ACTIP Bulletin no. 73

ACTIP bulletin no. 73, 1st May 2018

Next ACTIP meeting

Hosted by Bayer AG, in Düsseldorf, Germany
7 & 8 June 2018
The meetings are for ACTIP members and individually invited guests.
More information can be obtained via

In this issue
Communications & Courses
Biomanufactoring News
Vaccine News
Business News
European Commission News
Regulatory News
Clinical Trials News
Research News
Stem Cell News

Communications & Courses

Biotech advances in single-use platforms for commercial manufacturing

Sartorius Stedim Biotech has published a special report in association with BioProcess International that allows to:
– Explore recent advances in single-use process platforms.
– Learn about developments allowing the adoption in critical applications.
– Find out how teams of experts can help you implement end-to-end single-use bioprocesses.
– Be informed about cutting-edge developments in sensors and data analysis methods.
Download report at:

Drug development course 2018, 3rd Edition, Sept 30 – Oct 3, 2018, Llafranc, Spain

The course is planned in an intensive threeday schedule with a number of participants limited to 30 in order to facilitate the interaction among them and the lecturers, who will stay for the entire course duration.
The course will cover the following topics: cellular assays for target identification/validation; engineering cells for drug screening and validation; cell selection including primary cells, stem cells and organoids; biochemical assays; biomarkers; animal models including PK/PD, Assays bridging in vitro and in vivo; toxicology; production of reproducible cell material & criteria; assay automation; data analysis and storage.
A limited number of grants, covering the course fee (not travel cost), are provided by ESACT. Applicants to the grants should indicate it in the course application, together with a motivation statement. Priority will be given to young PhD students from academia.
For registration:

Cell culture-based viral vaccines course, 3rd Edition, Sept 18–21, 2018, Llafranc, Spain

ESACT offers an introductory intensive course in cell culture produced viral vaccines to meet the needs of the rapidly expanding research and development in academia and industry in the field. This course introduces the basic principles of vaccination and manufacturing of viral vaccines. Also, the course will provide an overview of the advanced strategies to respond to the challenges of new and established viral infection diseases.
The course has been designed to benefit those starting their research activity in this field, both from academia, industry or public health agencies. It is also of interest for those wishing to up-date their knowledge and know-how in cell production of viral vaccines.
The course will cover the following topics: principles of immunology and virology; cell lines for vaccine production; vaccine markets and immunization policies; upstream process development and intensification; analytical and potency assays; downstream processing of viral vaccines, formulation and delivery systems; equipment; QbD and PAT; regulatory issues; vaccine development and supply solutions. In addition, case studies will address recombinant vaccines, polio vaccines, Dengue vaccine development and influenza vaccines.
A limited number of grants, covering the course fee (not travel cost), are provided by ESACT. Applicants to the grants should indicate it in the course application, together with a motivation statement. Priority will be given to young PhD students from academia.
For registration:

Biomanufacturing News

MilliporeSigma looks beyond ‘burn and bury’ for single-use recycling

MilliporeSigma is urging industry to recycle single-use technologies, which can be made into speed pumps, parking bollards, and plastic palettes. While an advocate of single use technologies, which serve to decrease water and energy usage, by 2025 there will be around 112,000 tonnes of single use technology waste produced per year.
Source:, April 24, 2018.

Pall and Artesyn Biosolutions team to industrialize single-use technology

Pall Corporation and Artesyn Biosolutions will co-develop clinical to commercial-scale solutions for single-use automated bioprocessing systems. The partnership also advances Pall Biotech’s expansion of its Allegro single-use solution portfolio from upstream through downstream and to final filling.
Source:, April 18, 2018.

GE launches modular bioprocessing facility for viral vector vaccines

GE Healthcare Life Sciences has launched a prefabricated, modular bioprocessing facility designed for the manufacture of viral vector-based therapeutics. The latest factory in a box offering (KUBio BSL2) is designed for products that require a biosafety level 2 (BSL 2) environment, such as viral vector based vaccines, oncolytic viruses, and gene and cell therapies. The KUBio facility is designed, constructed and equipped according to current cGMP.
Source:, April 17, 2018.

CHO jumping: Samsung BioLogics to develop in-house cell line

Samsung BioLogics will begin offering its own CHO cell line for its customers’ development projects from 2019 to offer a one-stop services provider for biopharma firms.
Source:, March 20, 2018.

