ACTIP bulletin no. 71

ACTIP bulletin no. 71, April 2015

Next ACTIP meetings

ACTIP 25th Anniversary Meeting

Hosted by 4Tune Engineering & ACTIP

6-8 May 2015, Lisbon, Portugal

The meeting is for ACTIP members and individually invited guests.


ACTIP Ulm, Germany

Hosted by Rentschler

3-4 December 2015

The meeting is for ACTIP members and individually invited guests.


In this issue

Training Courses & Webinars
Biomanufacturing News
Vaccine News
Business News
European Commission News
Regulatory News
Clinical Trials News
Research News
Stem Cell News


Training Courses & Webinars

ESACT animal cell technology course, 5th edition

27 Sept – 1st Oct 2015, Llafranc, Spain

The ESACT Animal Cell Technology Course is an introductory course to Animal Cell Technology, providing an overview of the field from the more basic aspects to the final application. It should be of interest to those starting their research activity in Animal Cell Technology, both from Academia or Companies, and to those wishing an up-date of the state-of-the-art of Animal Cell Technology in a short course.

The Course is planned in an intensive four-day schedule with a number of participants limited to 30 in order to facilitate the interaction among them and with the lecturers.

For more information:

Cells for compound screening, 2nd edition

September 23-26, 2015, Llafranc, Spain

The training course covers all important aspects of mammalian cell based screens for the identification of biologically active compounds. The course focuses on high quality cell source and production, cellular assay optimization and assay applications.

The course is intended to train interested individuals (Lab managers, Scientists, Post-Docs, PhD students and Engineers) from Industry and Academia.

For more information:

Training session on the European Pharmacopoeia, 8th edition

9-10 July 2015, EDQM Premises, Strasbourg, France

The programme has been designed to enable participants to expand their knowledge and familiarise themselves with the work and procedures of the European Pharmacopoeia.
The programme covers:

  •  Overview of the European regulatory framework and the organisation of EDQM;
  •  Practical advice on using and interpreting the European Pharmacopoeia General Chapters and Monographs;
  •  Outline of how these monographs are elaborated and revised and of how to participate in this process;
  •  Outline of the policies and processes used to establish the European Pharmacopoeia Reference Standards;
  •  An overview of the regulatory requirements of the Procedure for Certification of Suitability of the Monographs and guidance on preparing a successful dossier.

For more info and registration:

Best practices for developing single-use TFF steps for biopharmaceuticals

20 May 2015, 3pm BST

The webinar will discuss how to select, size and scale the most appropriate Tangential Flow Filtration (TFF) for your application to ensure both development and subsequent manufacturing costs are optimized. The webinar will also describe how the selected filter must be integrated into a single-use manifold in such a way that automation can be applied to ensure process consistency and control.

Key Learning objectives:

  • Understand the benefits of single-use TFF technologies
  • Learn how to correctly select, size and scale TFF filters for single-use applications
  • Learn how to determine the optimum single-use TFF automation strategy
  • Understand how single-use sensor technology can be applied to enhance process control

For info and free registration:

Successful implementation of automation in single-use bioprocessing

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 is to understand:

  • The potential benefits of automating single-use bioprocessing steps.
  • How to evaluate the appropriate level of automation for your bioprocessing step.
  • Critical success factors in implementing a single-use automation project.
  • The capabilities of the automated solution developed by Parker Domnick hunter.

Available online upon free registration at:

Prevention of large-scale cell culture contamination: a risk based approach

A webinar to understand the risks posed by microorganisms and learn practical strategies for implementation of control techniques to provide a robust approach to preventing contamination events in your bioprocess.

Available online upon free registration at:


Biomanufacturing News

Sartorius’ new cell harvesting technology based on brewing sector filtration knowhow

Sartorius has launched a cell harvesting technology, the first to incorporate diatomaceous earth filtration more commonly associated with the brewing industry. The company incorporated biopharma grade Diatomaceous Earth (DE) in its Sartoclear Dynamics clarification system, which it is offering to the bioprocessing industry as a single-use technology for harvesting mammalian cell cultures with high cell densities. DE is a prehistoric algae used commonly as a filtration aid in the food and drink industry.

Source:, April 15, 2015.

SACF cell line platform CHOZN to be used by Chinese cancer drug firm BeiGene

Chinese cancer drug developer BeiGene will create production cell lines using a platform developed by Sigma Aldrich’s customer manufacturing services business. BeiGene will access SACF´s CHOZN cell line platform with the focus being the development of production cell lines for its candidate cancer monoclonal antibodies and protein therapeutics. The CHOZN platform, which is designed to maximize the production of monoclonal antibodies and other recombinant proteins uses SACF´s zinc finger nuclease (ZFN) gene editing technology and the glutamine synthetase CHO cell line launched in 2011.

Source:, March 13, 2015.

Horizon joins UK group to improve key biomanufacturing cell line

Horizon Discovery joined the UK Centre for Process Innovation and University of Manchester to improve the CHO cell line for biomanufacturing applications. The project will focus on engineering CHO cell lines using gene-editing techniques. Trial combinations of knock ins/outs will be performed before introduction into a host cell line. Targets will be chosen for their beneficial effect on recombinant protein expressions.

Source:, Feb 18, 2015.

Novasep signs € 4.7M bio-manufacturing deal with Celladon

Novasep has signed a € 4.7M agreement with Celladon, a clinical-stage biotechnology company, to prepare to supply the drug substance Mydicar for heart failure treatment. The deal covers scale-up and pre-validation studies and facility enhancement engineering at the Novasep bio-production plant in Seneffe, Belgium.

Source:, Feb 18, 2015.

Sartorius: US fully embracing single-use as bioprocess unit leads 13% growth

The US has caught up with Europe in adopting single-use technologies, says Sartorius which reported a 32% growth in sales for the region in 2014. The firms bioprocess solutions arm, which supplies products, technologies and services to the bio-manufacturing industry, represented 70% of the total revenue and within this sector both sales and order intake were up over 18% on last year, to €616M and €653M respectively.

Source:, Jan 29, 2015.

NeoStem Invetech to develop closed processing system for cell therapy

Cell therapy manufacturer NeoStem and contract manufacturer Invetech agreed to develop a new closed processing system for cell therapy manufacturing. Invetech will provide the system design and engineering development and NeoStem will develop applications for performing closed cell processing manipulations. The system will be applicable to a range of cell therapy processes in development and commercial stages, and will consist of an instrument platform, disposable flow path, and operating/application software for automated execution of user-selected protocols.

Source:, Jan 15, 2015.

Bi-specific antibodies: new and improved anticancer weapons?

Monoclonal antibodies are in wide use and recognized as therapeutically successful molecules for the treatment of cancer, inflammatory and infectious diseases, and other disorders. But some scientists argue that despite the clinical success of monoclonal antibodies, room for improvement in functionality remains.

By simultaneous recognition of two different targets, bispecific antibodies (bsAbs) and/or “dual specificity” antibodies can serve as mediators to redirect immune effector cells, such as Natural Killer cells and T cells to tumour cells to enhance tumour destruction. In addition, by targeting two different receptors in combination on the same cell, these antibodies can induce modifications of cell signalling, including the inactivation of proliferation or inflammatory pathways.

Scientists at Genentech have developed an approach to production challenges posed by making an antibody with two or more specificities that are non-immunogenic and that retain Fc region functionality. Christoph Spiess, Ph.D., and colleagues devised a production strategy that relies on the co-culture of two bacterial strains, each expressing a half-antibody and each with its natural light chain. The half antibodies are then assembled into functional bispecific antibodies. The researchers demonstrated that these half-antibodies could self-assemble after co-fermentation and co-purification, further streamlining production.

Source:, Dec 2, 2014.

Industry onboard with single-use, but still chromaphobic

Single-use continues to be adopted by the bioprocessing industry, due to its flexibility, efficiency and lower costs. However the most challenging step in the industries adoption of single-use is in the downstream processing, in particular chromatography systems, according to EMD Millipore.

Source:, Nov 19, 2014.

Extractables & leachables biggest barrier to single-use adoption

While the advantages of single-use technology have been well documented (e.g. low upfront costs, flexibility, expedition of process development, lower skills set requirements, reduction in cross contamination risk), extractables and leachables are the number one problem and hold back the adoption and scale-up of single-use technology, according to Apptech Director Wu Xi. Another limitation is the scalability, but this will be overcome by the development of materials made up of nano-composite fibres, which will strengthen the polymer bioreactor bags for bigger volumes.

Source:, Nov 17, 2014.

ViaCyte wins US patent to manufacture human pancreatic progenitor cells

Regenerative medicine company ViaCyte has been granted a US patent for the manufacture of human pancreatic progenitor cells from definitive endoderm cells. Pancreatic progenitor cells are precursors to endocrine cells and are designed to mature into cells found in the normal human islet that produce insulin and other regulatory hormones, after implantation into a patient.

Source:, Nov 11, 2014.

Directed evolution creates superior CHO cell lines

Directed evolution is an iterative approach where selective pressure is applied to a system to identify variants with desirable characteristics. FUJIFILM Diosynth Biotechnologies used directed evolution to improve CHO cells. The work describes:

  • key steps undertaken by the company to develop new host cell lines through directed evolution approaches.
  • assessments performed to identify the most suitable candidates.
  • how the performance of the final two candidates was measured during an exemplar cell line development process using a model mAb.

