ACTIP bulletin no. 69

ACTIP bulletin no. 69, May 2014

In this issue

Seminars, Courses and Awards
Biomanufacturing News
Vaccine News
Business News
News from the Commission
Regulatory News
Clinical Trial News
Research News
Stem Cell News
Agenda
Webinars

 

Seminars, Courses and Awards

ESACT animal cell technology course, 4th edition

28 Sept – 2 Oct 2014, 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.
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:  www.esact.org 

Cells for compound screening, 1st edition

October 2-5, 2014, 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 cell production and use for compound screening, establishment, criteria as well as different formats and use of physiological relevant cellular assays with good reproducibility.
The aim of the course is to train interested individuals from Industry and Academia.
For more information: www.esact.org

Italian researcher wins American stem cell award

Elena Cattaneo, a leading stem cell researcher in Italy, has been selected 2013 Stem Cell Person of the Year by the Knoepfler Lab at University of California, Davis. Cattaneo studies neural stem cells to help understand the causes of Huntington’s Disease.
Source: EuropaBio Newsletter Nov 4-8, 2013.

Mary Ann Liebert wins stem cell education award

Mary Ann Liebert, president and CEO of Mary Ann Liebert Inc., and publisher of GEN, was presented with the Stem Cell Education Award by the Genetics Policy Institute. The award was given at the World Stem Cell Summit held in San Diego. Liebert was cited for her outstanding “work in educating patients, researchers, and the broader stem cell community, and in raising the standard in medical research journalism.
Source: www.genengnews.com/gen-news-highlights, Dec 6, 2013.

Biomanufacturing News

Roche to invest 190M CHF in new production facility in Basel

Roche announced plans to invest 190M CHF in the construction of a new production facility in Basel, which will support the manufacturing of antibody-drug conjugate medicines. This development is part of an 800 M CHF investment by Roche to increase production capabilities for its biologic medicines across its global manufacturing network.
Source: Roche Media Release, Oct 14, 2013.

Genzyme to build $80M downstream plant in Framingham, MA

Sanofi-owned Genzyme will build a new $80M downstream processing facility for its Fabry disease treatment adjacent to its new Fabrazyme® (agalsidase beta) cell culture manufacturing site in Framingham, MA.
Downstream processing involves purification of material harvested from cell culture manufacturing. The final product, administered intravenously, is formulated and fill-finished in a separate facility in Waterford, Ireland, and shipped to multiple distribution centres for labelling, packaging, and shipping worldwide.
Source: www.genengnews.com/gen-news-highlights, Oct 15, 2013.

Advances in single-use bioreactor technology

Sartorius hosted a workshop focusing on advances in single-use bioreactor technology on June 23, 2013 in Lille, France. The attendees representing users from different biopharmaceutical companies incl. Novasep, Lonza and DSM shared their experience with implementing single-use bioreactors for conventional and high-cell-density concentrated fed-batch processes and for production of stem cells as advanced medicinal therapy products.
Source: Sartorius newsletter, Oct 30, 2013.

Largest biologics plant in world set as Roche U-turns over biosimilar fizzle

Genentech has recommissioned plans to make a Californian facility the largest biologics plant worldwide after anticipated revenue drop from biosimilars did not materialize. The subsidiary of Roche is set to invest 140M CHF into its cell culture facility in Vacaville pushing total capacity to 240.000 Liters. Engineering lots are anticipated to begin in June 2014, with full licensing in the 1st quarter of 2016.
Source: www.biopharma-reporter.com/, Nov 13, 2013.

GE launches cell expansion technology targeting cell therapy firms

GE Healthcare has launched a cell expansion technology designed to win business from cell therapy developers. “Cell therapies” include treatments in which cells are implanted into a patient to replace damage tissue.
Cells are first extracted from a healthy donor or the patient, multiplied to the point where they have therapeutic effects and finally re-implanted. At present there are over 2,500 clinical trials of cell therapies worldwide (clinicaltrails.gov), which have created market opportunity of which GE is keen to capture a share.
Source:: www.biopharma-reporter.com, Nov 15, 2013.

GE launches new programmable bioreactor for seed chain and process development

GE Healthcare has launched new single-use, bioreactor for seed train and process development operations and mAb production. The new system called Wave 25 is designed for seed train, process development and small scale bio-manufacturing operations.
Source: www.biopharma-reporter.com, Nov 22, 2013.

Cellexus claims new bioreactor better at mixing than rocking systems

Cellexus has launched a new bioreactor system known as CellMaker Plus that employs an air-based mixing system that is better at aerating cell cultures than rocking or large chamber systems. The new system is designed for the growth of bacteria and yeast cultures at high optical densities including oxygen “hungry” organisms knife E. Coli, which are commonly used in the manufacturing of biopharmaceuticals.
Source: http://www.biopharma-reporter.com, Dec 6, 2013.

AstraZeneca licenses mAb production tech from Lonza and BioWa

AstraZeneca’s biologics division MedImmune has licensed a cell line and antibody production tech from Lonza and BioWa. The agreement enables MedImmune to use the Potelligent CHOK1SV cell line for multiple therapeutic antibody drug candidates it is developing.
Source: www.biopharma-reporter.com, Dec 11, 2013.

Man-made tracheas move into clinical trial manufacturing phase

Synthetically engineered tracheas may be marketed sooner than previously thought as a Harvard Bioscience spinoff prepares to produce the “scaffolds” for growing tracheas in more clinical trials. Harvard Apparatus Regenerative Technology is making the bioreactors and plastic-based scaffolds used to create the artificial tracheas for the clinical trials in the EU after receiving a $5M grant.
Source: www.biopharma-reporter.com, Jan 22, 2014.

DECHEMA report on single-use technology now available

DECHEMA has published a status report on single-use technology in biopharmaceutical production. The report gives an overview of applications, potential and limitations of single-use technology in biopharmaceutical production. Manufacturers, developers, available products and current research activities in this field are analysed.
Source: http://biotech.dechema.de/Fachgemeinschaft+Biotechnologie/Gremien+der+Fachgemeinschaft/Single_Use_Technologie.html, March 12, 2014.

UK slots over $90M for large-scale cell therapy manufacturing centre

The U.K. government will allot £55 million (around $90.7 million) from its 2014/2015 budget for a new Cell Therapy Manufacturing Centre. The Cell Therapy Catapult, a translational centre of excellence, will manage the new Centre, which is scheduled to open during 2016/17.
The Cell Therapy Manufacturing Centre will be designed to provide the U.K. with the manufacturing facilities needed for later studies and commercialization, promoting retention of domestic expertise and jobs. In addition, many global cell therapy organizations with which the CT Catapult has engaged believe that a high-quality EU manufacturing base is essential for bringing their products to the European market, and this facility should also help to anchor their activities in the U.K.
Source: www.genengnews.com/gen-news-highlights, Mar 20, 2014.
GE to in-license Promosome mammalian cell lines
GE Healthcare Life Sciences has signed a licensing agreement for Promosome’s suite of mammalian cell line development technologies, which aim to increase protein expression in mammalian cell culture. Under the term of the agreement, Protesome has granted GE exclusive rights for the technologies and will receive milestone payments for technology transfer and subsequent royalties upon commercialization. Promosome technology suite, which is comprised of three complementary elements for optimizing protein translation, has been developed with the Scripps Research Institute.
Source: www.biopharma-reporter.com, April 30, 2014.

UCB to halve antibody discovery time with robot

UCB has launched a robot it says can halve antibody discovery time, increase capacity and improve quality and consistency of antibodies. The automated discovery process, named Technology Platform Access, opened last month in Slough, UK as part of $ 5.5m laboratory investment.
The platform screens B lymphocytes, to find the rare ones that produce the right antibodies for use in biopharmaceuticals. The platform joins together three robots: a work cell which set up cultures, a screening cell and a hit-picking cell which selects the successful B lymphocytes. It will be available for collaborative projects and for in-house research.
Source: www.biopharma-reporter.com, May 9, 2014.

Teva says no to future JVs with Lonza, but yes to biosimilar growth

UCB has launched a robot it says can halve Teva will not form another joint venture with Lonza but will look to grow its biosimilars pipeline. However, the Israeli generics firm says that “as we go forward will include Lonza” and that it has “deep respect for” Lonza.
The company says Teva was also making “significant efforts” to develop biosimilars for reference biologics whose patents end after 2020, although it acknowledged the work had some way to go. For biological going off-patent between 2015 and 2020 such as Rituxan and AbbVie’s Humira, the company plans to “do in-house development as well as partner with firms who might have products appropriate for their pipeline and focuses diseases”.
Source: www.biopharma-reporter.com, May 9, 2014.

 

Vaccine News

Nanoparticles show promise for vaccine delivery to lungs

Researchers at MIT developed a novel type of nanoparticle that protects a vaccine long enough to generate a strong immune response both in the lungs and in mucosal surfaces far from the vaccination site (e.g. gastrointestinal and reproductive tracts).
The protein fragments of the vaccine are encased in a sphere made of several layers of lipids that are chemically stapled to one another, making the particles more durable inside the body. This allows the particles to resist disintegration once they reach the lungs. With this packaging, the protein vaccine remains in the lungs long enough for immune cells lining the surface of the lungs to grab them and deliver them to T cells. Activating T cells is a critical step for the immune system to form a memory of the vaccine particles so it will be primed to respond again during an infection. In mice, HIV or cancer antigens encapsulated in nanoparticles were taken up by immune cells much more successfully than vaccine delivered to the lungs or under the skin without being trapped in nanoparticles. The study was published in Science Translational Medicine.
Source: www.genengnews.com/gen-news-highlights, Sep 26, 2013.

