in the EEA. This will involve proving the non-EEA drug is of comparable quality which means that ‘side-by-side analysis of the biosimilar product, from commercial scale and site, with EEA authorised reference product must be conducted’ according to the Agency. Additionally, filers using non-EEA approved comparators for preclinical research and human trials must justify their choice. The overall acceptability of such an approach and the type of bridging data needed will be a case-by-case and/or product-type decision, and is recommended to be discussed upfront with the Regulatory Authorities.
The EMA has already approved 19 biosimilar products including several monoclonal antibodies, the first of which was clear in July 2013, and is seen by many in the industry as one of the most progressive and influential regulation.
Source:, Oct 31, 2014.

Clinical Trial News

Pfizer & Roche join consortium of adaptive trial sponsors

Pfizer and Roche have joined a consortium of clinical trial sponsors that are looking to design and implement adaptive trial designs. The consortium was partly founded by CRO Icon’s new subsidiary Aptiv Solutions. Joining Novartis, Janssen and Eli Lilly, Pfizer and Roche’s membership in the consortium, known as ADDPLAN DF, seems rooted in their desire to improve dose-selection in late-phase clinical work, which remains a major barrier to resolving high failure rates in Phase III trials.
Source:, Aug 5, 2014.

Research News

Breaking through the Blood-Brain Barrier

Researchers from the Harvard Medical School have identified a gene in mice Mfsd2a and the molecule it produces, that may be responsible for limiting the blood-brain barrier’s (BBB) permeability. The BBB helps to maintain the delicate environment that allows the human brain to thrive. It is so discerning that it won’t let medicines pass through. Researchers haven’t been able to coax it to open up because they don’t know enough about how the barrier forms or functions. Because Mfsd2a has a human equivalent, blocking its activity could allow to open the BBB briefly and selectively to let in drugs to treat life-threatening conditions e.g. brain tumours and infections.
Most attempts to understand and manipulate the BBB function have focused on tight junctions, seals that prevent all but a few substances from squeezing between barrier cells. Dr. Gu and her team discovered that Mfsd2a appears to instead affect a second barrier-crossing mechanism that has received much less attention, transcytosis, a process in which substances are transported through the barrier cells in vesicles. Transcytosis occurs frequently at other sites in the body but is normally suppressed at the blood-brain barrier. Mfsd2a may be one of the suppressors. The study was published in Nature.
Source:, May 15, 2014.

New technology traces entire life of a cell

Scientists have developed a novel technique to trace the life history of individual cells back to their origins in a fertilized egg. By looking at the copy of the human genome present in healthy cells, they were able to build a picture of each cell’s development from the early embryo on its journey to become part of an adult organ.
More specifically, by looking at the numbers and types of mutations in a cell’s DNA, the researchers were able to assess whether the cell had divided a few times or many times and detect genetic signatures of the processes of DNA damage and repair that the cells had been exposed to during the life of the individual. Furthermore, comparing each cell’s mutations with those of other cells in the body enabled scientists to map out a detailed tree of development from the fertilized egg. The study was published in Nature.
Source:, June 30, 2014.

Surprise: Only 8.2% of human DNA is functional

Oxford University scientists report that only 8.2% of human DNA is likely to be doing something important, i.e., is functional. This number is quite different from one given in 2012, when a number of researchers involved in the Encyclopedia of DNA Elements project stated that 80% of our genome has some biochemical function.
According to the Oxford team, the 80% claim has been controversial, with many in the field arguing that the biochemical definition of ‘function’ was too broad that just because an activity on DNA occurs, it does not necessarily have a consequence. For functionality you need to demonstrate that an activity matters.
To reach their figure, the group took advantage of the ability of evolution to discern which activities matter and which do not. They identified how much of our genome has avoided accumulating changes over 100 million years of mammalian evolution a clear indication that this DNA matters; it has some important function that needs to be retained.
The rest of the human genome, the non-8.2%, is leftover evolutionary material, parts of the genome that have undergone losses or gains in the DNA code, often called “junk” DNA. The study was published in PLOS Genetics.
Source:, July 25, 2014.