More information available at:


Vaccine News

CMOs earn € 8.4M in EU grants to help scale-up manufacturing of thermo-stable vaccines

A new round of €8.4M in grants will help develop a scalable manufacturing process to wean vaccines off of their cold-chain dependency and help ensure they can be manufactured as stable and temperature independent products. The funds, part of which were awarded to CDMO Catalent, will be used for the evaluation, development and manufacturing scale-up of thermo stable and cold-chain independent nano-pharmaceutical virosome-based vaccine candidates. €5.3M is funded as part of EU Horizon 2020 and €3.1M will be provided by the Swiss State Secretary for Education, Research and Innovation. The EU Horizon 2020 project will last 3.5 years.

Source:, April 20, 2015.

Ebola breakthrough? Profectus vaccine succeeds in monkeys

An Ebola viral vector vaccine in development by Profectus BioSciences works in monkeys against the virus strain currently infecting humans in West Africa. A single dose of the vaccine which uses Profectus´s VesiculoVax vector platform protected the monkeys against the Makona strain of Ebola (Ebola Guinea strain), according to a recent study published in Nature.

Source:, April 10, 2015.

ImmunoCellular & PharmaCell to manufacture dendritic cell-based vaccine for trial

European CMO PharmaCell B.V. has finalized an agreement with ImmunoCellular Therapeutics to support its pivotal phase III clinical trial in Europe with ICT-107, a dendritic cell-based vaccine in development for glioblastoma multiforme, a form of brain cancer. Under the agreement PharmaCell will perform a technology transfer from its US-based contract manufacturing partner to its newly acquired Geleen facility in the Netherlands to support the clinical trial with IVCT-107.

Source:, March 19, 2015.

Type 1 diabetes vaccine set for trials this year

A type 1 diabetes vaccine will enter human trials this year according to the leader of the UK research team developing a preventative therapy for the autoimmune disorder. The project was announced by Diabetes UK, which is funding the project with fellow charity JDRF and supermarket group Tesco. The aim is to develop a vaccine that suppresses the immune system part that damages insulin-producing cells in diabetes mellitus.

Source:, March 11, 2015.

Unconventional design shows promise for new herpes vaccine

Herpes is one of the most common sexually transmitted diseases, affecting close to 500 million people worldwide, yet an effective vaccine against the virus has eluded scientists for decades. Howard Hughes Medical Institute researchers at Albert Einstein College of Medicine have designed a new vaccine that was able to prevent both the active and latent infection stages of herpes simplex virus type 2 (HSV-2).

Traditional approaches to HSV-2 vaccine design went with the assumption that the vaccine must stimulate the body to produce neutralizing antibodies, especially against the viral surface protein called glycoprotein D (gD-2), which HSV-2 uses to enter the host cell. Unfortunately, this methodology has not yielded a vaccine that has been able to prevent herpes infections within humans.

The Einstein team took a different approach when designing their live HSV-2 vaccine. They decided to delete gD-2 from the virus’ genome. This incapacitated the virus, making it unable to infect cells or cause disease. The researchers also hypothesized that the gD-2 deletion would stimulate the host immune system to produce different and more efficacious antibodies.

Mice immunized with their newly generated vaccine provided complete protection from subsequent challenges with wild-type HSV-2, either intravaginally or through the skin. Furthermore, the researchers were unable to detect any latent HSV-2 virus at the challenge site in the immunized mice. The study was published online in eLife.

Source:, March 9, 2015.

Production of wart drug with Ebola potential set to restart in New Jersey

Hemispherx will make a candidate Ebola treatment being trialled by the US military at its revamped plant in New Jersey. In trials at the United States Army Medical Research Institute of Infectious Diseases in Forst Detrick, Maryland low concentrations of Alferon “successfully protected human cells against the Ebola virus”. The genital wart treatment Alferon was approved by the US FDA in 1998, but has been off the market since 2008 after costs of production become too high.

Source:, March 5, 2015.

The Bill & Melinda Gates Foundation invests $52M in CureVac for vaccine development

The Bill & Melinda Gates Foundation is investing $52M in CureVac, a German biopharmaceutical company focused on mRNA-based vaccine technologies. The largest investment made by the foundation in a company, will support development of CureVac’s platform technology and the construction of a GMP production facility. The foundation will also provide separate funding for several projects to develop prophylactic vaccines based on CureVac’s mRNA platform. The organizations plan to develop and produce numerous vaccines against infectious diseases affecting people in developing countries.

CureVac said it is pioneering the use of natural and chemically unmodified mRNA as a data carrier to instruct the human body to produce its own proteins capable of fighting a wide range of diseases. Its mRNA vaccines are thermostable, which eliminates the demand for cold-chain storage and infrastructure, according to the company.

Source:, March 6, 2015.

Vaxxas raises $20M for nanopatch vaccine trails

Vaccine patch firm Vaxxas has raised $20M to fund clinical development of its pipeline of influenza, polio and bacterial infection vaccines. The patches consist of an array of nanoscale micro-needles that guide vaccines to the immune cells just under the surface of the skin in an approach that Vaxxas claims results in a stronger immune response that makes adjuvants unnecessary.

Source:, Feb 10, 2015.

Consortium Wins $115M toward work with J&J in Ebola vaccine

According to Johnson & Johnson more than €100M in grants from the Innovative Medicines Initiative (IMI) will support several consortia with global research institutions and non-government organizations (NGOs). The consortia will work with J&J’s Janssen Pharmaceutical Co. to speed up development of an Ebola vaccine regimen. The IMI is awarding the grant funding from its Ebola+ program toward development, manufacturing, and patient education for the vaccine regimen.

Source:, Jan 19, 2015.

Harvard & Korean work to help deliver 3D vaccines

Korean and Harvard University scientists have developed a way to non–surgically inject programmable biomaterial to deliver a 3D vaccine that can more effectively treat diseases like cancer or HIV. Tiny biodegradable rod-like structures made from silica, known as mesoporous silica rods can be loaded with biological and chemical drug components and then be delivered via injection. The rods spontaneously assemble at the vaccination site to form 3D scaffolds, which can recruit and fill up with dendritic cells.

Source:, Dec 19, 2014.

Novartis´ flu vaccine in the clear, says Italian regulators

Novartis’ seasonal flu vaccine Fluad has been deemed safe by the Italian Medicines Agency (AIFA) after testing the two batches connected to over a dozen deaths. The AIFA confirmed that the results of an analysis of the two batches in question came back completely negative. Test results confirm the safety of the vaccine, excluding the presence of endotoxins, and batches conform to the appearance and content of the vaccine antigen of the influenza virus.

Source:, Dec 2, 2014.

Merck & co. licences Newlink´s Ebola vaccine candidate

Merck obtained exclusive rights to the experimental Ebola vaccine candidate being advanced by a subsidiary of NewLink Genetics in return for joining NewLink in researching, developing, manufacturing, and distributing rVSV-EBOV. Merck will be granted exclusive rights to the rVSV-EBOV vaccine candidate which has been developed by Canadian and U.S. researchers as well as any follow-on products arising from the collaboration with NewLink. While the results of the ongoing Phase I trials are pending, the NIH said it will launch in early 2015 a Phase III study to evaluate the safety and efficacy of the rVSV-EBOV vaccine (see under ‘Clinical Trial News’), as well as another experimental Ebola vaccine co-developed by NIAID and GlaxoSmithKline.

Source:, Nov 24, 2014.

IDRI wins $11.9M NIH contract to make thermostable TB vaccine

The IDRI (Infectious Disease Research Institute) was awarded a $11.9M contract from the NIH’s National Institute of Allergy and Infectious Diseases to develop a thermostable tuberculosis vaccine. Currently most TB vaccines have a shelf life of around 2 years if they are continuously stored at refrigerated conditions. The process of lyophilizing a vaccine could allow the shipment of vaccines throughout the world without a continuous cold chain.

Source:, 21 Nov, 2014.


Business News

The top 10 mergers & acquisitions of 2014

The value of 2014’s top deals involving biopharma mergers and acquisitions has surpassed those of 2013:

1.   Allergan, acquired by Actavis: $66B

2.   Forest Laboratories, acquired by Actavis: approx. $28B

3.   Sigma-Aldrich, acquired by Merck KGaA: $17B

4.   GlaxoSmithKline Oncology Business, acquired by: Novartis: $16B.

5.   Merck & Co. Consumer Care Business, acquired by Bayer: $14.2B.

6.   Cubist Pharmaceuticals, acquired by Merck & Co: up to $9.5B

7.   Novartis Vaccines Business (excl. flu products) acquired by GSK: up to $8.85B

8.   InterMune, acquired by Roche: $8.3B

9.   Questcor Pharmaceuticals, acquired by Mallinckrodt: approx $5.8B

10.    Novartis Animal Health Division, acquired by Eli Lilly: approx $5.4B

Source:, Jan 2, 2015.