PsiOxus Therapeutics wins EuropaBio Most Innovative European Biotech SME award

PsiOxus Therapeutics won the esteemed award now in its 4th year due to their radical research and innovative breakthroughs in meeting societal grand challenges in the field of healthcare. The company is committed to developing a new approach to cancer therapy, by using oncolytic vaccines that are potent killers of cancer cells, but with minimal effects on normal healthy cells.
Source: www.europabio.org, Oct 2, 2013.

Roche cancer vaccine pact could net immatics $1B-plus

Roche will oversee clinical development and commercialization of all immunotherapies generated by Immatics biotechnologies under a cancer vaccine and immunotherapy collaboration that could net the German clinical-stage biopharma more than $1B.
The collaboration will focus on research, clinical development, and commercialization of a number of new tumour-associated peptide (TUMAP)-based cancer vaccine candidates and other immunotherapies in oncology, targeting primarily gastric, prostate, and non-small cell lung cancer.
Source: www.genengnews.com/gen-news-highlights, Nov 13, 2013.

100 Million reasons to love vaccines

Vaccines have prevented more than 100 million serious cases of contagious diseases in the US since 1924. Scientists from the University of Pittsburgh looked at the number of reported cases of polio, measles, rubella, mumps, hepatitis A, diphtheria and whooping cough before and after vaccines were available. The projections are based on how many cases would have occurred if a vaccine had not been developed. The research was published in the New England Journal of Medicine. It showed how important vaccines have been in a historical context and why they still matter today.
Source: www.humanosphere.org, Dec 2, 2013.

TB vaccine may prevent MS, if given early

Bacille Calmette-Guérin (BCG), a live vaccine used to prevent tuberculosis, may prevent multiple sclerosis (MS) if given early at the disease’s first sign, a neurological episode called clinically isolated syndrome.
In a double-blind study that included 73 people who evidenced clinically isolated syndrome, 33 were vaccinated with BCG, and 40 received a placebo. All of the participants had brain scans once a month for six months. They then received the MS drug interferon β-1a for a year. After that, they took the MS drug recommended by their neurologist. The development of definite MS was evaluated for five years after the start of the study. Results showed that 58% of the vaccinated people had not developed MS, compared to 30% of those who received the placebo. No major side effects were detected during the study, and there were no differences in side effects between those who received the vaccine and those who didn’t. The study was published in Neurology
Source: www.genengnews.com/gen-news-highlights, Dec 5, 2013.

HPV vaccine successes

The HPV vaccine prevents the spread of the cancer-causing human papillomavirus. But because HPV is transmitted through sexual contact, some critics say the vaccine promotes promiscuity, others fret about potential side effects. As a result, only a third of teenage girls in the U.S. have received the three recommended shots.
A nationwide survey revealed that infection rates from cancer-causing HPV strains have sharply declined since the vaccine was introduced in 2006. Over 8,000 girls and women between the ages of 14 and 59 were evaluated, which found that infection rates with the strains of HPV that cause cancer had fallen 56 percent for 14 to 19 year-old girls. Among adolescent girls, infection rates fell from 11.5 percent of sexually active girls in 2006 to 5.1 percent in 2013 according to the Centers for Disease Control and Prevention. By increasing three-dose coverage to 80 percent, 53,000 more cases of cervical cancer could be prevented. Since HPV has been linked to anal, penile and some throat cancers in men, experts recommend that boys also get vaccinated.
Source: EuropaBio Newsletter, Jan20-24, 2014.

Novel “vaccine” delivery method shuts the door on HIV

Scientists at Caltech have demonstrated that a technique they developed to protect mice from a lab strain of HIV delivered intravenously also works against the real-world form of HIV usually transmitted across mucosal surfaces. The new delivery method Vectored Immuno Prophylaxis (VIP), injects a small harmless virus and delivers genes to the muscle tissue, instructing it to generate specific antibodies.
According to the investigators VIP is capable of protecting humanized mice from intravenous as well as vaginal challenge with diverse HIV strains despite repeated exposures. Animals receiving VIP that expresses a modified VRC07 antibody were completely resistant to repetitive intravaginal challenge by a heterosexually transmitted founder HIV strain, suggesting that VIP may be effective in preventing vaginal transmission of HIV between humans. The study was published in Nature Medicine.
Source: www.genengnews.com/gen-news-highlights, Feb 12, 2014.

New hope for better vaccines against meningitis and pneumonia

A scientific team reports the discovery of the presence of a new subtype of innate lymphoid cells in human spleen essential for the production of antibodies. This discovery clears the path for the identification of novel strategies to develop more efficient vaccines against encapsulated bacteria.
The work was done by the B-cell biology research group at the Institut Hospital del Mar d’Investigacions Mediques in Barcelona. Among the collaborators of the group are researchers from Icahn School of Medicine at Mount Sinai in New York and Riken Research Centre for Integrative Medicine in Japan. The research involved in vitro studies with isolated cells from human spleen samples and in vivo studies performed with different mouse models. The work explored the function of the innate lymphoid cells in homeostasis.
The investigators identified retinoic acid receptor-related orphan nuclear receptor γt + innate lymphoid cells (ILCs) near the marginal zone (MZ), a splenic compartment that contains innate-like B cells highly responsive to circulating T cell independent (TI) antigens. Splenic ILCs established bidirectional crosstalk with MAdCAM-1+ marginal reticular cells by providing tumour-necrosis factor and lymphotoxin, and they stimulated MZ B cells via B cell–activation factor, the ligand of the costimulatory receptor CD40 and the Notch ligand Delta-like 1. The splenic ILCs further helped MZ B cells and their plasma-cell progeny by co-opting neutrophils through release of the cytokine GM-CSF. Consequently, depletion of ILCs impaired both pre- and post-immune TI antibody responses. Thus, ILCs integrate stromal and myeloid signals to orchestrate innate-like antibody production at the interface between the immune system and circulatory system.” The work was published in Nature Immunology.
Source: www.genengnews.com/gen-news-highlights, Feb 24, 2014.

NIAID grant will fund HIV vaccine research

Four Seattle institutions and The Rockefeller University were awarded a 7-year $9.8M grant from NIH’s National Institute of Allergy and Infectious Diseases (NIAID) toward developing a vaccine designed to elicit broadly neutralizing antibodies against HIV-1.
The consortium will use the grant to fund the initial phase of the project, which will include the optimization and preclinical evaluation of two vaccine candidates. The collaboration aims to address two longstanding frustrations researchers have in the challenge of creating an HIV vaccine. One is how to elicit broadly neutralizing antibodies against the virus a task that requires stimulating B cells that on their surface express B-cell receptors, each cell expressing a specific receptor. The other, is learning what titers you have to make of the antibodies in order for them to be protective, or how to make them last long enough to be truly effective against the virus.
Source: www.genengnews.com/gen-news-highlights, Mar 10, 2014.

EU awards CureVac for RT stable MRNA vaccine development

CureVac has been awarded a € 2M grant by the EC for the development of room temperature (RT)-stable mRNA-based vaccines. According to the EC, the vaccines could overcome one of the biggest barriers to disease prevention in developing countries. The instability of single stranded RNA is mainly caused by the presence of RNAses. CureVac´s proprietary method and RNAse-free production environment ensures stability of its RNA vaccines. In addition the lack of need for cell cultures and hen´s egg-based methods was key to winning the award.
Source: www.biopharma-reporter.com, Mar 11, 2014.

Sanofi and SK to co-develop pneumococcal conjugate vaccine

Sanofi Pasteur, the vaccines division of Sanofi, said it will partner with SK Chemical to co-develop a pneumococcal conjugate vaccine (PCV), in a deal expected to net the South Korean company at least $23 million and make use of the Asian nation’s first cell-culture based manufacturing plant. If the vaccine proves successful, SK Chemical would produce the vaccine at its cell-culture based GMP production facility in Andong, with Sanofi Pasteur launching the product for commercial sale. Both companies agreed to share profits outside of Korea, where SK would hold exclusive rights to commercialize the vaccine.
Source: www.genengnews.com/gen-news-highlights, Mar 19, 2014.

Texas biomanufacturing centre to produce vaccines against bioterror threats

Armed with a $285m investment from the US Department of Health and Human Services, the Texas A&M Center of Innovation in Advanced Development and Manufacturing (CIADM) is now using advanced manufacturing techniques to grow the government’s stockpile of vaccines to fight bioterror threats. As a public-private partnership, the centre also leverages the expertise of Texas A&M University, small biotech firms and the development and manufacturing capabilities of large pharma companies.
The grant is part of three investments totalling $593 m which includes Novartis and Emerging Manufacturing Operations in Baltimore of $86m and $221m respectively. The three participants are required to establish surge capacity to quickly manufacture influenza vaccines in a pandemic and secure a pandemic influenza vaccine candidate currently under development.
Source: www.biopharma-reporter.com, May 9, 2014.