Wyss Institute Launches Organs-on-Chips-Focused Startup

According to Wyss, an Organ-on-a-Chip is a cell culture device that contains hollow channels lined by living cells and tissues that mimic organ-level physiology. The device is capable of producing levels of tissue and organ functionality not possible with conventional culture systems and also allows real-time analysis of biochemical, genetic, and metabolic activities within individual cells. In May 2014, Wyss announced its researchers developed a bone marrow-on-a-chip that reportedly can reproduce the structure, functions, and cellular makeup of bone marrow. The team also developed an instrument that can automate the Organs-on-Chips and link them together by flowing medium that mimics blood, creating a “Human-Body-on-Chips” to replicate whole body-level responses a technology that the institute says could lead to better and more accurate drug testing and reduce the need for animal testing as well.
Source:, July 28, 2014.

NIH awards $14.5M for research on DNA sequencing techniques

The NIH awarded a total of about $14.5M in grants to eight research teams that will use the funds over 2-4 years toward research into new DNA sequencing technologies. The majority of the funding was awarded to 5 universities.
• The Scripps Research Institute $4.4M to produce protein nanopore arrays in order to sequence tens of thousands of DNA molecules in parallel, with the eventual goal of sequencing a human genome in as little as 10 minutes.
• University of California, San Diego, $3.7M to develop a system using microfluidics that will enable accurate genome sequencing of a single mammalian cell.
• UC Santa Cruz $2.29M to sequence single DNA molecules by using a nanopore device consisting of a sensor that touches, examines and identifies each nucleotide in a DNA strand as an enzyme motor moves it through the pore.
• University of Washington, Seattle, $1.7M to develop new molecular biology techniques to efficiently and cost-effectively stitch together genomes across long distances.
• University of Pennsylvania $880,000 to developing a synthetic nanopore from graphene, with the goal of enabling detection of individual DNA bases without the need to slow down the DNA molecule as it passes through a pore.
• The remaining funding was awarded to three companies.
Source:, Aug 4, 2014.

Bioengineers create functional 3D brain-like tissue

Researchers from the Tissue Engineering Resource Center at Tufts University report the creation of 3D brain-like tissue that functions like and has structural features, including grey-white matter, similar to tissue in the rat brain and that can be kept alive in the lab for more than two months.
The scientists have already used the material to study chemical and electrical changes that occur immediately following traumatic brain injury and, in a separate experiment, changes that occur in response to a drug. The tissue could provide a superior model for studying normal brain function as well as injury and disease, and could assist in the development of new treatments for brain dysfunction. The study was published Proceedings of the National Academy of Sciences.
Source:, Aug 12, 2014.

Novel antibiotic class created

Scientists at Massachusetts Institute of Technology (MIT) designed a new class of antibiotic, which seeks and destroys resistance genes in bacteria. The new approach could be used to genetically engineer bacteria in our bodies to become less dangerous. The technology might also lead to new treatments for metabolic diseases like obesity.
In a series of laboratory experiments, the researchers showed they could produce a molecular “conditional-lethality device” capable of highly targeted action against the “bad” bacteria in a consortium of different strains. The new antibiotic uses an RNA-guided nuclease called a “Crispr” to hunt down and chop up target genes inside bacterial cells. The bacteria targeted in the experiments included a strain of E. coli, which can cause severe diarrhoea and kidney failure.
In separate experiments the researchers showed they could change the genetic makeup of bacteria without killing them. The study was published in Nature Biotechnology.
Source: EuropaBio Newsletter, 22-26 Sept, 2014.