StemBioSys closes $8M in funding to help launch its stem cell culture system

San Antonio, Texas-based StemBioSys has closed an $8M round of Series A preferred stock financing that it will use to help launch its bone marrow High Performance MicroEnvironment stem cell culture system. The system replicates the 3D space in which stem cells naturally reside and proliferate, which enables users to isolate and grow stem cells. The funding is to ensure the scaling up initial manufacturing operations, along with on-going refinements with new equipment, looking into selling the product by the middle of 2015.

Source:, April 17, 2015.

Sartorius Stedim Biotech acquires BioOutsource

Sartorius Stedim Biotech (SSB) acquired Glasgow-based BioOutsource, which provides contract testing services monitoring the safety and quality of biologicals and vaccines to global biopharmaceutical clients. The privately owned firm earned revenue of around €9M over the past 12 months. “By adding contract testing services, we will be able to support our biopharma clients better in their drive to fast-track their new drug candidates through the development and clinical test phases and facilitate lot release testing in large scale manufacturing,” noted Reinhard Vogt, member of SSB’s board”.

Source:, April 17, 2015.

Celgene Takes $45M Minority Stake in Mesoblast

Celgene agreed to buy a minority stake in Mesoblast valued at $45M. In return, the biotech giant has gained a six-month right of first refusal on Mesoblast’s mesenchymal lineage adult stem cell product candidates designed to treat a variety of diseases and disorders. The diseases and disorders include unspecified oncologic diseases, inflammatory bowel diseases, organ transplant rejection as well as acute graft versus host disease (GVHD), for which Mesoblast in February 2015 announced positive results from its Expanded Access Program (EAP) Protocol 275 study for one of its lead products, MSC-100-IV. A Phase III trial including about 60 paediatric patients with aGVHD, was recruiting patients as of January 7, according to Mesoblast agreed to spend up to $85M in October 2013 to acquire the entire culture-expanded mesenchymal stem cell (MSC) business of Osiris Therapeutics. The deal will give Celgene an approximately 4.5% stake in Mesoblast.

Source:, April 13, 2015.

BMS & uniQure launch up to $1B+ gene therapy collaboration

Bristol-Myers Squibb (BMS) will gain exclusive access to uniQure’s gene therapy technology platform for multiple targets in cardiovascular diseases, under a collaboration that could net the Dutch gene therapy developer more than $1B. The companies agreed to collaborate on up to 10 targets including uniQure’s lead gene therapy program for congestive heart failure. The program is intended to improve clinical outcomes for patients with reduced ejection fraction by restoring the heart’s ability to synthesize S100A1, a calcium sensor and master regulator of heart function.

Source:, April 6, 2015.

Horizon Pharma to acquire Hyperion Therapeutics for $1.1B

Horizon Pharma will acquire Hyperion Therapeutics for about $1.1B in a deal that adds two complementary products to the buyer’s orphan disease drug portfolio. The acquisition will increase from five to seven the number of products marketed by Horizon, and diversify its revenue sources in orphan drugs, the company stated. Horizon will add to its portfolio the two marketed products of Brisbane, CA-based Hyperion Ravicti® oral liquid and Buphenyl® tablets and powder. Both treatments are indicated for urea cycle disorders, a collection of inherited metabolic disorders said to affect about 2,100 people in the U.S., of which approximately 1,100 have been diagnosed.

Source:, March 30, 2015.

Fujifilm buys Cellular Dynamics for $307M

Fujifilm plans to acquire Cellular Dynamics International (CDI) for about $307M. Once the transaction is completed, CDI will continue to run its operations in Madison, Wisconsin and Novato, California as a consolidated subsidiary of Fujifilm. The acquisition will allow Fujifilm to gain entry into the area of iPS cell-based drug discovery support services. CDI’s technology platform enables the production of high-quality fully functioning human cells, including induced pluripotent stem cells (iPSCs), on an industrial scale. CDI’s iCell product catalogue encompasses 12 different iPSC based cell types, including iCell Cardomyocytes, iCell Hepatocytes, and iCell Neurons. Fujifilm has developed highly biocompatible recombinant peptides that can be shaped into a variety of forms for use as a cellular scaffold in regenerative medicine in conjunction with CDI’s products, according to the company.

Source:, March 30, 2015.

Teva to acquire Auspex for $3.5B

Teva Pharmaceutical Industries will acquire Auspex Pharmaceuticals for about $3.5B, in a deal the buyer asserts will not only enhance its revenue and earnings, but strengthen its core central nervous system drug portfolio. The deal has been approved by the boards of both companies, with key Auspex shareholders agreeing to support the acquisition by Teva. Auspex specializes in treatments for movement disorders that apply the company’s deuterium chemistry to known molecules, which according to the company improves their safety and efficacy profiles. Auspex’ deuterium technology platform is being applied in the company’s lead investigational product, the NDA-ready SD-809 (deutetrabenazine), developed for the potential treatment of chorea associated with Huntington’s disease, tardive dyskinesia, and Tourette syndrome.

Source:, March 30, 2015.

BioInvent and CRT expand collaboration with University of Southampton to develop new immunotherapy treatments for cancer

BioInvent and Cancer Research Technology Ltd (CRT), the commercialization and development arm of Cancer Research UK, announce the start of a two-year research collaboration with leading antibody researchers at the University of Southampton.

The program aims to develop new immunotherapy treatments for cancer based on preclinical work by the Southampton group showing the potential to treat cancer using antibodies targeting OX40 and 4-1BB – known ‘co-receptors’ that help stimulate the production of killer T-cells during an immune response.

Source: BioInvent press release, March 30, 2015.

Biotech fund Flagship Ventures raises $537m

The venture capital fund that launched Acceleron, Moderna, and Bind Therapeutics has raised $537M for another round of funding. It is the fifth and largest fund so far for Flagship Ventures, based on Cambridge, Massachusetts, since its founding in 2000. It will invest the funds through its Venture Labs division, which focuses on therapeutics, health technology and sustainability start-ups. The firm plans to launch up to five new companies per year from its next investment round, as well as increasing its staff.

Source:, Mar 27, 2015.

Kite Pharma acquires T-Cell Factory for $21M up front

Kite Pharma acquired privately held T-Cell Factory (TCF) for €21M upfront, and renamed it Kite Pharma EU, in a deal designed to establish a European presence for the buyer and strengthen its T Cell Receptor (TCR) gene therapy product platform. TCF has focused on discovery and development of tumour-specific TCRs for broad use in cancer treatment based on its TCR-GENErator™ platform. TCRs allow targeting of tumour antigens found inside cancer cells, as well as surface antigens.

Source:, March 18, 2015.

Google-backed 23andMe puts gene tech to work in drug discovery

Google-backed gene testing firm 23andMe is creating a therapeutics group, headed by ex-Genentech veteran Richard Scheller. Dr. Scheller will lead an R&D team using genetic data to uncover the biological mechanisms of common and rare diseases to identify new therapies. This therapeutics group will be connected to the 23andMe´s research database. 23andMe was founded in 2006 in Mountain View, California and received $3.9M investment from Google within a year. The FDA granted the company the first ever authorisation to market a genetic test directly to consumers. This allows individuals to check if they are carriers for Bloom Syndrome.

Source:, March 18, 2015.

Concordia buys Covis drugs

Concordia Healthcare will acquire substantially all of the commercial assets of Covis Pharma and Covis Injectables for $1.2B cash, in a deal that expands the buyer’s portfolio of branded and generic drugs. The Covis drugs being acquired consist of 18 branded drugs, authorized generic products, and injectables, in therapeutic areas that include cardiovascular, central nervous system, oncology, and acute care.

Source:, March 9, 2015.

AbbVie acquires oncology drug firm Phamacyclics for $21B

AbbVie agreed to acquire Pharmacyclics and its flagship anticancer product, Imbruvica® (Ibrutinib), for the treatment of hematologic malignancies. AbbVie will pay a total of $21B. AbbVie officials say the acquisition accelerates the company’s clinical and commercial presence in oncology, strengthening its pipeline and establishing its position in hematological oncology, which they label as an “attractive and rapidly growing market” now approaching $24B globally. Imbruvica is a Bruton’s tyrosine kinase inhibitor approved for use in four indications to treat three different types of blood cancers.

Source:, March 5, 2014.

Novartis ordered to halt drug production in Japan for 15 days

Novartis’ Japanese unit has been ordered to temporarily halt production of drugs by Japan’s health Ministry as punishment for delayed reporting of side effects. The drug maker’s unit will also have to stop selling drugs during the period.

Source:, Feb 27, 2015.

Shire steps further into rare diseases with $70M acquisition

Shire acquired privately-held Meritage for an upfront fee of $70M and additional contingent payments based on the achievement of development and regulatory milestones. Under the terms of the deal, Shire has acquired the global rights and undertaken the further development of Meritage´s Phase 3-ready compound, oral Budesonide suspension for the treatment of adolescent and adults with eosinophilic esophagitis, a rare, chronic inflammatory gastrointestinal disease.

Source:, Feb 24, 2015.

Valeant to acquire Salix for $14.5B

Valeant Pharmaceuticals International will acquire Salix Pharmaceuticals for $14.5B, in a blockbuster deal designed to catapult the combined company to leadership in the U.S. gastrointestinal drug market. The boards of both companies have approved the deal, which is expected to close in Q2 of 2015, subject to customary closing conditions and regulatory approval.