Up to down: BIA and Nuvonis to address ‘last frontier in bioprocessing’

BIA Separations has teamed with Nuvonis Technologies to help vaccine and cell therapy developers tackle the transition from upstream to downstream processing The partnership will combine Nuvonis’ Vero cell bank with BIOA’s Convective Interaction Media monolithic chromatography products as an offering to customers looking for a single source in their process development.
Source:, March 15, 2018.

iBio: Plants, not cell lines, for nextgeneration antibodies

iBio has expanded its offering to develop and manufacture Fc fusion therapeutics using its plant-based expression system. The platform is based on vectors, engineered to contain the target sequence within a viral replicon transferred to an Agrobacterium host, used to introduce target DNA into the plant cell nucleus. The replicon then directs the production of large amounts of target specific messenger RNA in plant cells.
Source:, March 13, 2018.

Draper touts new bioprocessing method as ‘next big advance in CAR T-cell treatment

Draper has designed a microfluidic transduction device (MTD) it claims will help CAR T-cell therapy developers lower costs, up efficiency and potentially bring manufacturing to the patient’s bedside. The device reduces the amount of vector required to achieve high levels of transduction, removes the expense of additional touch labour and reduces potential of contamination compared to open systems.
Source:, March 2, 2018.

Concurrent purification approach could cut ADC manufacture by 25%,ADC Bio

ADC Bio has developed a concurrent conjugation and antibody purification process, not reliant on Protein A capture resin. The Wales, UK-based contract manufacturing organisation is yet to have given the process a name, but says it undertakes bioconjugation for antibody-drug conjugates (ADCs) after both the monoclonal antibody and cytotoxic fragments have been manufactured, with purification of both carried out simultaneously. According to ADC Bio this represents a manufacturing paradigm by integrating several downstream methods and thus reducing the number of CMOs involved in the supply chain.
Source:, Feb 16, 2018.

Lonza rejigs network to tap ‘huge potential’ of cell and gene therapies

Lonza will combine its cell and gene therapy capabilities through four centres of excellence worldwide, following a number of investments. In 2017 the firm completed construction of the Pearland Houston facility and meanwhile acquired the Dutch cell and gene therapy maker PharmaCell last June. The other two centres are located in Portsmouth, New Hampshire and Tuas, Singapore both offering clinical and commercial services.
Source:, Feb 16, 2018.

Nordic project takes ‘manufacturing-on-demand’ approach to future drugs

Nordic Universities will investigate 3D printing, electrospraying, and microfluidics in an industry supported collaboration aimed at revolutionising production in an age of personalised medicine. The collaboration known as Patient-Oriented-Product (POP) will use $6M of funding from NordForsk. The project aims to develop a number of alternative platforms based on manufacturing-on-demand approach together with Nordic-based pharmaceutical companies.
Source:, Feb 7, 2018.

AbSci hopes to make E. Coli great again through engineered expression system

AbSci says its genetically engineered E. coli expression platform has produced yields exceeding 20 g/L for difficult to express products and threatens the dominance of mammalian systems. The SoluPro expression platform technology is based on semi-oxidizing cytoplasm, engineered from the E. coli genome, which the firm says is ideal for protein production, even at commercial scale and can drastically reduce the need for CHO and other mammalian expression platforms.
Source:, Feb 7, 2018.

Making an HIV cure: AGT completes process development for T-cell therapy

American Gene Technologies International (AGT) said its HIV candidate is the most complex cell therapy the industry has delivered to date. It’s genetically modified autologous T-cell product AGT103-T is intended to be an HIV functional cure. Last month, the firm announced completion of pilot runs resulting in a product AGT believes is nearly 4 times the level necessary to restore HIV immunity in HIV positive patients. The product was developed at AGT labs in Rockville, Maryland and AGT is now preparing for a Phase I clinical trial this summer involving 18 patients.
Source:, Feb 6, 2018.

Adaptimmune begins in-house T-cell therapy manufacture at PA plant

Adaptimmune Therapeutics has manufactured the first SPEAR-T cells for a patient at its Navy Yard facility, which will be the primary site for its future manufacturing requirements. The company’s Specific Peptide Enhanced Affinity Receptor (SPEAR) T-cell platform enables the engineering of T-Cells to target and destroy cancer, including solid tumours. Adaptimmune is currently conducting clinical trials with SPEAR T-cells targeting melanoma-associated antigens MAGE-A4, MAGE-A10 and Alpha-fetoprotein across several solid tumour indications.
Source:, Feb 6, 2018.