Stem Cell News

Brain tumour killed with gel-encapsulated herpes-loaded stem cells

Oncolytic herpes simplex virus (oHSV) appeared to have potential against malignant glioblastoma multiforme, the most common brain tumour in human adults. Researchers of the Harvard Stem Cell Institute, used mesenchymal stem cells (MSCs), which are attractive drug delivery vehicles because they trigger a minimal immune response and can be utilized to carry oncolytic viruses.
After loading human MSCs with oHSV, the researchers injected the gel-encapsulated cells into glioblastoma tumours developed in mice. Then the researchers used multiple imaging markers to track the virus which passed from the stem cells to the first layer of brain tumour cells and subsequently into all of the tumour cells.
Using imaging proteins to watch in real time how the virus combated the cancer, Dr. Shah’s team noticed that the gel kept the stem cells alive longer, which allowed the virus to replicate and kill any residual cancer cells that were not cut out during the debulking surgery. This translated into a higher survival rate for mice that received the gel-encapsulated stem cells. The study was published in Journal of the National Cancer Institute.
Source:, May 19, 2014.

Neurons signal stem cells to make new neurons

Although neuroscientists have long suspected that the brain has some capacity to generate new neurons, they have struggled to uncover details of the brain’s capacity for repair and, possibly, self-improvement. Neuroscientists surmised that new neurons emerge when stem cells activate certain internal processes in response to environmental cues. But neuroscientists were still unsure what the nature of these cues might be.
Duke researchers reported that stem cells respond to requests by adult neurons for additional neurons. The adult neurons constitute a previously unidentified population of choline acetyltransferase (ChAT)+ neurons. Because the neurons reside in the mouse subventricular neurogenic niche, prime real estate around the lateral ventricles, the researchers speculate that it may serve as a kind of factory, fulfilling orders needed to help the learning brain keep up with new tasks.
In the study, newly produced neurons were destined for the olfactory bulb, which occupies a fairly large portion of the mouse brain, accounting for the mouse’s ability to process the sense of smell. New neurons in the olfactory bulb could help support the learning of new scents. The study was published in Nature Neuroscience.
Source:, June 3, 2014.

Stem cell shock articles retracted

Two controversial stem cell articles have been retracted. The articles, prepared by scientists based at RIKEN, asserted that adult cells could be transformed into pluripotent stem cells by means of physical stresses, such as acid shock, instead of genetic manipulation. While the articles immediately gained blockbuster status, they also attracted intense scrutiny. Then, soon enough, skeptical readers pointed to irregularities in the article’s figures. In addition, plagiarized passages came to light. Finally, and perhaps most seriously, the article’s findings seemed to defy replication.
Although all the co-authors of both papers have agreed to the dual retraction, they still hope that their work will be confirmed. The retracted papers will still be hosted on the Nature website, but they have been watermarked to indicate their retracted status.
Source:, July 2, 2014.

Stem cells turned into blood

Researchers at the University of Wisconsin-Madison, along with colleagues at three other institutions, report the discovery of two genetic programs responsible for turning stem cells into both the red and white cells that make up human blood. Their finding is important because it identifies how nature itself makes blood products at the earliest stages of development and provides researchers with the tools to make the cells themselves, investigate how blood cells develop, and produce clinically relevant blood products.
During development, blood cells emerge in the aorta. There blood cells including hematopoietic stem cells, are generated by budding from a unique population of hemogenic endothelial cells. Two distinct groups of transcriptional regulators capable of inducing distinct hematopoietic programs were identified including: pan-myeloid (ETV2 and GATA2) and erythro-megakaryocytic (GATA2 and TAL1), that can directly convert human stem cells into the hemogenic endothelial cells, which subsequently develop into various types of blood cells. The factors were capable of making the range of human blood cells including white blood cells, red blood cells, and megakaryocytes. The study was reported in Nature Communications.
Source:, July 15, 2014.