Source:, Feb 23, 2015.

Mundipharma associate launches Remsima in UK and prepares for wider roll out

Mundipharma’s associate Napp Pharmaceuticals launched Celltrion’s Remicade biosimilar Remisma in the UK today, just 24 hours after the originator lost patent protection.

Source:, Feb 25, 2015.

LabCorp completes acquisition of Covance

Laboratory Corporation of America® Holdings (LabCorp®) announced the completion of its acquisition of Covance Inc., At closing, the purchase consideration was valued at an equity value of approximately $6.2B and an enterprise value of approximately $5.7B. This combination creates the world’s leading healthcare diagnostics company, providing clinical laboratory services and end-to-end solutions for drug and diagnostics development and commercialization.

Source:, Feb 19, 2015.

Hospira launches first mAb biosimilar in major EU markets

Hospira announced the launch of the first biosimilar monoclonal antibody (mAb), Inflectra (Infliximab), in major European markets, following the patent expiry of J&J’s Remicade. Hospira has yet to disclose a price, but analysts expects it could be 20-30% cheaper than brand Remicade.

Source:, Feb 17, 2015.

Vaxin acquires Immune Targeting Systems

Vaxin acquired Immune Targeting Systems (ITS), in a deal intended to strengthen the buyer’s focus on developing public health and biodefense vaccines. The combined company will have multiple clinical stage assets. These include NasoVAX™, a Phase II-ready seasonal flu program; a hepatitis B therapeutic vaccine, for which the company expects to initiate a Phase I/II clinical study in the first half of 2015. Vaxin’s NasoVAX platform uses needle-free intranasal delivery, with the goal of achieving broad immunity against disease pathogens more rapidly than conventional vaccines.

Source:, Feb 17, 2015.

GSK Acquires GlycoVaxyn

GlaxoSmithKline (GSK) has acquired GlycoVaxyn for $190M, more than two years after taking a minority stake in the vaccine developer as part of a collaboration to develop new antibacterial conjugate vaccines. The companies said they will work to “maintain the autonomy and agility of GlycoVaxyn whilst delivering the scale and support that GSK can provide. GlycoVaxyn is in Phase I clinical trials with a vaccine candidate against E. coli infections, and is expected to launch a Phase I study for a vaccine against Shigellosis (severe diarrhoea) in the U.S. in Q1 2015.

Source:, Feb 11, 2014.

Thermo Fisher Scientific acquires single-use specialist ASI

Thermo Fisher Scientific acquired Advanced Scientifics (ASI), which provides single-use technologies for customized bioprocessing solutions, for $300M in cash. ASI designs, manufactures, and delivers customized single-use systems and equipment for the preparation, processing, storage, and transportation of biopharmaceuticals.

Source:, Feb 5, 2014.

Pfizer to buy Hospira for $17B

Pfizer will acquire Hospira for about $17B in cash and debt, in a deal designed to enhance the buyer’s established drug business and global reach with the potential for growth in injectable drugs and infusion technologies as well as biosimilars. The deal is the first major acquisition Pfizer has announced since last year when it tried but failed to acquire AstraZeneca for $105B. In buying Hospira, Pfizer is seeking to add revenue growth in order to recoup expected losses as many of its top-selling drugs face patent-cliff expirations of exclusivity.

Source:, Feb 5, 2015.

AstraZeneca inks four CRISPR collaborations

AstraZeneca entered into four research collaborations looking to tap into the power of Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) gene-editing technology. Company officials say the technique will allow AstraZeneca to identify and validate new drug targets in preclinical models that closely resemble human disease. The firm will share cell lines and compounds with its partners and work with them to publish findings peer-reviewed journals.

  • The Wellcome Trust Sanger Institute will focus on deleting specific genes relevant to cancer, cardiovascular, metabolic, respiratory, autoimmune, and inflammatory diseases and regenerative medicine to understand their role in these conditions.
  • The Innovative Genomics Initiative zeroes in on either inhibiting (CRISPRi) or activating (CRISPRa) genes to understand their role in disease pathology.
  • From Thermo Fisher Scientific AstraZeneca will receive RNA-guide libraries that target individual known human genes and gene families. AstraZeneca can screen these guides against cell lines to identify new disease targets.
  • The Broad Institute and Whitehead Institute is designed to evaluate a genome-wide CRISPR library against a panel of cancer cell lines with a view to identifying new targets for cancer drug discovery.

Source:, Jan 29, 2015.

Pharma turning to injectable systems to protect biologics

Big Pharma firms like AbbVie are partnering with delivery device makers to guard against biosimilar competition according to Unilife CEO, Alan Shortall. Combining a drug with the exclusive use of a drug delivery system adds real value to compete successfully against biosimilars and other biologics. Humira is one AbbVie drug set for biosimilar competition. The US patent is set to expire in 2016, and there are a number of firms developing biosimilars.

Source:, Jan 22, 2015.

Biotech Initial Public Offerings (IPOs) boom has arrived in Europe

The European biotech industry experienced a stock market boom in 2014. A total of €2.4B was poured into biotech companies in Europe via the stock exchange, an increase of 25% compared to the previous year (2013: €1.9B). The number of biotech IPOs tripled to 15. A total of 150 biotech companies with a market capitalisation of €66B are now listed on the 15 most important stock exchanges in Europe. These are the key findings of the report “Comparative Analysis of European Biotech Stock Markets 2015”, which was presented during a press conference by BIOCOM AG in Berlin, Germany.

Source: EuropaBio Newsletter Jan 19-23, 2015.

Cellectis patent could block CRISPR

A patent issued to French biotech Cellectis threatens to block gene editing tools currently in use by several big pharma and service companies. The broad patent covers all gene-editing technologies that depend on chimeric endonucleases to alter and repair DNA, providing the DNA sequence has at least 12 base-pairs, and only for in vitro applications. Cellectis claims the IP could cover Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) gene-editing tools.

Source:, Jan 9, 2015.

Sartorius sells Intec to focus on bioprocessing and lab

Sartorius AG announced plans to sell its industrial weighing and control tech business, Intec, as part of a long-term strategy to focus on bioprocessing and laboratory equipment.

Source:, Jan 5, 2014.

Otsuka to acquire Avanir for $3.5B

Otsuka Pharmaceutical will acquire Avanir Pharmaceuticals for $3.5B, in a deal intended to expand the buyer’s portfolio of CNS therapies. The acquisition expected to close in Q1 of 2015 combines Otsuka’s psychiatric products, led by the anti-psychotic Abilify® (Aripiprazole), with Avanir’s marketed Nuedexta, a combination of dextromethorphan and quinidine that is the first FDA-approved treatment indicated for Pseudobulbar Affect, and several Avanir drug candidates.

Source:, Dec 2, 2014.

Chinese excipient market set to more than double by 2017

With more than 90 excipient manufacturers in China dedicated to the pharma industry, the country expects its output of excipients to more than double to about $8.9B.

Source:, Nov 26, 2014.

Questions rose over new $2.6B price tag to bring a new drug to market

The Tufts Center for the Study of Drug Development is out with a new study claiming that the average cost of bringing a new drug to market is around $2.6B, though some are saying that number seems inflated. For more information:

Source:, Nov 26 2014.

Benelux biotech sector attracts strong interest from top segment of US investors

KBC Securities, supported by FlandersBio and Flanders Investment & Trade, hosted the 3rd edition of its Annual Benelux Biotech & Healthcare Conference in New York. The conference offered a unique opportunity to meet with top executives of 14 companies and 1 pre-IPO company. At the 1st edition in 2012, the attending companies represented € 9.6B in market capitalization, of which € 2.1B by SMEs. The represented value has increased by over 70% to € 16.3B, of which € 3.8B by SMEs. This value increase was driven by a growth in market value of existing companies (+30% since 2012) as well as the addition of 5 new biotech companies that went public in the last 18 months. Three of the companies were listed on Euronext Brussels and two on the NASDAQ.

Source: EuropaBio Newsletter 24-28 Nov, 2014.

Pharma companies can still improve access to medicines

More Pharma companies are experimenting with innovative access-oriented business models. Companies are granting more licenses to developing country companies to make and distribute generic versions of their medicines, while policies to improve access to medicines continue to get better organized, according to the 2014 Access to Medicine Index.

Source: EuropaBio Newsletter 17-21 Nov, 2014.

Top 7 CROs to exceed 50% market share

As PRA Health Services goes public this week, nearly all of the top CROs are now public and the top tier of these companies could control about half of the outsourcing industry, Parexel CFO Ingo Bank said. The CRO market is growing at a rate of about 7%, and it´s conceivable that 2.3 years from now the top CROs could make up about 60 % of the market. Recent shift to strategic partnerships makes the larger CROs better positioned to capture new opportunities.

Source:, Nov 12, 2014.

Into world of single-use with two acquisitions

Lab service provider VWR is moving into the industry of single-use bioprocessing with the acquisitions of the companies Integra Companies and STI Components. Integra companies provide single-use connectivity solutions including tubing, hoses and gaskets for biopharma companies.