Paragon expansion to address ‘deficit in gene therapy CDMO capacity’

CDMO said it is building a 150,000 sq. ft. biomanufacturing facility in Maryland in preparation for a double-digit growth in the gene therapy space. The facility will make clinical batches of gene therapies for customers by Q4 with commercial manufacturing capabilities offered in the next 2 years. The plant will be equipped with 5 out of 25 several 500L and 2000L single-use bioreactors.
Source:, Jan 24,2018.

Vaccine News

Sanofi: €350m vaccine investmentpart of ‘digital factory’ ambition

A new vaccine manufacturing facility in Toronto, Canada will boast single-use tech and closed continuous process to help improve productivity and performance, Sanofi said. The firm announced it is investing €350M to build a facility equipped to produce five-component acellular pertussis antigen used in its diphtheria and tetanus vaccines.
Source:, April 13, 2018.

Personalized cancer vaccine shows promise in patients with advanced ovarian cancer

A new personalized vaccine made by exposing a patient’s own immune cells to the contents of a patient’s own tumour cells has been shown to improve immune responses and increase survival rates in patients with ovarian cancer. The vaccine, which was evaluated in a pilot clinical trial conducted at the University of Pennsylvania School of Medicine, was administered alone or in combination with other immunomodulatory drugs. Because the vaccine performed so well, amplifying T-cell responses against both common antigens and patient-specific neoantigens expressed on tumours, the scientists who ran the trial concluded that the vaccine warrants further clinical testing. One patient, after two years of vaccinations, was disease-free for another five years without further treatment.
The findings were published in the journal Science Translational Medicine.
Source:, April 12, 2018.

Synthetic peptide platform to make orally active & room-temperature vaccines

Researchers are developing a vaccine production platform designed to make ingestible, temperature-stable synthetic drugs. Scientists from James Cook University in Australia and Cardiff University in Wales have made a synthetic influenza A vaccine using the platform, as detailed in a study published in the Journal of Clinical Investigation. The proof of concept study shows vaccinated mice were protected from potentially lethal doses of swine flu, as were human cells in the lab.
Source:, March 20, 2018.

Universal flu vaccine developer BiondVax signs CRO to advance Ph III trial

BiondVax is working closely with a CRO to advance its universal flu vaccine candidate, which will be manufactured in-house and in collaboration with a CMO to support techtransfer pending a successful trial. BiondVax is the developer of the universal flu vaccine candidate M-001 comprised of nine epitopes common to the vast majority of influenza strains, including type A and B.
Source:, March 15, 2018.

Novavax: Flu vaccine failings due to ‘outdated’ egg-based manufacturing

Egg adaptation is the biggest culprit in this year’s poor flu vaccine efficacy said Novavax, a firm developing a product based on recombinant protein nanoparticles. The severity of the 2017-2018 flu season has led to questions of how well this year’s vaccines have worked. The FDA suggested the efficacy was around 25%. There are a number of reasons behind this, but most of the blame has been put down on the H3N2 influenza strain.
Source:, March 15, 2018.

Sanofi looks to cell culture tech for universal flu vaccine

Sanofi Pasteur has licensed SK Chemicals’ mammalian cell culture platform for vaccine production in a deal worth up to €126M. The mammalian cell culture technology is used to make SK Chemicals’ commercial trivalent and quadrivalent seasonal influenza vaccines and now Sanofi will use it as part of its own vaccine development program.
Source:, Feb 14, 2018.

Universal flu vaccine a nanoparticle that is all stalk, no head

Designs for universal flu vaccines try to emulate the relatively changeless parts of viral proteins, not the highly variable parts that are targeted by seasonal vaccines. The changeless parts, however, tend to be unstable. To develop a vaccine candidate that can present stabilized versions of these relatively changeless structures, scientists based at Georgia State University built double-layered protein nanoparticles.
The scientists fabricated PNps [protein nanoparticles] approximately the size of the influenza A virions with a core of M2e displaying a shell of conserved hrHA domains. The nanoparticle protects the antigenic protein so it won’t be degraded and immune cells have a good ability to take in this nanoparticle, so this nanoparticle is much, much better than a soluble protein to induce immune responses.
To determine the effectiveness of the nanoparticle vaccine, the researchers immunized mice twice with an intramuscular shot. Then, the mice were exposed to several influenza viruses: H1N1, H3N2, H5N1, and H7N9. Immunization provided universal, complete protection against lethal virus exposure and dramatically reduced the amount of virus in the lungs. The work was published in Nature Communications.
Source:, Jan 24, 2018.