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

Takeda has received a $70m subsidy from the Japanese government to expand production capacity of its cell-culture pandemic influenza vaccine. Under the agreement, Takeda will improve its vaccine production facilities to the point where it can supply the government with an extra 8m people’s doses in the event of a flu pandemic.
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: www.biopharma-reporter.com, May 9, 2014.

 

Business News

Santorius to acquire TAP Biosystems

Sartorius Stedim Biotech to acquire TAP Biosystems Group and to expand the buyer’s portfolio of single-use fermenters. The deal is to be closed at year’s end subject to customary closing conditions including the approval of TAP Biosystems’ shareholders. The boards of both companies have unanimously approved the transaction.
In November 2013, UK-based fermentor maker TAP Biosystems has approved the $45M takeover bid by Sartorius to expand its small-scale cell culture tech offering.
Sources: www.genengnews.com/gen-news-highlights, Oct 7, 2013; and www.biopharma-reporter.com, Nov 22, 2013.

BioInvent and Bayer extend and broaden collaboration for the discovery and development of therapeutic antibodies

BioInvent International AB announced it has signed an extension to its 2008 license agreement with Bayer Pharma AG for the development of antibodies from BioInvent’s n-CoDeR® libraries. Under the terms of the extension, Bayer will broaden its access to BioInvent’s discovery and development technology platform.
BioInvent will receive an undisclosed license fee to cover new projects plus success-based milestone payments and royalties on products already in development. The n-CoDeR® libraries contain more than 20 billion highly diverse, fully human antibody fragments that have been created using BioInvent’s patented approach, generating antibodies with high affinity and selectivity.
Source: BioInvent, Oct 14, 2013.

Roche to invest 800M CHF in global biologic medicine manufacturing network

Roche announced it plans to invest 800M CHF within its global manufacturing network to increase production capabilities for its biologic medicines over the next five years. The investment will be spread across sites in Penzberg, Germany, Basel, Switzerland, as well as Vacaville and Oceanside, USA. Approximately 500 new jobs are expected to be created in conjunction with the facility expansions.
Source: Roche Media Release, Oct 14, 2013.

BioInvent granted US patent for the F.I.R.S.T.™ antibody screening technology

BioInvent International AB announced that it has been granted a US patent for its antibody and concomitant target discovery technology F.I.R.S.T.™. Traditional drug discovery methods involve the identification of drugs candidates that bind to known disease targets. The F.I.R.S.T.™ technology is a novel and unique discovery method, which allows for antibodies and disease targets to be identified simultaneously. BioInvent has used F.I.R.S.T.™ successfully in several proprietary discovery projects and offers F.I.R.S.T.™ as well as traditional antibody discovery screening to commercial partners.
Source: BioInvent, Oct 15, 2013

Roche and Polyphor join efforts to combat multi-drug-resistant bacterial infections

Roche and Polyphor Ltd. announced that they have entered into an exclusive worldwide license agreement to develop and commercialise Polyphor’s investigational macrocycle antibiotic, POL7080, for patients suffering from bacterial infections caused by Pseudomonas aeruginosa. POL7080 belongs to a new class of antibiotics that has demonstrated clinical safety and tolerability in a Phase I clinical trial and kills Pseudomonas aeruginosa by a novel mode of action.
Antimicrobial resistance represents a major threat to public health worldwide, leading to 25,000 deaths and related costs of over €1.5B in healthcare expenses and productivity losses in the EU alone each year. Pseudomonas aeruginosa accounts for 1 in every 10 hospital-acquired infections in the US and is listed as one of the six most dangerous drug-resistant microbes.
Source: Roche Group Media Relations, Nov 4, 2013.

Patheon to go private, combine with DSM Pharma in $195M Deal

Patheon would be taken private under a deal that would entail the company being acquired by Royal DSM’s business group, combined with the buyer’s pharmaceutical products operations, then spun off into a new joint venture designed to create a powerhouse contract development and manufacturing organization (CDMO). The new CDMO provisionally named NewCo, with a permanent name to be announced in coming months would offer an array of services from finished dosages for drugs to manufacturing of APIs.
Source: www.genengnews.com/gen-news-highlights, Nov 19, 2013.

Roche and Molecular Partners launch $1B+ cancer drug alliance

Roche and Molecular Partners entered into a potentially 1$B collaboration giving Roche rights to develop a new class of cancer treatments that combine its toxic agents with Molecular Partners’ DARPin® biologics.
DARPins are non-antibody-based small proteins where a variable region has been engineered for target binding. DARPins are ideal targeting agents to deliver toxic agents to tumours to kill cancer cells based on their small size and high binding affinity, which enable them to hone in on and penetrate deep into solid tumours. They also have a higher selectivity for tumour cells compared to other biologics (e.g. antibody drug conjugates), based on their ability to bind to different and multiple epitopes in parallel.
Source: www.genengnews.com/gen-news-highlights, Dec 4, 2013.

GE Healthcare Acquiring Thermo Businesses

GE Healthcare said it agreed to acquire Thermo Fisher Scientific’s cell culture media and sera, gene modulation technologies, and magnetic beads businesses, all for a combined $1.06B. Thermo agreed to sell the businesses in November as a condition of winning EC approval to acquire Life Technologies for $13.6B, plus assumption of $2.2B in the acquired company’s debt. The EC contended that keeping the businesses in Thermo’s hands would have significantly reduced competition.
Source: www.genengnews.com/gen-news-highlights, Jan 6, 2014.

Sigma-Aldrich launches ‘knock-out’ way to create liver toxicity assays

Sigma-Aldrich has teamed with Biopredic to produce genetically modified human liver cell lines and assays that could cut costs by reducing animal toxicology studies in drug discovery. Sigma-Aldrich intends to use its Zinc Finger Nuclease gene editing technology to create a range of gene knock-out and reporter lines from the HepRG human liver cell lines, licensed from cell supplier Biopredic International.
Source: www.outsourcing-pharma.com, Jan 21, 2014.

Thermo takes on a new Life

Thermo Fisher Scientific said it closed on its acquisition of Life Technologies for $13.6B, plus assumption of the acquired company’s $1.5B in debt a blockbuster deal that creates a powerhouse in biopharma lab instrumentation and supplies with revenues of $17B and 50,000 employees in 50 countries.
That workforce is expected to shrink over three years, however, with Thermo restating last week it was committed to cutting a total $275M in costs over that period. Thermo said it will achieve most of that reduction, or $250M, by combining global infrastructure with Life Tech, and the remaining $25M by combining commercial capabilities.
Source: www.genengnews.com/gen-news-highlights, Feb 4, 2014.

U.S. global R&D share now trails Asia’s

The policy making body of the U.S. National Science Foundation reported that Asia’s largest economies led by China, Japan, and South Korea now perform a larger share of global R&D than the United States, whose high-tech manufacturing activity is now nearly matched by China’s.
The share of the world’s R&D performed in the U.S. has decreased since 2001, from 37 to 30%. In Western Europe it also fell from 26 to 22%. By contrast, the worldwide R&D share by Asia rose from 25 to 34% led by China, whose share nearly quadrupled from 4% to 15% in 2011, when it spent $208B.
Source: www.genengnews.com/gen-news-highlights, Feb 7, 2014.

Bayer clinches Algeta acquisition with $2.6B bid

Bayer said it will proceed with plans to buy Algeta after its $2.6 billion bid for shares in the Norwegian biotech surpassed expectations. The deal will unite the German pharma-and-chemical conglomerate with its partner in developing the recently-launched prostate cancer drug Xofigo.
Source: www.genengnews.com/gen-news-highlights, Feb 26, 2014.

AbbVie, Amgen and Roche most exposed to biosimilar competition

The biosimilar market is expected to ramp up in Asia and Europe, even as it’s yet to be initiated in the US, with AbbVie, Amgen and Roche’s blockbuster biologics the most threatened according to a Moody’s Investors Service new report.
The report “Biosimilars: Parsing the Industry´s pipelines” says biosimilars are at least three years away in the US, which is the world´s largest pharmaceutical market. While in Europe more than a dozen products are now commercialized, in the US no company has yet filed for approval of a biosimilar drug since the existence of the FDAs new pathway for biosimilars. The report includes a table listing biosimilar products currently in late stage development for the US market and the developer’s patent expiries.
Source: www.biopharma-reporter.com, 28 Feb, 2014.

DPx launched as global provider

DPx was launched as a leading global contract development and manufacturing organization (CDMO) for the pharmaceutical and related industries. Formerly known by its provisional name NewCo, DPx is the result of a USD $2.65 billion transaction between JLL Partners, a leading middle-market private equity firm, and Royal DSM, the global Life Sciences and Materials Sciences company. DPx represents the privately held parent company of the Patheon, Banner Life Sciences, and DSM Fine Chemicals businesses. With global headquarters in Durham, N.C., DPx has a footprint of 24 locations across North America, Europe, Latin America and Australia with more than 8,000 employees.
Source:www.fiercepharmamanufacturingcom/press-releases/dpx-launches-global-provider-pharmaceutical-services-fine-chemicals-and-pro, March 12, 2014.

Charles River buys Argenta and BioFocus

Charles River Laboratories said it will acquire the CRO services division of Galapagos including the Argenta and BioFocus businesses in a deal designed to strengthen the acquiring CRO at a time when more biopharmas are outsourcing their drug discovery and preclinical services. Charles River will pay Galapagos €129 million cash plus up to €5 million in payments based on future performance. The acquisition is expected to close early in the second quarter, subject to customary closing conditions.
Source: www.genengnews.com/gen-news-highlights, Mar 13, 2014.