Source:, Nov 11, 2014.


European Commission News

Commission Expert Group on Rare Diseases

The Commission Expert Group on Rare Diseases gathered on 12-13 March in Luxembourg. The meeting focused on key developments on European Reference Networks for rare diseases, rare diseases registries and genetic testing. The report can be consulted at:

Source: EuropaBio Newsletter, March 27, 2015.

SME instrument: essential tips for applicants

The Executive Agency for SMEs published a new list of tips for potential applicants to the Horizon’s 2020 SME Instrument, a EC funding and coaching programme. Read carefully these pointers, which could make a difference to the success of your project. More info at:

Source: EuropaBio Newsletter, March 13, 2015.

EC to launch new rare disease joint action

Vytenis Andriukaitis, the European commissioner for health, urges member states to do more to combat rare diseases. He announced that a joint action on rare diseases will be set-up in 2015.

Source: EuropaBio Newsletter Feb 27, 2015.

EC supports innovative SMEs with €117 M in new grants

Over 275 innovative small and medium-sized enterprises (SMEs) are on track to receive a total of €117M in new grants from Horizon 2020, the EU’s research and innovation funding programme. These are the latest results of calls under the new €3B SME Instrument, launched under Horizon 2020 to help innovative small firms get ideas from the lab to the market and help generate growth and jobs for Europe. This new funding brings the total amount from the SME Instrument so far destined for innovative companies to €125M.

More info at:

Source: EuropaBio Newsletter 12-16 Jan, 2015.

Building a new innovation fast track to bring ideas quicker to the market

The EC has launched the new, €200M Fast Track to Innovation (FTI) pilot initiative to promote innovation by reducing the time it takes to bring innovative ideas to market. Over the next two years, the pilot initiative will be open to all types of participants, particularly from industry, while stimulating first-time applications from industry and SMEs to Horizon 2020, the EU’s research and innovation funding programme. Projects funded under the pilot are to be ‘business-driven’ to ensure promising innovative ideas are exploited faster.

More info at:

Source: EuropaBio Newsletter 12-16 Jan, 2015.

EFPIA and IMI launch €260M Ebola programme

The programme will result in projects aimed to address the development, manufacture, transport and storage of vaccines. Further calls for proposals will be launched in the coming months. The first projects are expected to begin in early 2015.

More info:

Source: EuropaBio Newsletter 12-16 Jan, 2015.


Regulatory News

Record number of medicines for rare diseases recommended for approval in 2014

In 2014, the European Medicines Agency recommended the highest number of orphan designated medicines for marketing authorisation in a year. Out of the 82 medicines for human use recommended, 17 are intended for the treatment of a rare disease, providing therapies for patients who often have only few or no treatment options. Among them is the first medicine for the treatment of Duchenne muscular dystrophy (Translarna) as well as the first treatment for erythropoietic protoporphyria, a rare genetic disease which causes intolerance to light (Scenesse). The past year also saw the first recommendation worldwide of a therapy based on stem cells. The orphan medicine (Holoclar) is a treatment for limbal stem cell deficiency (LSCD), a rare eye disease.

Source: EuropaBio Newsletter 12-16 Jan, 2015.

Netherlands Medicines Evaluation Board says no ‘relevant differences’ between biosimilars and reference products

The Medicines Evaluation Board (MEB) of the Netherlands has updated its position about biosimilars, saying the relatively new products “have been proven to have no relevant differences compared to an innovator biological medicinal product as far as quality, safety and efficacy are concerned”. Since 2010, when the MEB adopted its first position on biosimilars on that patients ”must be kept on a biological medicinal product as much as possible if they respond well to it clinically”, the board considered the most recent literature and experiences in the evaluation of biosimilars and deemed that “this strict condition is no longer valid”.

Source:, April 8, 2015.

EU approves Roche’s Avastin plus chemotherapy for women with advanced cervical cancer

Roche announced that the EU approved Avastin (bevacizumab) in combination with standard chemotherapy for the treatment of adult patients with persistent, recurrent or metastatic carcinoma of the cervix. Unlike the majority of cancers, cervical cancer is most commonly diagnosed in younger women, between the ages of 35 and 44.2 Each day it is estimated that 90 women are diagnosed with cervical cancer in Europe, and around 35 of these women will die from the disease. Avastin’s EU approval is an important development in a disease area where, until now, treatment options were limited to chemotherapy.

Source: Roche Media Release, April 8, 2015.

UK politician pledge would ban 89% of animal research, say scientists

British members of parliament have signed an anti-animal cruelty pledge, which scientists warn would “cripple” disease research and outlaw 89% of preclinical animal research. The promise calls for a ban on experiments on cats and dogs an end to “non-medical” animal testing, monkey importation, genetic modification of animals and extreme suffering, as well as greater government disclosure.

Source:, March 31, 2015.

Obama administration lays out plan vs. antibiotic-resistant bacteria

Obama’s administration laid out a five-year, five-point plan for combating and preventing antibiotic-resistant bacteria. According to the administration cited statistical estimates from the U.S. Centers for Disease Control and Prevention (CDC), drug-resistant bacteria cause 2 million illnesses and about 23,000 deaths each year nationwide. The National Action Plan five priorities should underpin federal policy across agencies to lower those numbers as well as govern the spending of the $1.2B sought by the president against resistance in FY2016. The five priorities are:

  • Slowing the emergence of resistant bacteria and preventing the spread of resistant infections through “judicious” use of antibiotics in healthcare and agriculture settings;
  • Strengthening national disease surveillance efforts, based on a “one-health” approach that according to CDC “recognizes that the health of humans is connected to the health of animals and the environment”;
  • Advancing the development and use of rapid diagnostic tests, that could reduce unnecessary antibiotic use;
  • Accelerating basic and applied research and development, with the goal of increasing the number of candidate drugs at all stages of the development pipeline.
  • Improving international collaboration and capacities, such as the establishment of a common U.S.-European Union system for sharing and analyzing bacterial resistance patterns for priority pathogens, and elevation of antibiotic resistance as an international priority for global health and security.

Source:, March 27, 2015.

How does EMA calculate it fees?

The European Medicines Agency has published an explanatory note on the fees payable to the Agency for its monitoring of the safety of medicines authorised in the European Union. The document describes types of fees, fee exemptions and explains how the Agency charges and collects fees from marketing-authorisation holders of medicinal products for human use.

Source: EuropaBio Newsletter, March 13, 2015.

US FDA expects more resources to fund biosimilar reviews and plant inspections

Biosimilars will mean more facility inspections and reviews according to the US FDA, which is confident its funding will be increased accordingly. The FDA approved its first biosimilar last week, giving Sandoz`s Zarxio the thumbs up in a decision that was widely expected and seen as the breakthrough the copycat biologics sector was waiting for (see article below). It remains to be seen if biosimilars take off in the US. What is certain is that they are already increasing the FDA´s workload.

Source:, March 10, 2015.

FDA approves first US biosimilar, Sandoz´s Zarxio

The FDA approved ZarxioTM (Filgrastim-sndz) for the treatment of neutropenia associated with chemotherapy. The approval includes all indications included in the reference product’s (Neupogen) label. Sandoz, a Novartis company, thus became the first pharma firm to receive approval of a biosimilar in the U.S. through the new FDA biosimilars pathway. “Filgrastim has proven clinical value in treating patients at increased risk of neutropenia, but it is underused in the U.S. for a variety of reasons, including price,” said Louis Weiner, M.D., chairman of the department of oncology and director of the Lombardi Comprehensive Cancer Center at Georgetown University. “Biosimilars have the potential to increase access and the approval of Zarxio may reduce costs to the healthcare system”.

Updates: A district court judge from California has denied an injunction by Amgen meant to stop the launch of the first biosimilar approved by the US FDA Sandoz’s Zarxio (filgrastim-sndz).

Sources:, March 6, 2015; and for update:, March 20, 2015

Testoterone-makers must conduct cardio clinical trial

The US FDA has ordered makers of testosterone therapies to change their labelling and conduct clinical trials to investigate heart attack and stroke risk. The regulation arose after an FDA Advisory Committee found studies and expert input that associates testosterone with a possible increased cardiovascular risk.

Source:, March 4, 2015.

Apotex´s Neupogen biosimilar could be battling Sandoz´s copycat in the US in 2015

Apotex’s Neupogen (Filgrastim) copycat Grastofil could be on US pharmacy shelves as early as December 2015, after the US Food and Drug Administration (FDA) started its review this week.

Source:, Feb 26, 2015.

WHO advocates use of “smart” syringes to stop disease spread

‘Smart’ syringes that can’t be reused should be the drug and vaccine delivery technology of choice for healthcare programmes according to the WHO, which says needle reuse is spreading deadly infectious diseases.

Source:, Feb 23, 2015.

SME registration procedure to reduce administrative burden

The European Medicines Agency (EMA) has simplified the procedure for the renewal of the status of micro, small or medium enterprise (SME). The changes will reduce the administrative burden for companies that are already registered as SMEs with the Agency and wish to maintain their SME status.

Source: EuropaBio Newsletter 9-13 Feb, 2015.