Stem cells made available for API development by European biobank

The Fraunhofer Institute in Germany and its partners began building a library of pluripotent stem cells in January 2014 collected from patients with specific diseases or genetic mutations. The project know as the European Bank for induced pluripotent stem cells (EBiPSC) is now ready to start supplying the collected stem cells to active pharmaceutical ingredient (API) developers.
Source:, Sept 2, 2014.

Advanced cell technology wins US patent for dendritic cell manufacturing process

Regenerative medicine company Advanced Cell Technology (ACT) has been issued a US patent from the US Patent and Trademark Office covering methods of manufacturing dendritic cells using a scalable process involving a renewable stem cell source as the starting materials. The derivation process covered by the patent provides a means for commercial scale manufacturing of an allogeneic, off-the-shelf immunotherapy platform.
Source:, Sept 10, 2014.

Stem cells directly steered to injured organs

Scientists at the Cedars-Sinai Heart Institute infused antibody-studded iron nanoparticles into the bloodstream to treat heart attack damage. The study addresses a central challenge in stem cell therapeutics: how to achieve targeted interactions between stem cells and injured cells. Although stem cells can be a potent weapon in the fight against certain diseases, simply infusing a patient with stem cells is no guarantee the stem cells will be able to travel to the injured area and work collaboratively with the cells already there.
In an attempt to target healing stem cells to the site of the injury, researchers coated iron nanoparticles with two kinds of antibodies that recognize and bind specifically to stem cells and to injured cells in the body. After the nanoparticles were infused into the bloodstream, they successfully tracked to the injured area and initiated healing. The study was published in Nature Communications.
Source:, Sept 10, 2014.

Human stem cell clock reset to zero

Scientists from the Wellcome Trust, the Babraham Institute, and the European Bioinformatics Institute have found a way to reset human pluripotent stem cells to a pristine state a developmental state equivalent to cells found in an embryo before it implants in the womb (7–9 days old).
The scientists reverted human cells to ground-state pluripotency. Although mouse cells respond to a protein called leukemia inhibitory factor (LIF) entering in a state of naïve pluripotency, human cells do not. To overcome this difficulty, the researchers used re-programming methods to express two different genes, NANOG and KLF2. These genes caused the network of genes that controls the cell to “reboot” and induce the naïve pluripotent state.
The reset human stem cells showed a loss of methylation marks throughout the genome. By removing such marks, the new procedure essentially wipes away epigenetic memories, pushing the cells back to a more permissive state. Without an epigenetic memory of their previous lineages, the cells effectively achieved “blank slate” status and gained unrestricted potential to become any adult cell. The findings were published in the journal Cell.
Source:, Sept 12, 2014.

New Technique produces higher Quality Pluripotent stem cells

To make iPSCs, researchers expose adult cells to a mixture of genes that are active in embryonic stem cells. iPSCs can then be made to differentiate into other cell types such as nerve or muscle. However, the standard combination of reprogramming factors incl. Oct4, Sox2, Klf4, and Myc (OSKM) leads to a high percentage of serious genomic aberrations in the cells.
Scientists at the Hebrew University of Jerusalem reasoned that changing the reprogramming factors could reprogram the adult cells in a more controlled way and yield high-quality iPSCs. Working with mouse cells, the group used bioinformatic analysis to design a new suite of reprogramming factors (Sall4, Nanog, Esrrb, and Lin28 (SNEL).
The new SNEL cocktail created fewer colonies of iPSCs, but approx. 80% of those produced passed the most stringent pluripotency test. This is highly preferable to the traditional OSKM cocktail, which produces a large number of colonies but the majority of which fail the pluripotency test.
The scientists hypothesizes that SNEL may reprogram cells better than OSKM because it does not rely on the master regulators Oct4 and Sox2, which might activate part of the adult cell genome. The study was published in Stem Cell.
Source:, Sept 17, 2014.


Japanese Association for Animal Cell Technology (JAACT) 2014
10-14 November 2014, Kitakyushu, Japan
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