Neostem to acquire CSC in potential $124M-plus stem cell deal

NeoStem said it will acquire California Stem Cell (CSC) in a stock-and-cash deal that could total more than $124 million and will add a late-stage technology to the acquiring company’s pipeline. Through the deal, NeoStem will take over development of CSC’s Melapuldencel-T, an autologous melanoma initiating (stem) cell immune-based therapy intended to eliminate the tumour cells capable of causing disease recurrence, beginning with the launch of a pivotal Phase III trial. NeoStem said Melapuldencel-T was a platform technology that could be expanded into other indications, such as hepatocellular carcinoma and other immune-responsive tumour types.
Source: www.genengnews.com/gen-news-highlights, Apr 14, 2014.

Novartis scoops GSK´s oncology portfolio and trades away its vaccine division

Novartis has agreed to acquire GlaxoSmithKline’s oncology pipeline for as much as $16B, while GSK will acquire Novartis’ vaccine business, excluding the flu vaccines, for as much as $7B. In a separate transaction, Novartis also agreed to divest its animal health division to Eli Lilly for approx. $11B. The transactions are expected to be completed by the first half of 2015 and are subject to standard regulatory and shareholders’ approvals.
Source: www.in-pharmatechnologist.com, Apr 23, 2014.

AstraZeneca refuses $100B bid, but Pfizer confident of compelling offer

AstraZeneca says Pfizer has “very significantly undervalued” the company after confirming rumours it had received an offer worth £59bn ($100bn). Pfizer made an offer to AstraZeneca in January 2014. Of $86.62 per share, formed 30% by cash and 70% by shares, the suggested $100B purchase would have combined the pharma giants into a new listed company with management in the US and UK.
Source: www.in-pharmatechnologist.com, April 28, 2014.

Bayer buys Merck & Co´s OTC business for $12.2B

Bayer says it will become the Over-The-Counter (OTC) leader in North America having acquired Merck & Co.’s consumer health business for $14.2bn (€10.2bn). The addition of key Merck brands will almost double sales of OTCs in North America, to $2.2B, making Bayer key leader in this geography as well as the number two leader in Europe.
Source: www.in-pharmatechnologist.com, May 6, 2014.

Huntingdon buys Harlan to form 3rd largest preclinical CRO

Huntingdon Life Sciences has acquired contract research and animal models firm Harlan Laboratories, making it the third largest preclinical research organisation. The merger, of which financial details have not been revealed, will create a company with annual revenue of around $500m, according to Huntingdon’s managing director Andrew Gay.
The deal would bring Harlan’s complementary Research Model & Services (RMS) business to the new entity on top of increased contract research services. The deal will see Huntingdon’s five mostly European locations complemented by 33 global Harlan sites with a total of approximately 3,500 staff working for the new entity.
Source: www.biopharma-reporter.com, May 9, 2014.

 

News from the Commission

COSME: € 2.3B to foster the competitiveness of SMEs over the next 7 years

The EC welcomed the adoption of the Competitiveness of Small and Medium-sized Enterprises (COSME) programme by the European Parliament. COSME aims to ease the access to credit problems that small businesses currently face. With a € 2.3 billion-budget over 2014-2020 COSME will for instance provide a guarantee facility for loans to SMEs of up to €150 000.
Source: EuropaBio Newsletter, November 25-29, 2014.

Horizon 2020 launched with €15 billion over first two years

The EC has presented calls for projects under Horizon 2020, the European Union’s €80 billion research and innovation programme. Worth more than €15B over the first two years, the funding is intended to help boost Europe’s knowledge-driven economy and tackle issues that will make a difference in people’s lives.
Source: http://europa.eu/rapid/press-release_IP-13-1232_en.htm?locale=en, Dec 11, 2013.

Lower number of animals used in drug R&D in 2011 says new EC report

Fewer animals have been used to test the safety of life saving drugs in Europe in 2011 than in previous years according to the 6th edition of a research report by the EC. The new report shows that across the 27 EU members states 11.5M animals were used in 2011, which is half compared to the previous assessment in 2008. The document lists how many animals are used in each country and breaks down figures in specific applications including preclinical toxicology and pharmaceutical products.
Source: www.outsourcing-pharma.com/, Dec 16 2013.

EBE says EC must do more on biodrug assessments

In a report on the use of omics sciences to develop personalized drug, the EC concluded “while challenges remain legislation is flexible enough to address current needs and to authorise personalised medicines in a timely manner”. The European Biopharmaceutical Enterprises (EBE) including Amgen, Roche, Eli Lilly, Genzyme and others announced that it disagrees with this conclusion. The organisation suggests collaborating on quality standards for genomic data and greater use of fast track and adaptive evaluation models by regulators.
Source: www.biopharma-reporter.com, Dec 18, 2013.

EU updates GMP principles on qualification and validation

European drug manufacturers will have to control the critical aspects of their operations through qualification and validation over the lifecycle of the product and process, according to a new draft good manufacturing principles GMP guideline from the EC.
Source: www.in-pharmatechnologist.com, Feb 11, 2014.

European partnership wins $115M from IMI to ENABLE antibiotic R&D

A consortium of more than 30 European companies and universities will work to develop new antibiotics against gram-negative pathogens, through a six-year program called European Gram Negative Antibacterial Engine (ENABLE), to be funded with €85 million from the Innovative Medicines Initiative (IMI).
Through open calls, ENABLE aims to expand a preliminary portfolio of programs into a full development pipeline. ENABLE’s ultimate goal is to deliver at least one new antibacterial candidate against gram-negative infections into Phase I clinical trials by 2019.
Source: www.genengnews.com/gen-news-highlights, Feb 12, 2014.

EU will require publication of trial results

The European Parliament has passed a law requiring all biopharmas and academic researchers to make public all results of clinical trials on the continent. By a 594–17 vote, with 13 abstentions, members of Parliament approved the new EU Clinical Trials Regulation. It requires trial sponsors to post full reports, including those of failed trials, once a drug is approved for marketing by the EC.
The measure was revised from its original version by allowing companies to publish only detailed summaries of their trials in a public database until the drug under study is EC-approved. Also, biopharmas will be able under the new law to submit applications for trial approval once through a single EU portal, with member states having to approve a single decision and a fixed deadline for review. A final version of the regulation is expected to be published this summer, and take effect in 2016.
Source: www.genengnews.com/gen-news-highlights, Apr 4, 2014.

EC intends to improve regulation on advanced therapy medicinal products

The EC published a report on the application of the Regulation 1394/2007 on advanced therapy medicinal products (ATMPs). The report outlines the situation of ATMPs in the EU and assesses the impact of the Regulation on the development of advanced therapies. The EC acknowledges the current concerns and difficulties faced by ATMP developers, as was outlined in EuropaBio’s comments to the public consultation in early 2013. As only four ATMPs have been granted marketing authorizations since the application of the ATMP Regulation in December 2008, the Commission’s intention to improve various aspects of the ATMP Regulation is a positive sign towards the industry and patients.
Source: EuropaBio Newsletter, April 4-11, 2014.

 

Regulatory News

Is FDA sacrificing safety for speed? New study raises the question

A study published by JAMA Internal Medicine, raising the issue whether safety has been sacrificed by a provision of the FDA Amendments Act of 2007 (FDAAA) allowing expedited approvals of drugs deemed to offer significant therapeutic advancement for “serious or life-threatening conditions that would address an unmet medical need.
T.J. Moore of the Institute for Safe Medication Practices and C.D. Furberg, M.D., Ph.D., of the Wake Forest University School of Medicine, studied development times, clinical testing, post-market follow-up, and safety risks for the 20 drugs approved by the FDA in 2008.
Eight of the 20 drugs were approved via expedited review, the rest, under standard review. Expedited drugs took a median of 5.1 years of clinical development to win FDA marketing approval, compared with 7.5 years for drugs approved through standard review. Far fewer patients were tested for drug efficacy during expedited approvals a median of 104 patients, compared with a median 580 for standard-review drugs. Moreover many post-marketing studies requested by the FDA to gather additional evidence on the safety of expedited drugs have not yet been completed.
According to the authors, the shift has made it more difficult to balance the benefits and risks of new drugs, and further systematic assessment of the standards and procedures for testing new drugs is needed.
Source: www.genengnews.com/gen-news-highlights, Oct 29, 2013.

EMA approves first MAb Biosimilars

The European Medicine’s Agency (EMA) has approved the first monoclonal antibody (MAb) biosimilars (Remsima and Inflectra) and expects to approve increasingly complex biosimilars as the regulatory framework becomes more flexible.
Remsima and Inflectra contain a version of the MAb infliximab originally authorized in the biological product Remicade, a Johnson & Johnson and Merck & Co`s rheumatoid arthritis drug.
Source: www.biopharma-reporter.com, Oct 30, 2013.

CLL drug yields breakthrough approval for Genentech

The FDA approved its first “breakthrough” medicine Genentech’s Gazyva™, in combination with Chlorambucil chemotherapy for people with untreated chronic lymphocytic leukemia (CLL). The Roche subsidiary trumpeted the approval as FDA’s fifth for a new drug developed by the company in the past three years.
Genentech persuaded the FDA of the significance of positive progression-free survival results from the Phase III CLL11 trial. In CLL11, which studied a total 356 participants, patients treated with Gazyva™ in combination with chlorambucil chemotherapy had significantly (84%) reduced risk of disease progression or death compared with the chemotherapy alone. Patients who received Gazyva™ lived significantly longer without their disease getting worse (median of 23 months) as compared to those who received chlorambucil alone (11.1 months).
Source: www.genengnews.com/gen-news-highlights, Nov 1, 2013.