FDA seeks modest budget bump for 2016

The US Food and Drug Administration is requesting a budget of $4.9B for fiscal year 2016, a 9% increase over the enacted budget for 2015. The overall request includes $147.7M for initiatives tied to several key areas, including the regulation of next generation sequencing services, management of drug shortages and inspections. The funding for the work with drugs and biologics would remain largely flat compared to 2015.

Source:, Feb 3, 2015.

MHRS says quality system deficiencies are most common issue from inspectors

Deficiencies related to manufacturers’ quality systems are by far the most prevalent issue cited from inspections by the UK’s MHRA (Medicines and Healthcare Products Regulatory Agency), according to a report on the 2013 inspections. For more details and the report:

Source:, Nov 20, 2014.

China´s draft biosimilar guideline mirrors EU & US in more general ways

Like the US and EU, China’s SFDA is looking to ensure that approved biosimilars are proven to be similar to their reference drugs, according to an unofficial translation of the guideline by BioCentury.

Source:, Nov 12, 2014.


Clinical Trials News

WHO: incentives and enforcement needed to improve trial data transparency

Publishing summary clinical trial results poses no threat to patient privacy according to the WHO, which says enforcing transparency laws and incentivizing sponsors is key to making sure data from all trials is reported.

Source:, April 15, 2015.

BioInvent plans to initiate clinical development of TB-403 with ThromboGenics

BioInvent announced that a Phase I/IIa program with TB-403 in medulloblastoma patients is expected to be initiated during the second half of 2015. Medulloblastoma is a rare, life-threatening cancer that exclusively affects children and adolescents. The favourable safety profile of TB-403 has been demonstrated in previous clinical trials in patients with other diseases. The project is conducted in collaboration with ThromboGenics NV.

Source: BioInvent press release, March 17, 2015.

Study finds most US clinical trial data are not publically available in a timely fashion

Despite legal reporting requirements, the vast majority of results from clinical trials are not reported on within a year of their ending, according to a new study from the New England Journal of Medicine .

Source:, March 13, 2015.

Tekmira Ebola RNA drug starts phase II trial

A clinical trial of Tekmira’s non-vaccine Ebola therapy began in Sierra Leone. The Phase II study of the drug candidate is the second to be financed by the UK Welcome Trust´s $4.8M Ebola fund. An earlier trial of the antiviral Brincidofovir was abandoned this year following a sharp drop in Ebola cases at the study site in Liberia. This second candidate is a synthetic small interfering (siRNA) drug, which targets the virus strain behind the outbreak in Guinea, Liberia and Sierra Leone. It works by blocking certain Ebola genes to reduce viral replication.

Source:, March 13, 2015.

Merck-NewLink Ebola vaccine trail starts in Guinea

A Phase III trial of Merck & Co.’s Ebola vaccine has been launched in Guinea. rVSV-ZEBOV is a recombinant vector vaccine based on the vesicular stomatitis virus and expresses glycoproteins of the Zaire Ebola strain. A second vaccine will be tested in a sequential study, as soon as supply becomes available. The trial will be run by WHO, Health Ministry of Guinea, Médecins Sans Frontières, Epicentre and the Norwegian Institute of Public Health, following promising data from early clinical trials last year.

Source:, March 10, 2015.

Germany´s IQWiG questions transparency of proposed database of EU clinical trial results

The German Institute for Quality and Efficiency in Health Care (IQWiG) says the EMA’s proposal to establish a publicly accessible database of clinical trial data does not do enough to make the data transparent.

Source:, Feb 27, 2015.

Roche´s Phase III study of Gazyva/Gazyvaro showed significant benefit in refractory indolent non-Hodgkin´s lymphoma

Roche announced positive results from the Phase III GADOLIN study, which evaluated treatment options for people with indolent non-Hodgkin’s lymphoma who are refractory to MabThera®/Rituxan® (Rituximab) treatment. At a pre-planned interim analysis, an independent data monitoring committee determined that the study met its primary endpoint early. The study showed that people lived significantly longer without disease worsening or death (progression-free survival, PFS) when treated with Gazyva (Obinutuzumab) plus bendamustine followed by Gazyva alone, compared to bendamustine alone. The study was stopped prior to its protocol-specified final analysis due to the high level of benefit seen in the Gazyva arm compared to the bendamustine arm. There were no unexpected adverse events with Gazyva.

Source: Roche Group Media Relations, Feb 4, 2015.

BioInvent´s BI-1206 antibody to enter collaborative Phase I/II trail

BioInvent has reached an agreement with Cancer Research UK, Cancer Research Technology (CRT), and Leukaemia & Lymphoma Research (LLR) to take its investigational drug, BI-1206, into a collaborative phase I/II trial for patients with chronic lymphocytic leukaemia (CLL) and non-Hodgkin lymphoma (NHL). The first in man study will be funded and conducted by Cancer Research UK, CRT and LLR. BioInvent has been granted the option to take up an exclusive license to the study data, subject to payment of milestones and royalties to Cancer Research Technology.

A recent study, partly funded by Leukaemia & Lymphoma Research and Cancer Research UK, has shown that some cancer cells are able to draw monoclonal antibodies inside themselves, making them invisible to immune cells. However, the researchers showed that the new BI-1206 antibody developed by BioInvent, can effectively prevent this drug destruction process and enhance cancer killing by binding to a molecule called FcgRIIB. BI-1206 showed success in mice in overcoming resistance to monoclonal antibodies like rituximab, currently used to treat different types of lymphoma and leukaemia. The study was published on the April 13, 2015 online issue of the journal Cancer Cell.

Source: BioInvent press release, Jan 20 & April 14, 2015.

Phase I success for GSK Ebola vaccine speed-up trial

Regulatory fast-tracking and researchers working weekends are behind GSK’s speedy Phase I Ebola vaccine trial, the company says. The candidate was well tolerated and produced an immunological response in all 20 American volunteers who received it during a trial by the National Institutes of Health.

Source:, Nov 28, 2015.

Medidata: prepare to embrace digital biomarkers in trials or lose competitiveness

The advent of Big Data in clinical trials will mean companies not using digital biomarkers are unlikely to exist by 2025, according to the president of cloud-based technology firm Medidata. Some clients have strategies based on redefining the way efficacy is demonstrated using mHealth generated biomarkers/surrogate endpoints. The tipping point will come once a pharma company is successful in convincing a regulator or payer such data is more valuable than the standard.

Source:, Nov 25, 2014.

Analysis finds 100% SDV has minimal impact on overall data quality

An analysis of clinical trial data by Medidata and industry group TransCelerate BioPharma has found that the value of Source Document Verification (SDV) in a risk-based approach to clinical trial site monitoring is minimal. The analysis revealed that only 1.1% of total site-entered electronic Case Report Form data are correct as a results of SDV. The companies say that the results support the TransCelerate-proposed methodology for risk-based monitoring.

Source:, Nov 20, 2014.

China sets new requirements for hospitals running clinical trials

The Chinese authorities are supplementing their current regulations around clinical trials with new mandates for the hospitals that conduct the studies. New requirements include the need to establish internal rules and standards of procedures for administrating clinical studies, to centralize the financing at the hospital level and maintain constant supervision of trials.

Source:, Nov 10, 2015.

Research News

Genentech makes cell line identification faster and cheaper

Genentech scientists have created a faster, cheaper way to identify cell lines, a boom for CMOs and preclinical researchers. The researchers proposed a framework for cell line annotation using short tandem repeats and single nucleotide polymorphisms profiles. The blueprint sets out a way of combatting problems of cell line misidentification, contamination and poor annotation. The framework was published in Nature.

Source:, April 22, 2015.

Returning pancreatic cancer cells to normal

A silent disease is often characterized as showing little to no signs or symptoms until the disease has progressed to an advanced stage. Pancreatic adenocarcinoma is one such type of disease and accounts for approximately 85% of all pancreatic cancer cases. There are few treatment options for this disease and prognosis is often very poor, yet new research from a collaboration of scientists at the University of California, San Diego, Purdue University, and led by a team at Sanford-Burnham Medical Research Institute (SBMRI) in La Jolla, may provide some much needed hope for the future.

The researchers were able to observe that pancreatic cancer cells could be coaxed to revert toward a more normal acinar cell phenotype with greatly reduced tumorigenic properties. The investigators achieved this through the addition of the transcription factor E47, which typically controls genes involved in growth and differentiation.

“For the first time, we have shown that overexpression of a single gene can reduce the tumor-promoting potential of pancreatic adenocarcinoma cells and reprogram them toward their original cell type,” said Pamela Itkin-Ansari, Ph.D., adjunct professor in the Development, Aging, and Regeneration Program at SBMRI. “Thus, pancreatic cancer cells retain a genetic memory which we hope to exploit.” The study was published in Pancreas.

Source:, April 21, 2015.

New method holds promise for treating deadly brain cancer

Glioblastoma multiforme (GBM) is the most common and aggressive malignant primary brain tumor that affects humans. To date there are very few treatment options and overall patient prognosis is poor. However, recent findings from researchers at Northwestern Medical School look at a new treatment option that has the potential to dramatically change the outcome of patients afflicted with this form of brain cancer.