A problem shared: EMA and US FDA team on generics site inspections

The EMA and US FDA will conduct joint site inspections and share the results of assessments of bioequivalence data for generic drugs under an announced accord. The collaboration begins with an 18-month pilot phase and expands the good clinical practice (GCP) focused data sharing partnership.
Source: www.in-pharmatechnologist.com, Dec 19, 2013.

US biopharma regulators to get 2014 budget they wanted

The US regulatory scientists who oversee biopharmaceuticals appear to be getting the funding they asked for after politicians pass a $1.2TR (€882B) Government spending plan for fiscal 2014. The agreed fiscal 2014 budget provides the FDA $2.552B in funding, which is an increase of $91M on the previous financial year and is in line with the amount requested.
Source: www.biopharma-reporter.com, Jan 17, 2014.

Breakdown of US State substitution laws on biosimilars and bio-differences

The US biosimilars market is becoming complicated even before the US FDA has issued manufacturers with clear guidance, thanks to a rapidly evolving patchwork of varying State substitution laws. Although the Patient Protection and Affordable Care Act, also known as Obamacare (signed into law in 2010), included provisions on biopharmaceuticals and biosimilars, it left up to individual States to decide the precise requirements needed to substitute a biosimilar for a prescribed reference product.
To date Florida, Norht Dakota, Oregon, Utah, Virgina and Indiana have introduced rules governing substitution but rules can vary significantly amongst states.
Source: www.biopharma-reporter.com, May 9, 2014.

Clinical Trial News

New UK rule: sponsors must log trials in public database for ethical approval

Soon sponsors and CROs seeking ethical approval for clinical trials in the UK will have to register them in a public database under the Health Research Authority’s (HRA) transparency plan. The requirement applies to trials of investigational medicinal products, medical devices as well as studies combining the two. Such requirements must be completed within six weeks of the recruitment of the first patient for drug studies. Studies meeting these criteria that are not registered will be deemed to be in breach of good research practice. The new HRA scheme is part of a wider European effort to make clinical research more transparent (see also News from Commission from 4 April 2014), amid criticism that pharmaceutical industry sponsors routinely withhold data from trials that do not yield favourable results for their products.
Source: www.outsourcing-pharma.com, Sept 16, 2013.

Top 5 reasons clinical trials fail & possible solutions (operational factors)

1. Challenges with Feasibility Process

  • Leverage your investigator database.
  • Instead of generic feasibility, focus on higher-quality questions of fewer sites.
  • Close the loop with non-selected sites.
  • Be transparent on enrolment projection.
  • Set expectations from the very beginning.

2. Not Including Site Feedback

  • Get site feedback on protocol development early in the process.
  • Walk through the protocol as a typical patient. If possible remove potential hurdles.
  • Use a Work Breakdown Structure to layout your project deliverables.

3. Protocol too Complex

  • Consider making improvements to your initial study design process
  • For complex protocols, don’t skimp on training and site visits.

4. Expectations Around Site Budgets

  • Discuss site budget during feasibility process to get expectations in-line and avoid delays.
  • Remember that as your protocol changes, your site budget changes.
  • Don’t rely on old site budgets

5. Enrolment & Retention Problems

  • Provide tools for site staff to use when explaining trial to potential patients.
  • Utilize centralized recruitment support.
  • Utilize monitoring visits to discuss issues and brainstorm solutions.
  • Think of factors that might impact patient interest and retention.

Source: www.genengnews.com/gen-news-highlights, Nov 1, 2013.

New IMI project to revolutionise clinical trials for Alzheimer`s drugs

The Innovative Medicines Initiative (IMI) is launching a major new €53M project that will pioneer a novel, more flexible approach to clinical trials of drugs designed to prevent Alzheimer’s disease. The project will focus its efforts on improving the system of ‘proof of concept’ studies, early stage clinical trials in which researchers seek to determine if a candidate drug is safe and has an impact on the disease in humans.
The project will test a new way of running proof of concept trials, in which several candidate drugs are simultaneously compared to a placebo. In this scenario, only about 20% of patients are in the placebo group, compared to 50% in conventional trials. Furthermore, this novel ‘adaptive’ trial design allows researchers to adapt the trial design in response to emerging results. E.g. if a candidate medicine appears to be particularly effective in only certain categories of people, then assignment of that medicine can be preferentially directed to those people to confirm this finding. Similarly, new candidate drugs can be added to the trial and medicines that prove ineffective can be dropped. The design also allows to test both individual drugs and combinations of different medicines. This innovative trial design has already been found to be effective for testing new treatments for breast cancer.
Source: www.imi.europa.eu/content/11th-call-alzheimers-topic, Dec 11, 2013.

Sanofi aligns data sharing with industry groups

Sanofi will abide by principles for the sharing of clinical data announced by U.S. and European biopharma industry groups. The principles go beyond recent industry norms but fall short of the total open-access sought by Europe’s chief drug regulator and at least one pharma giant.
Starting this month, Sanofi will provide access to clinical trial data and related documents, including clinical study reports (CSR), for studies sponsored by Sanofi companies that conduct clinical studies in humans. The studies must have been submitted to U.S. and European Union regulatory agencies, and the product supported by the studies must have been approved by both agencies on or after January 1, 2014.
Sanofi’s practices fall in line with those agreed to by the European Federation of Pharmaceutical Industries and Associations (EFPIA) whose president is Sanofi CEO C.A. Viehbacher and the Pharmaceutical Research and Manufacturers of America (PhRMA).
Source: www.genengnews.com/gen-news-highlights, Jan 3, 2014.

Research News

Potential brain tumour drug can distinguish cancer from healthy cells

Scientists from the UK Samantha Dickson Brain Cancer Unit, which is funded by The Brian Tumour Charity, at the UCL Cancer Institute in London focused on glioblastoma, the most common type of brain tumour. Using cells growing in the lab, they treated glioblastoma cells and healthy cells with more than 150 potential drugs, and compared the responses. A molecule called J101, was able to stop the cancer cells growing but left the normal cells alone.
J101 blocks a cellular ‘messaging’ molecule called polo-like kinase 1 (Plk1), which tells cancer cells to multiply. The researchers found three drugs already in clinical development that stop the growth of cancer cells in the same way and pass easily into the brain as J101.
Tests in mice showed that one of the reasons this new molecule is able to tell the difference between healthy brain cells and cancer cells may be due to a faulty p53 gene. Mistakes in p53 are the most common gene faults found in glioblastoma tumours. The study was published in PLOSONE.
Source: Cancer Research UK, Oct 30, 2013.

Stay-or-stray protein implicated in spread of cancer cells

A group of researchers at the University of Bristol and Birmingham, while studying breast and prostate cancer cells, found that a particular cellular mechanism enhances cancer’s ability to spread.
This mechanism involves a transcription factor known as proline-rich homeodomain protein (PRH). If PRH levels are low, or if PRH’s subcellular distribution is unfavourable that is, if PRH is relatively unavailable in the nucleus, cancer cells migrate faster. To confirm this finding, the scientists also determined that without PRH, cancer cells move through a porous gel more efficiently.
Scientists have been aware of PRH’s role in controlling cell growth and specification for some time, but this is the first time PRH has been implicated in the movement of cancer cells. Findings were published in Oncology.
Source: www.genengnews.com/gen-news-highlights, Nov 19, 2013.

Frederick Sanger, father of genomic era, dies at 95

Frederick Sanger, the British biochemist and double Nobel Laureate whose research laid essential groundwork for the sequencing of amino acids and later DNA, has died at age 95. Dr. Sanger’s sequencing work earned him two Nobel prizes in chemistry the first in 1958 for the sequencing of human amino acids; the second in 1980 for his development of a technique to sequence human DNA.
Source: www.genengnews.com/gen-news-highlights, Nov 20, 2013.

Need for standards in life science research

A report entitled “The Case for Standards in Life Science Research: Seizing Opportunities at a Time of Critical Need,” released by the Global Biological Standards Institute (GBSI) takes a hard look at the problem of irreproducibility and finds that it is experienced by about 75% of academics and industry scientists. It also concludes that irreproducibility is largely the consequence of inadequate standards next to the absence of standards. Scientific misconduct however is practically insignificant.
The study, conducted by a third-party organization interviewed almost 60 stakeholders across the life science community. The report integrates the findings of these interviews, an extensive review of the literature, and other sources, including government agencies and pharmaceutical industry. A key finding is that irreproducibility is a pervasive, systemic problem across research settings and profoundly affects the entire community, from bench scientists to patients.
The GBSI stated that it is coordinating a global effort to increase the level of credible, replicable, and translatable results in life science research by driving the expanded development and adoption of standards through policy initiatives, thought leadership, and education.
Source: www.genengnews.com/gen-news-highlights, Dec 11, 2013.

Life Tech teams Up with NIH to make gene silencing data publicly available

Large-scale information on the biochemical makeup of small interfering RNA (siRNA) molecules is now available for free to all researchers as the result of a collaboration between the NIH National Center for Advancing Translational Sciences (NCATS) and Life Technologies.
Until now, a major limitation in the scientific community’s use of RNAi data has been the lack of a publicly available dataset, along with siRNA sequences directed against every human gene. To address this problem, NCATS and Life Technologies are providing all researchers with access to siRNA data from Life Technologies’ Silencer Select siRNA library, which includes 65,000 siRNA sequences targeting more than 20,000 human genes. Simultaneously, NCATS is releasing complementary data on the effects of each siRNA molecule on biological functions. The information is available to the public free-of-charge through NIH’s public database PubChem.
Source: www.genengnews.com/gen-news-highlights, Dec 11, 2013.

MedImmune and Johns Hopkins sign $6.5M five-year research pact

MedImmune, AstraZeneca’s global biologics research and development arm, and The Johns Hopkins University entered into a five-year, $6.5M research collaboration. The collaboration aims to extend both MedImmune’s portfolio of biologics and AstraZeneca’s portfolio of small molecule compounds. Key projects will include:

  • Oncology: explore the role of immune system cells in tumour growth and use findings to identify new cancer drug targets.
  • Respiratory, inflammation, autoimmunity: study the mechanisms underlying rheumatoid arthritis and validating potential key therapeutic targets.
  • Infectious disease: assessing monoclonal antibody combinations to aid in clinical candidate selections to more effectively prevent recurrent infections in high-risk patients. Advance ongoing work by MedImmune to prevent serious and costly drug-resistant infections.
  • Antibody discovery and protein engineering: find innovative ways to manufacture complex next-generation biologic drugs.

Source: www.genengnews.com/gen-news-highlights, Dec 11, 2013.

NIH makes $40M available

NIH has begun soliciting requests for the $40 million in grant funding set aside by the federal government under President Obama’s Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative.
NIH said its first wave of spending in the BRAIN initiative will focus largely on technology development designed to advance basic science. Awards are expected to be announced in Sept 2014.
NIH’s $40 million is part of an overall $110 million expected to be spent on BRAIN in 2014 by NIH and two other federal agencies, National Science Foundation, and the Defence Advanced Research Projects Agency (DARPA).
Source: www.genengnews.com/gen-news-highlights, Dec 18, 2013.

Peptide from sea bacterium may turn the tide of MRSA resistance

Marine bacteria may yield the next generation of Methicillin-resistant Staphylococcus aureus (MRSA) fighting drugs according to a team that used a peptide from a Pacific Ocean bug to render the hospital superbug less lethal.
Research showed that Solonamide B, a peptide extract from an organism called “Photobacterium halotolerans” found in the Solomon Islands stops Staphylococci making both toxins that can kill infected patients and proteins that help resistant strains like MRSA withstand treatment.
Source: http://www.in-pharmatechnologist.com, Jan 9, 2014.

Editing the human genome one letter at a time

A challenge that prevents researchers from efficiently generating and studying genetic diseases is that they can exist at frequencies as low as 1%, making the task of finding and studying them labour intensive. Researchers at the Gladstone Institute designed a special fluorescent probe that distinguishes mutated sequence from the original sequences. This allowed sorting through both sets of sequences and detecting mutant cells even when they made up as little one in every thousand cells. The level of sensitivity is more than one hundred times greater than traditional methods.
To engineer the genome, they developed a method that allows efficient detection of a mutation, sib-selection (originally a yeast cloning method) and isolation of rare, scarless clones with the desired mutation. Using a recently developed droplet digital PCR and adapted iPS cell growth conditions they isolated rare mutant clones with unprecedented efficiency. The method was used to introduce disease-associated point mutations into five genes (PHOX2B, PKP2, RBM20, PRKAG2 and BAG3).
According to the researchers the method provides a novel way to capture and amplify specific mutations that are normally exceedingly rare and could be the basis for the next phase of human genetics research. The work was published in Nature Methods.
Source: www.genengnews.com/gen-news-highlights, Feb 10, 2014.

Microrobot gives tissue engineering and bioprinting a boost

Scientists at Brigham and Women’s Hospital and Carnegie Mellon University developed a microrobotic technique to assemble the components of complex materials, which are critical to tissue engineering and 3D printing.
Untethered magnetic microrobotic coding was used for precise construction of individual cell-encapsulating hydrogels. The microrobot can move one hydrogel at a time to build structures, which is critical as human tissue architecture is complex, with different cell types at various levels and locations. The location of the cells determines how the structure will ultimately function.
Further benefits may be realized by using numerous microrobots together in bioprinting, the creation of a design that can be utilized by a bioprinter to generate tissue and other complex materials in the laboratory environment. The study was published in Nature Communications.
Source: www.genengnews.com/gen-news-highlights, Feb 11, 2014.

Nanoshells deliver drugs as smart bombs to cancer cells

Scientists in China say they have created nanoparticles that are capable of delivering drugs to targeted cancer cells. The multifunctional “smart” gold nanoshells could lead to more effective cancer treatments by overcoming a major limitation of modern chemotherapy techniques, i.e., the ability to zero in on specific cancer cells and leave healthy cells untouched.
Small peptides on the surface of the nanoshells are the key to the improved targeting ability, guiding the nanoshells to specific cancer cells and attaching to markers on the surface of the cells. The acidic environment of the cancer cells then triggers the offloading of the drugs. The nanostructure of the nanoshells could also allow near-infrared light to be absorbed and converted into heat, opening up the possibility for targeted hyperthermia treatment whereby cancer cells are exposed to slightly higher temperatures than usual to destroy them. The study was published in Biomedical Materials.
Source: www.genengnews.com/gen-news-highlights, Feb 14, 2014.

Changing skin cells into beating heart cells

The reprogramming of skin cells into heart cells has required the insertion of several genetic factors to spur the reprogramming process. However there are potential problems with scaling this gene-based method into successful therapies.
A research team at the Gladstone Institutes used skin cells extracted from adult mice to screen for small molecules that could replace the genetic factors. The team previously harnessed the power of small molecules to reprogram skin cells into neurons and, more recently, insulin-producing pancreas cells.
The team identified a defined small molecule cocktail that enables the highly efficient conversion of mouse fibroblasts into cardiac cells with only one transcription factor, Oct4, without any evidence of entrance into the pluripotent state. The small molecule-induced cardiomyocytes spontaneously contract and exhibit a ventricular phenotype. Furthermore, the induced cardiomyocytes pass through a cardiac progenitor stage. The study was published in Cell Reports.
Source: www.genengnews.com/gen-news-highlights, Feb 21, 2014.

Getting a better peek at small molecules inside live cells

In a study published in Nature Methods, a team led by Dr. W. Min, professor of chemistry, reports on the development of a general method to image a broad spectrum of small biomolecules such as small molecular drugs and nucleic acids, amino acids, and lipids for determining where they are localized, and how they function inside cells.
The group departed from the conventional paradigm of fluorescence imaging of fluorophores, and pursued a novel combination of physics and chemistry. Specifically, they coupled an emerging laser-based technique called stimulated Raman scattering (SRS) microscopy with an alkyne tag, a chemical bond that, when it stretches, produces a Raman scattering signal at a unique frequency. This new technique avoids perturbation that occurs with large fluorescent tags, while obtaining high detection specificity and sensitivity by SRS imaging. By tuning the laser colours to the alkyne frequency and scanning the laser beam across the sample SRS microscopy can pick up the unique stretching motion of the C=C bond carried by the small molecules and produce a three-dimensional map of the molecules inside living cells and animals.
Source: www.genengnews.com/gen-news-highlights, Mar 3, 2014.

Homo Minutus evolves as a bench top human in lab

Scientists report that they have taken a step closer to creating a “benchtop human” on which to carry out lab and toxicology tests. Homo minutus, as it is named, is not a real person but rather an interconnected human organ construct. The latest advance is the successful development and analysis of a constructed human liver that responds to toxic chemical exposure. Dr. Wikswo, professor and director of the Vanderbilt Institute for Integrative Biosystems Research and Education (VIIBRE) said the achievement is the first result from a five-year, $19M multi-institutional effort led by himself and Dr. Iyer, scientist at Los Alamos National Laboratory (LANL).
The project is developing four interconnected human organ constructs liver, heart, lung and kidney that are based on a miniaturized platform nicknamed ATHENA (Advanced Tissue-Engineered Human Ectypal Network Analyzer). The project is supported by the Defence Threat Reduction Agency. Ultimately, the goal is to connect the individual organ modules chemically in a fashion that mimics the way the organs are connected in the body, via a blood surrogate. The ATHENA researchers hope that this homo minutus, with its ability to simulate the spatial and functional complexity of human organs, will prove to be a more accurate way of screening new drugs for potency and potential side-effects than current methods.
Source: www.genengnews.com/gen-news-highlights, Mar 27, 2014

Lab-grown vaginas a success years after patient implantation

Vaginal organs engineered in the laboratory can be used successfully in human beings, say researchers. The researchers base this assertion on the results of follow-up visits by the first four-implant recipients, young women who were aged 13–18 years at the time the engineered organs were surgically implanted.
The strategy used for growing the vaginas involved the application of cells to a biodegradable, three-dimensional scaffold which had been used earlier, but only on relatively small structures, such as urethras and replacement bladders. Nonetheless, work with smaller structures had demonstrated that once cell-seeded scaffolds are implanted in the body, nerves and blood vessels form and the cells expand and form tissue. At the same time the scaffolding material is being absorbed by the body, the cells lay down materials to form a permanent support structure gradually replacing the engineered scaffold with a new organ. This tissue engineering approach was developed by researchers led by A. Atala, M.D., director of Wake Forest Baptist Medical Center’s Institute for Regenerative Medicine. The work has been published in the Lancet.
Source:www.genengnews.com/gen-news-highlights, Apr 11, 2014.