The traditional standard of care for GBM has been targeted radiation and chemotherapeutic drugs, which are designed to damage DNA in rapidly dividing cancer cells, preventing them from further proliferation. The current study looks to deliver a microRNA molecule to target genes that are overexpressing proteins thought to play a major role in tumor progression.

“Our study identified miR-182 as a glioblastoma tumor suppressor that reduces the expression of several oncogenes that promote cancer development,” said Alexander Stegh, Ph.D., assistant professor of neurology at Northwestern University Feinberg School of Medicine. Dr. Stegh and his team developed a nanostructure delivery vehicle, called spherical nucleic acids (SNAs), that could traverse the blood-brain barrier to deliver the miR-182 microRNA molecule to the tumor cells. Once miR-182 gained access to the cell it was able to down-regulate several oncogenes involved in tumor development. The study was published in Genes & Development.

Source:, April 6, 2015

DNA “Lego” to accelerate bio-drug production

A new technique for creating DNA constructs will accelerate development of bacterial strains for drug production. The “BASIC system”, uses standardized DNA parts that, like Lego, have the same joining device, so parts will fit together in any order. The system has advantages over others in being able to put multiple pieces of DNA together simultaneously. The technique could offer industry a way to automate the design and manufacture of DNA and it has been developed as an open source method. BASIC will be used in a high-throughput automated process in SynbiCITE, the innovation and knowledge centre based at Imperial College of London, which is promoting the adoption of synthetic biology by industry.

Source:, March 18, 2015.

More funding for biotech research

Funders in the US, Canada and the UK have offered nearly $2m to advance research into progressive neuro-degenerative diseases. The Biomarkers Across Neurodegenerative Disease (BAND) programme is a joint initiative of the Alzheimer’s Association and the Michael J. Fox Foundation for Parkinson Research in the US, the Weston Brain Institute in Canada and Alzheimer´s UK, which joins as the programme makes a second round of research grant award available. The projects investigate the connection in the biology and clinical symptoms of Alzheimer’s, Parkinson’s, frontotemporal dementia (FTD) and other brain deteriorating diseases.

Source: EuropaBio Newsletter, March 13, 2015.

DNA “wormholes” increase cancer risk

Single-letter genetic variations within parts of the genome once dismissed as ‘junk DNA’ can increase cancer risk through wormhole-like effects on far-off genes, according to researchers in the U.K. They found that DNA sequences within “gene deserts,” so called because they are completely devoid of genes, can regulate gene activity elsewhere by forming DNA loops across relatively large distances. The research, led by scientists at The Institute of Cancer Research, London, helps solve a mystery about how genetic variations in parts of the genome that don’t appear to be doing very much can increase cancer risk. Researchers developed a new technique to look at the looping interactions and discovered that single-letter DNA variations linked to the development of bowel cancer were found in regions of the genome involved in DNA looping. The study was published in Nature Communications.

Source:, Feb 19, 2015.

Is epigenetics giving your cells culture shock?

Scientists from the MRC Human Genetic Unit at the University of Edinburgh, U.K. and Linköping University, Sweden, have revealed how quickly cells change their molecular identity when grown under laboratory conditions.

“We were astonished by the speed and spread of the changes. Many cultured cells used in research have been grown for decades and as a result are likely to have very different properties from the cells they supposed to model. This suggest that we have to be circumspect about the interpretation of some previous experiments, and our data reinforces a growing realization that cell line models of human diseases, particularly cancer, can be poor surrogates for many aspects of in vivo biology”.

To determine the adaptation to culture conditions the researchers isolated genomic DNA from primary mouse embryonic fibroblasts at various time-points and from mixed gender. While they found a number of indicators that cultured cells endured an array of changes under the laboratory environment, the most pronounced alteration was observed with 5-hydroxymethylcytosine. The researchers also noticed extensive changes in gene expression for over 7,200 genes from the cultured cells. Many of these changes reflected the adaptation process onto a plastic 2D surface, as they were genes linked to cell adhesion pathways. Interestingly, some of the changes due to cell culture adaptation could be prevented with the addition of vitamin C to the culture media. The study was published in Genome Biology.

Source:, Feb 4, 2015.

Sensing the cramped conditions of intracellular living

The ability to accurately measure the effects of molecular crowding within cells is an area that has eluded researchers for many years. However, scientists at the University of Groningen in the Netherlands have developed a new molecular sensor that provides quantitative information about the concentration of macromolecules in mammalian and bacterial cells.

Despite its importance, molecular crowding is often neglected in biochemical studies due to a lack of dependable measurement techniques. In this study, scientists were able to measure molecular congestion inside live cells at high resolution and in real time. This is a considerable step forward, since previously it was only possible to estimate measurements from average concentrations and cell volumes.

The investigators designed a protein spring that takes advantage of Förster resonance energy transfer (FRET) technology. The protein spring, which contains fluorescent markers on both ends, is excited by a laser that causes one end to fluoresce blue. The blue light than excites the second marker on the opposite end, which will then emit a yellow fluorescence. The transfer of energy from one marker to the other is directly proportional to the distance between the markers. Dr. Boersma and Dr. Poolman designed the protein structure to contain a series of α-helical peptide linkers that act like a hinge spring, allowing the whole protein to change conformation under crowded conditions. Furthermore, they encoded the sensor on two separate artificial genes, one for bacterial and one for mammalian cell systems. The study was published in Nature Methods.

Source:, Feb 2, 2015.

Unboiling egg whites, unscrambling proteins, keeping biotech liquid

Protein manufacturers have struggled to find efficient means of recycling valuable proteins. Such techniques could, for example, reduce costs for cancer treatments, food production, and other segments of the $160B global biotechnology industry. Researchers, at the University of California, Irvine, and South Australia’s Flinders University, report that they have figured out a way to unboil the egg white of a hen egg. The unlikely-sounding feat dramatically demonstrates a new protein-refolding process that works much faster than a conventional technique, a kind of molecular dialysis.

To re-create a clear protein known as lysozyme once an egg has been boiled, Dr. Weiss and his colleagues added a urea substance that chews away at the whites, liquefying the solid material. That’s half the process; at the molecular level, protein bits are still balled up into unusable masses. For the other half of the process, the scientists confined the tiny bits of protein to thin, microfluidic films and subjected them to sheer stress by means of a high-powered vortex fluid device. The protein bits were forced back into untangled, proper form. The new process was described in the journal ChemBioChem.

Source:, Jan 27, 2015.

mtDNA levels directly linked to frailty and mortality

Scientists from Johns Hopkins University report that the amount of mitochondrial DNA (mtDNA) found in peoples’ blood directly relates to how frail they are medically. This DNA may be a useful predictor of overall risk of frailty and death from any cause 10-15 years before symptoms appear.

The team says its findings contribute to the scientific understanding of aging and may lead to a test that could help identify at-risk individuals whose physical fitness can be improved with drugs or lifestyle changes. If low numbers of mitochondria are shown to cause or contribute to frailty, the team can use their simple test on lab-grown cells to assess the effectiveness of new drugs to increase the numbers of mitochondria. The study was published in the Journal of Molecular Medicine.

Source:, Dec 17, 2014.

Reproducibility of research remains problematic

A new report from Sigma-Aldrich exposes practices that compromise the reproducibility of published research and suggests strategies for improving reproducibility. The second annual State of Translational Research Survey Report is based on a survey commissioned by Sigma-Adrich and the Association for the Advancement of Science as well as the Academic Drug Discovery Consortium.

The survey revealed that only 22% of respondents had complete success in the last year reproducing other labs’ published work. Respondents cited the primary suspected causes of experimental irreproducibility to be poor controls, the rush to publish, and insufficient samples sizes. Half of respondents also blamed the reproducing lab’s failure to understand or follow experimental protocols. One of the take-away points was that several simple quality control practices could be performed more consistently, including testing for mycoplasma, validating reagents for purity and identity, and screening for misidentified cell lines.

Source:, Dec 11, 2014.

Published genomes contain sequences from contaminating organisms

According to scientists at Johns Hopkins University, a public database, GenBank, contains draft assemblies of animal and plant genomes that have been contaminated by bacterial and viral sequences. In particular, a draft assembly of domestic cow, Bos taurus, contained 173 small contigs that appeared to derive from microbial contaminants. Surprised by this finding, the scientists delved deeper and discovered the presence of cow and sheep DNA in the supposedly finished genome of a pathogenic bacterium, Neisseria gonorrhoea. The researchers assert that their findings illustrate the need to carefully validate findings of anomalous DNA that rely on comparisons to either draft or finished genomes. They are particularly concerned that anomalous DNA could cause problems for the rapidly growing field of microbiome analysis. Findings were published in the journal PeerJ.

Source:, Nov 25, 2014.

Schizophrenia linked to excess protein during brain development

Researchers at Rutgers report that a gene associated with schizophrenia plays a role in brain development and may help to explain the biological process of the disease. B. Firestein, Ph.D., professor in the department of cell biology and neuroscience, says too much protein expressed by the NOS1AP gene, which has been associated with schizophrenia, causes abnormalities in brain structure and faulty connections between nerve cells that prevent them from communicating properly.