Organs-on-a-chip advance goes to the very marrow

Current in vitro models of hematopoiesis fail to capture the complexities of bone marrow, which has an integral relationship with bone. Within bony nooks and crannies, bone marrow cells find varied hematopoietic niches, which accommodate cells that have very particular preferences such as for warmer or cooler spots. Moreover, bone marrow cells communicate with each other by secreting and sensing a variety of biomolecules, which act locally to tell them whether to live, die, specialize, or multiply.
Undeterred by these complexities, scientists at Harvard’s Wyss Institute for Biologically Inspired Engineering developed a bone marrow-on-a-chip that they say reproduces the structure, functions, and cellular makeup of bone marrow. The scientists first engineered new bone in vivo, removed it whole, and perfused it with culture medium in a microfluidic device. The engineered bone marrow, wrote the authors, retained “hematopoietic stem and progenitor cells in normal in vivo-like proportions for at least one week in culture.” The work has been published in Nature Methods.
Source: www.genengnews.com/gen-news-highlights, May 6, 2014.

Stem Cell News

Answering the question: what will stem cells become?

Scientists at the University of Toronto say they have developed a technique that can rapidly screen human stem cells and better control what they will turn into. The researchers developed a high-throughput platform to test many compounds or drugs at once, with controllable environments to screen human pluripotent stem cells (hPSCs) in. With it, they can control the size of the stem cell colony, the density of cells, and other parameters in order to better study characteristics of the cells as they differentiate or turn into other cell types. Studies were done using stem cells in microenvironments optimized for screening and observing how they behaved when chemical changes were introduced.
Single-cell protein expression profiling, revealed that Oct4 and Sox2 costaining discriminates pluripotent, neuroectoderm, primitive streak, and extraembryonic cell fates. The findings were published in Nature Methods.
Source: www.genengnews.com/gen-news-highlights, Oct 23, 2013.

Stem cell leaders call for human embryome project

Just as an international consortium was formed to map and sequence the human genome, now a group of stem cell and regenerative medicine scientists say it’s critical that a similar project is established focused on the human embryome. The message was delivered by a panel discussion at the World Stem Cell Summit in San Diego: “From Mapping the Genome to Mapping the Embryome: The Urgent Need for an International Initiative,” moderated by M. West, Ph.D., CEO of Biotime.
Dr. West and his colleagues adamantly believe that there needs to be a large international effort aimed at mapping the cellular and molecular basis of all human life starting with the fertilized egg and working its way up to the body of the adult. This is what it is termed the embryome.
The major problem in identifying them, is that no one has ever mapped the molecular markers or even a rudimentary cell ontology tree, i.e., mapped out the tree from the fertilized egg to the cells of the human body.
Source: www.genengnews.com/gen-news-highlights, Dec 5, 2013.

Bone marrow stem cells could defeat drug-resistant tuberculosis

Bone-marrow stem cells are known to migrate to areas of lung injury and inflammation and repair damaged tissue. Since they also modify the body’s immune response and could boost the clearance of tuberculosis (TB) bacteria, researchers from Stockholm’s Karolinska University Hospital, tested them in patients with TB.
In a phase 1 trial, 30 patients with either Multi-Drug-Resistant or Exclusively-Drug-Resistant TB aged between 21 and 65 who were receiving standard TB antibiotic treatment were also given an infusion of around 10 million of their own stem cells from their own bone marrow. During 6 months of follow-up, the researchers found that the infusion treatment was generally safe and well tolerated, with no serious side effects. Analyses of the results showed that 16 patients treated with stem cells were deemed cured at 18 months compared with 5 of 30 TB patients not treated. The study was published in The Lancet medical journal.
Source: EuropaBio Newsletter, Jan 6-10, 2014.

Living brain cells generated from biobanked Alzheimer’s tissue

Researchers at the New York Stem Cell Foundation Research Institute and the Columbia University Medical Center report that they have generated induced pluripotent stem (iPS) cells lines from non-cryoprotected brain tissue of patients with Alzheimer’s disease.
The new stem cell lines allow researchers to “turn back the clock” and observe how Alzheimer’s develops in the brain, potentially revealing the onset of the disease at a cellular level long before any symptoms associated with Alzheimer’s are displayed. These reconstituted Alzheimer’s cells could also provide a platform for drug testing on cells from patients that were definitively diagnosed with the disease.
Until now, the only available method to definitively diagnose Alzheimer’s disease was to examining the brain of deceased patients. The study was published in Acta Neuropathologica Communications.
Source: www.genengnews.com/gen-news-highlights, Jan 8, 2014.

Belgian scientists repair bones with new stem cell technique

Belgian medical researchers have succeeded in repairing bones using stem cells from fatty tissue, with a new technique they believe could become a benchmark for treating a range of bone disorders. The team at the Saint Luc University clinic hospital in Brussels have treated 11 patients, eight of them children, with fractures or bone defects that their bodies could not repair, and a spin-off is seeking investors to commercialize the discovery.
The groundbreaking technique of Saint Luc’s centre for tissue and cellular therapy removes a sugar cube sized piece of fatty tissue from the patient. The stem cells are then isolated and used to grow bone in the laboratory. The work has been published in the journal Biomaterials.
Source: EuropaBio Newsletter, Jan 13-17, 2014.

Link identified between stem cell and leukemia cell

Research from the University of California’s San Diego School of Medicine has identified a protein that is named, Lis1 that is needed for hematopoietic stem cell formation as well as leukemia cells. The discovery has the potential of being a new target in the treatment of leukemia and may provide more effective methods of chemotherapy as the protein regulates and sustains the cell’s growth.
The research team deleted the Lis1 protein from hematopoietic stem cells in the mouse, which caused an excessive differentiation that depleted the reserve of the undifferentiated stem cells. The lack of this reserve then caused a loss of cells that are specialized for forming new blood. This test finally ended in a “bloodless mouse” that was lethal and resulted in the mouse embryos dying before reaching birth.
The study then examined mice leukemia models with the Lis1 protein when it was turned off. The control mice were give blast-crisis chronic myeloid leukemia (CML) and then the mice were treated with the drug tamoxifen. These mice all died, but mice given the same type of leukemia with cells that were engineered to lose the Lis1 protein did not develop into leukemia in the mice. Findings were published in the journal Nature Genetics.
Source: EuropaBio Newsletter, Feb 3-7, 2014.

Stem cell bank to consolidate Europe’s stem cell surplus

Demand for induced pluripotent stem (iPS) cells threatens to outstrip supply, despite the proliferation of stem cell banking initiatives, many of which are simply too small to achieve industrial scale. Many research projects that generate iPS cells have little motivation to produce stem cell surpluses, though they could easily do so.
To resolve this bottleneck in the distribution of iPS cells, a consortium of 26 partners has formed to establish the European Bank for Induced Pluripotent Stem Cells (EBiSC) with the support from the Innovative Medicines Initiative (IMI). The EBiSC, a €35M project, will start systematically cataloguing and distributing iPS cells.
Source: www.genengnews.com/gen-news-highlights, Feb 4, 2014.

Commercial embryonic stem cell culturing now possible

Commercial-scale human Embryonic Stem cell (hES) culturing is now a possibility thanks to a new technique that the developer’s claim resolves the ethical, technical and regulatory issues that have held back research. The new approach developed by scientists from the Karolinska Institute in Sweden and Duke-NUS Graduate Medical School in Singapore involves growing a harvested embryonic stem cell into a culture on a matrix of two adhesion proteins a recombinant of Laminin-521 that is associated with pluripotent stem cells and epithelial cadherin. As a result, it is now possible to take a single hES cell and culture it in vitro thereby providing sufficient material for a wide range of applications. A key aspect of the new hES growth matrix is its human origin, which is important for the production of cell therapies. Currently most commercial growth matrix technologies are based on animal cells or human fibroblasts, which can contaminate stem cells.
Source: http://www.biopharma-reporter.com, Feb 5, 2014.

Ireland secures stem cell supplies with new manufacturing centre

The new Centre for Cell Manufacturing Ireland (CCMI) in Galway means Ireland-based scientists will be able to manufacture culture-expanded stem cells for drug research for the first time. The CCMI plans to supply stem cells for use in clinical trials following regulatory approval arising from pre-clinical data generated at the Regenerative Medicine Institute (REMEDI) in Galway. In addition to stem cells manufacture the CMMI are offering a customized advanced therapeutic medicinal product (ATMP) manufacturing services.
Source: http://www.biopharma-reporter.com, Feb 12, 2013.