The research indicates that an over-abundance of a protein in the NOS1AP gene resulted in the dendrites being stunted in the developing brains of rats. She and her colleagues found that too much of the NOS1AP protein in brain cells didn’t allow them to branch out and kept them deep within the neocortex, the portion of the brain responsible for higher functioning skills, such as spatial reasoning, conscious thought, motor commands, language development, and sensory perception. In the control group of rats in which NOS1AP chemical protein was not overexpressed, the cellular connections developed properly, with cells moving out to the outer layers of the neocortex and enabling the nerve cells to communicate. The study was published in Biological Psychiatry.

Source:, Nov 24, 2014.


Stem Cell News

AstraZeneca and Harvard partner on stem cell therapies for diabetes

AstraZeneca entered into a 5 year research collaboration with the Harvard Stem Cell Institute (HSCI) to adapt a technique that creates human beta cells from stem cells for use in screens of AstraZeneca’s compound library in the search for new treatments for diabetes. The collaboration also aims to better understand how the function of beta cells declines in diabetes. Research findings will be made available to the broader scientific community through peer-reviewed publications.

AstraZeneca will provide funding for a team of investigators at HSCI as well as establishing an in-house team in Mölndal, Sweden, dedicated to the collaboration. Scientists from each organization will work together to understand the biology behind the loss of human beta cell function and mass in diabetes, and to screen compounds against the cells produced to search for potential new medicines that could restore beta cell activity in diabetic patients.

Source:, March 25, 2015.

First stem-cell therapy in Europe

Chiesi’s Holoclar (an ex vivo expanded autologous human corneal epithelial cells containing stem cells), has been approved for moderate to severe limbal stem cell deficiency due to physical or chemical burns to the eye(s) in adults.

Source: EuropaBio Newsletter, Feb 27, 2015.

Cyntata validates stem cell manufacturing process at Wisconsin biomanufacturing site

Australian stem cell and regenerative medicine company Cynata Therapeutics is now set to scale up manufacturing of its mesenchymal stem cells (MSCs) for therapeutic use. The company´s novel stem cell manufacturing process known as Cumerus has been successfully validated at Waisman Biomanufacturing´s Madison, Winsonsin facility after trials confirmed the process is capable of producing MSCs for therapeutic application, consistently, efficiently and economically in a GMP production environment.

Source:, Feb 24 2015.

Stem cells from teeth may restore sight to the blind

Scientists at the University of Pittsburgh working with mice showed how stem cells harvested from teeth extracted during routine dental procedures can potentially be used to restore sight in those suffering from corneal blindness.

The team decided to focus on adult dental pulp stem cells (DSC) as a possible solution. They began by collecting DSC from molar teeth discarded after routine extractions at the university’s dental school and then treated the cells in a special solution that caused them to differentiate into corneal cells (nematocysts). When they tested the DSC-generated nematocysts they found they had the same properties as those grown naturally in the human eye. Then they seeded the cells onto a corneal-shaped magnifier substrate to see if they could engineer corneal tissue. Four weeks later, the cells had grown into a structure that mimicked the complex organization of an actual cornea. To evaluate how the DSC-generated nematocysts would perform, they labelled them with a dye and then injected them into the right eyes of mice (left eye was injected with medium, as control). When they tested the mice’s eyes five weeks later, the DSC-generated nematocysts had remained in the corneas and behaved similar to natural nematocysts. Their corneas were clear, without signs of rejection. The study was published in Stem Cells Translational Medicine.

Source:, Feb 23, 2014.

Exit from dormancy provokes DNA-damage-induced attrition in haematopoietic stem cells

Tissue-specific adult stem cells, including hematopoietic stem cells, normally exist in a relatively inactive state where they divide infrequently and have low energy burdens on the body. “Our theory was that this state of dormancy protected hematopoietic stem cells from DNA damage and therefore protects them from premature aging”, said M. Milsom, Ph.D., from the Heidelberg Institute for Stem Cell Research and Experimental Medicine.

Under conditions of stress, such as infection or chronic blood loss, hematopoietic stem cells are forced into action, which leads to rapid cell division in order to produce new blood cells and repair damaged tissue. The investigators showed that in such times of stress, stem cells produced elevated levels of reactive metabolites that have been reported previously to directly damage DNA. While DNA repair mechanisms are present in stem cells, the team hypothesize that continued exposure to stress increases the potential to introduce mutations due to the repair mechanisms being unable to keep up with the demand to rectify the damage. This inefficient repair would ultimately lead to cell death or the development of conditions (e.g. leukaemia).

The scientists also studied how the stress response impacted mice with the premature aging disorder, known to be associated with defective DNA repair, called Fanconi anaemia. Patients with this disorder suffer major cardiovascular complications and are at a very high risk for developing cancer. Mouse models of Fanconi anaemia have the same DNA repair defect as human patients, but the mice never spontaneously develop the bone marrow failure observed in nearly all patients. When Fanconi anaemia mice were exposed to scenario that mimicked a prolonged viral infection, they were unable to efficiently repair the resulting DNA damage, leading to stem cell failure. Normal mice showed a gradual decline in hematopoietic stem cell numbers, while the stem cells in the Fanconi anaemia mice were almost completely depleted, which ultimately led to bone marrow failure and insufficient production of blood cells to sustain life. The research was published in Nature.

Source:, Feb 20, 2014.

On-off switch for critical stem cell gene discovered

University of Toronto researchers investigating stem cells in mice report for the first time an instance of a relationship between the Sox2 gene, which is critical for early development, and a region elsewhere on the genome that effectively regulates its activity. The discovery could mean a significant advance in human regenerative medicine, as the Sox2 gene is essential for maintaining embryonic stem cells that can develop into any cell type of a mature animal.

“We studied how the Sox2 gene is turned on in mice, and found the region of the genome that is needed to turn the gene on in embryonic stem cells,” said Jennifer Mitchell, Ph.D., of U of T’s department of cell and systems biology. The study was published in Genes & Development.

Source:, Dec 15, 2014.

Re-do of stem cell “shock induction” fails

A purported stem cell advance that generated both excitement and scepticism upon its announcement nearly a year ago and gave rise to nagging questions, retractions of published articles, findings of scientific misconduct, and even a suicide in the months that followed has now suffered what may amount to its ultimate debunking. The would-be breakthrough, called stimulus-triggered acquisition of pluripotency (STAP) cells, prompted a five-month-long replication attempt that failed, announced officials at the RIKEN Center for Developmental Biology in Kobe, Japan.

RIKEN scientist Haruko Obokata, who led the initial STAP studies, was among those attempting to replicate the work she and her colleagues described in a pair of articles that appeared in Nature. Obokata, professing confusion and dismay at having failed to repeat the STAP phenomenon, announced her resignation through a statement presented by RIKEN officials.

Source:, Dec 19, 2014.

European court opens door for stem cell patenting

The European Court of Justice ruled that human embryonic stem cell patents could be allowed if organisms can’t develop into human being. The decision overturns a 2011 European Union ruling that outlawed patents on hESC research, affirming that pluripotent human stem cells derived from parthoenogenetically activated oocytes can be patented in Europe.

Parthenogenesis refers to the initiation of embryogenesis without fertilization by oocyte activation in the absence of sperm though a variety of chemical and electrical techniques. The activated oocyte contains a single or double set of maternally derived chromosomes but does not contain paternal DNA.

While patents on hESC have been upheld in the United States, the European Patent Office has refused to grant many of the same patents on ethical grounds if the commercial exploitation of those patents goes against public order or morality. Combined with a principle of non-commercialization of the human embryo, human body, and its products led Europe to deny the patentability of hESC.

Source:, Dec 18, 2014

New pluripotent mouse stem cell type

An international team of scientists say they have produced a new class of mouse stem cells called F-class cells. Their result came after looking at different response outcomes of somatic reprogramming by fully characterizing reprogrammed cells of pluripotent stem cell states.

“We demonstrate that by maintaining elevated reprogramming factor expression levels, mouse embryonic fibroblasts go through unique epigenetic modifications to arrive at a stable, Nanog-positive, alternative pluripotent state. In doing so, we prove that the pluripotent spectrum can encompass multiple, unique cell states.”

The scientists believe their novel reprogramming ability can eventually be used to develop artificial cells for applications in regenerative medicine and drug discovery. The frequency at which F-class cells arise in transposon-based reprogramming, in combination with their advantageous properties, presents the opportunity to study and utilize a novel pluripotent cell type in biology, medical research and future medicine. The study was published in Nature.

Source:, Dec 11, 2014.

$100M gift launches Allen Institute for cell science

With a gift of $100M, philanthropist/Microsoft co-founder Paul G. Allen launched a new research institute to investigate and model the behaviour and complex living machinery of human cells. The Allen Institute for Cell Science says its inaugural project will be creating the Allen Cell Observatory. Researchers plan to produce a dynamic, visual database and animated models of cell parts in action, with the goal of enabling predictions about cell behaviours, thus accelerating cell biology knowledge as well as disease research.

The cell science institute will begin by studying the transition of induced pluripotent stem cells into heart muscle and epithelial cells, creating computational models of the cells’ behaviour as the first part of the larger visual database.

Source:, Dec 8, 2014.



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