Study sets sights on substandard stem cells

In the absence of quality control standards, commercially sourced stem cells may or may not meet the expectations of drug developers. Dissatisfied with this state of affairs, a team of scientists at Harvard University’s Wyss Institute for Biologically Inspired Engineering has proposed a stem cell quality index.
The scientists were particularly interested in stem cell-derived cardiomyocytes, but it is conceivable that their suggestions could be generalized to other kinds of differentiated cells. A set of 64 crucial parameters from more than 1000 were identified, by which to judge stem cell-derived cardiomyocytes. The result was a “multiparametric quality assessment rubric” which promises to give scientists and pharmaceutical companies the ability to quantitatively judge and compare the value of the countless commercially available lines of stem cells. The work has been published in Stem Cell Reports.
Source: www.genengnews.com/gen-news-highlights, Mar 7, 2014.

Misconduct found in acid-bath stem cell study

A committee organized by the RIKEN Centre for Developmental Biology has concluded that RIKEN’s Dr. H. Obokata, the lead researcher of a controversial stem cell study, is guilty of scientific misconduct. The study yielded two papers that asserted adult stem cells could be rendered pluripotent by means of external stimuli, such as an acid bath or mechanical stress. The results described in the papers published in Nature have not been replicated, despite repeated attempts by other research groups. In addition, numerous questions about the original papers have been raised.
The RIKEN committee has thus far confined its investigation to six issues, which were outlined in an interim report issued March 14. Of the six issues considered, four were dismissed as mere errors. Two issues, however, were deemed to constitute scientific misconduct. The first issue refers to a figure showing an electrophoresis gel. One lane of this gel, Dr. Obokata admits, was swapped for another, but she insists that she was merely showing a clearer version of the lane, not fabricating evidence. The second issue is the use of the same figures to illustrate different studies which concerned the RIKEN investigators because the two studies were so different. In Dr. Obokata’s thesis, the figures were used to illustrate cells that had been stressed with a pipette. In the Nature paper, the figures supposedly showed cells that had been subjected to an acid bath.
In a written statement, RIKEN also indicated that it will explore ways to prevent recurrences of misconduct, and that RIKEN researchers will continue attempts to replicate the results of the study while also seeking third-party validation.
Source: www.genengnews.com/gen-news-highlights, Apr 1, 2014.

UK scientists announce breakthrough in stem cell manufacturing

Scientists at the University of Nottingham have created a new combination of hydrogels that allows dense tissue structures to be produced from human pluripotent stem cells (hPSC) in a single step process never achieved before.
A new stem cell microenvironment was created, which allows for both stem cell self-renewal and the evolution into heart cells. The material is a hydrogel containing two polymers and an aligate-rich environment, which allows proliferation of cells with a chemical switch to render the environment collagen-rich when the cell population is large enough. The discovery could be useful for early stage stem cell manufacturing companies that are looking to speed products through early development.
Source: www.biopharma-reporter.com, April 2, 2014.

Bone marrow stem cells may help patients recover from stroke

Researchers at the University of California-Irvine’s Sue & Bill Gross Stem Cell Research Center report that stem cells taken from bone marrow may help patients recover from a stroke. S. Cramer, M.D. a neurologist, and W. Zhao, Ph.D., a biomedical engineer, identified 46 studies that examined the use of mesenchymal stromal cells (MSCs), a type of multipotent adult stem cells mostly processed from bone marrow, in animal models of stroke. They found MSCs to be significantly better than control therapy in 44 of the studies.
In preclinical studies, MSCs have consistently improved multiple outcome measures, with very large effect sizes. Results were robust across species studied, administration route, species of MSC origin, timing, degree of immunogenicity, and dose, and in the presence of comorbidities. In contrast to meta-analyses of preclinical data for other stroke therapies, higher-quality MSC preclinical studies were associated with larger behavioral gains. These findings support the utility of further studies to translate MSCs in the treatment of ischemic stroke in humans.
Source: www.genengnews.com/gen-news-highlights, Apr 10, 2014.

Human neural stem cells become neurons in monkey brains

A team of scientists based in Korea and Canada who transplanted human neural stem cells (hNSCs) into the brains of nonhuman primates (NHPs) report that the hNSCs had differentiated into neurons at 24 months and did not cause tumours.
The hNSCs were labelled with magnetic nanoparticles to enable them to be followed by magnetic resonance imaging. The researchers, who did not use immunosuppressants, claim their study is the first to evaluate and show the long-term survival and differentiation of hNSCs without the need for immunosuppression. The researchers maintain that hNSCs could be a key source for cell replacement and gene transfer for the treatment of Parkinson’s disease, Huntington’s disease, Alzheimer’s disease, amyotrophic lateral sclerosis, spinal cord injury, and stroke. The study has been published in Cell Transplantation.
Source: www.genengnews.com/gen-news-highlights, Apr 24, 2014.

Agenda

European Biotechnology Congress
15-18 May 2014, Lecce, Italy
For more info: http://eurobiotech2014.eu/php/

World Stem Cells Regenerative Medicine Congress
20-22 May 2014, London, UK
For more info: www.terrapinn.com/conference/stem-cells/index.stm

Biosimilars 2014
21-22 May, Berlin, Germany
For more info: www.informa-ls.com/event/biosimilars2014

BioForum 2014
28-29 May, Lodz, Poland
For more info: http://cebioforum.com/index.php/media-about-us

Rentschler-Congress the “3. Laupheimer Zelltage 2014”
2-3 June 2014, Laupheim, Germany
This time focusing on “Processes for Virus-based Biologics”.

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For more info: www.laupheimer-zelltage.com

Asia Biotech Invest
3-5 June, Hong Kong, China
For more info: http://asiabiotechinvest.com

Vaccine Technology V
8-13 June 2014, Playa del Carmen, Mexico
For more info: www.engconf.org/conferences/biotechnology/vaccine-technology-v-2

BioVision 2013: The World Life Sciences Forum
5-6 June 2014, Lyon, France
For more info: www.biovision.org/index.html

3D Cell Culture 2014 – Advanced Model Systems, Applications & Enabling Technologies
25-27 June 2014, Freiburg, Germany
For more info: www.dechema.de/3DCC2014

UPCP 2014 – 3rd International Congress on Personalized Medicine
26-29 June, Prague, Czech Republic
For more info: http://2014.upcp.org

3rd European Bio refinery Training School
7-10 July 2014, Budapest, Hungary.
For more info: http://klimainnovacio.hu/en/biorefineryschool

16th European Congress on Biotechnology (ECB16)
13-16 July 2014, Edinburg, Scotland

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Important: ACTIP members are entitled to a 10% discount* on the registration fee.
For more info: www.ecb16.com
*Discount applies only to the flat rate registration fee and does not stack on top of registration fees that have existing reduced rates.

SwedenBio: Nordic Life Science Days -Invest, Partner, Network
7-9 September 2014, Stockholm Waterfront, Sweden
For more info: www.nlsdays.com

BioPharm America™ 2014—7th Annual International Partnering Conference
22-24 September 2014, Boston, MA, USA
For more info: www.ebdgroup.com/bpa/index.php

EDQM: 50 years of leadership in the quality of medicines
06-08 October 2014, Strasbourg, France
For more info: www.edqm.eu/en/Conference-50th-Anniversary-of-the-EDQM-1617.html

BIO-Europe® 2014 – 20th Annual International Partnering Conference
3-5 November 2014, Frankfurt, Germany
For more info: www.ebdgroup.com/bioeurope/media/about_conf.php

Japanese Association for Animal Cell Technology (JAACT) 2014
10-14 November 2014, Kitakyushu, Japan
For more info: http://jaact2012.jaact.org

BioFIT 2014
2-3 December 2014, Lille, France
For more info: www.biofit-event.com/biofit-2014/about-biofit/

24th ESACT meeting
31 May – 3 June 2015, Barcelona, Spain
For more info: www.esact2015.com

 

Webinars

Sartorius Webinars on “Innovative PAT solutions for advanced automation and process control”

Online Control of Glucose and Lactate during fermentation
14 May 2014, 9.00 – 10.00 and 18.00-19.00 CEST
Automation of bioprocesses – minimize run-to-run variability (Part 1)
19 May 2014, 9.00 – 10.30 and 18.00-19.30 CEST
Automation of bioprocesses – minimize run-to-run variability (Part 2)
20 May 2014, 9.00 – 10.30 and 18.00-19.30 CEST
Integrated Single-Use Sensor Control for bioprocess product stream
25 June 2014, 9.00 – 10.00 and 18.00-19.00 CEST
For more info: www.sartorius.com/en/company/exhibitions-conferences

Sartorius Webinars on “Applied Chemometrics for efficient bioprocess development and robust manufacturing”

Optimization of bioprocesses – minimize time-consuming experiments with DoE (Part 1)
21 May 2014, 9.00 – 10.30 and 18.00-19.30 CEST
Optimization of bioprocesses – minimize time-consuming experiments with DoE (Part 2)
22 May 2014, 9.00 – 10.30 and 18.00-19.30 CEST
BioPAT® Chemometrics Toolbox for efficient bioprocess development and robust manufacturing
28 May 2014, 18.00-18.45 CEST & 24 June 2014, 18.00-18.45 CEST
For more info: www.sartorius.com/en/company/exhibitions-conferences

Sartorius Webinar on “Automated mini bioreactors enable next generation process development”

How the ambr15™ microbioreactor can transform cell line development
3 June 2014, 9.00 – 9.45 and 16.00-16.45 CEST
Process development and optimization using ambr250™ for mammalian and microbial processes
2 July 2014, 9.00 – 9.45 and 16.00-16.45 CEST
For more info: www.sartorius.com/en/company/exhibitions-conferences

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