Personalized dendritic cell vaccines could improve cancer Immunotherapy

Dendritic cell vaccines, a promising cancer immunotherapy approach, usually come from dendritic cells that have been force-fed with tumour antigens. Dendritic cells, however, can also be modified so that they gorge themselves on antigen-carrying exosomes released by tumour cells. Although ordinary dendritic cells usually consume antigens from lab-grown cancer cells, modified dendritic cells may feast on antigen-carrying exosomes from a patient’s own tumour, improving the prospects for personalized dendritic cell vaccines.
Scientists at the Swiss Institute for Experimental Cancer Research, part of EPFL developed modified dendritic cells. The scientists created a lentivirus-encoded chimeric receptor. They call it an extracellular vesicle (EV)-internalizing receptor, or EVIR. It has been optimized to enable the selective uptake of cancer-cellderived EVs by dendritic cells.
After constructing the EVIR, the EPFL team demonstrated that it endowed dendritic cells with the capacity to specifically recognize cancer cell–derived extracellular vesicles.
Although the new technology has the potential to increase the efficacy and specificity of dendritic cell vaccines, further preclinical work is required before it can be translated into a cancer treatment. The work was published in the journal Nature Methods.
Source:, Jan 23, 2018.

Business News

Biogen and Ionis launch $1B+ expanded neuro drug partnership, following Spinraza success

Six years after they began partnering to successfully develop the spinal muscular atrophy treatment Spinraza® (nusinersen), Biogen and Ionis Pharmaceuticals have launched a 10-year, $1 billion-plus expanded collaboration to develop more neurological drugs.
The companies plan to develop novel antisense drug candidates for a broad range of neurological diseases for which few treatments now exist, including dementia, neuromuscular diseases, movement disorders, ophthalmology, diseases of the inner ear, and neuropsychiatry.
Biogen will retain the first choice of neurology targets on which to exclusively collaborate with Ionis. Ionis agreed to oversee the identification of antisense drug candidates based on selected targets, while Biogen will be responsible and pay for nonclinical studies, clinical development, manufacturing and commercialization.
Source:, April 20, 2018.

Sanofi using Berkeley Lights’ tech to catalyze cell line development

Berkeley Lights has signed a new agreement with Sanofi through which the company will use its Beacon OptoFluidic Platform to fast-track cell line development. The benefit of the Beacon Platform is the speed in which it can identify the top producing, stable clones to be taken in scale up. In just 45 days the platform can identify the top 48 clones.
Source:, April 20, 2018.

Shire sells cancer business for $2.4B

Shire has sold its oncology business to French firm Servier S.A.S as it looks to better align its long-term strategy.
Source:, April 17, 2018.

GSK hands off rare disease gene therapy portfolio to Orchard Therapeutics

GlaxoSmithKline said it will hand off its rare disease gene therapy portfolio including its European-approved gene therapy Strimvelis™ to two-year-old start-up Orchard Therapeutics in return for taking a 19.9% stake in the acquirer, as well as undisclosed milestone payments and royalties, and a seat on Orchard’s board.
Source:, April 12, 2018.

Mylan to commercialise Fujifilm’s Humira biosimilar in Europe

Fujifilim Kyowa Kirin Biologics has teamed up with Mylan for the commercialisation of its Humira (adalimumab) biosimilar, FKB327. The EMA accepted to review the Market Authorization Application for adalimumab biosimilar in May 2017. If approved the product will be launched by drug maker Mylan.
Source:, April 11, 2018.

Alexion to acquire Wilson Therapeutics for $855M

Alexion Pharmaceuticals plans to shell out $855M to acquire Wilson Therapeutics, the companies said, in a deal that would add its Phase III candidate for the rare genetic liver disorder and company namesake Wilson disease to the buyer’s shifting pipeline.
Source:, April 11, 2018.

Novartis upping presence in gene therapies through $8.7bn AveXis buy

Novartis will add an AAV9 gene delivery platform and manufacturing facility through the $8.7B acquisition of AveXis. Novartis would gain breakthrough-designated candidate AVXS-101, an intended one-time gene replacement therapy for spinal muscular atrophy, which it aims to submit to the US FDA for review by Q2 2018.
Source:, April 10, 2018.

Roche completes acquisition of Flatiron Health

Roche announced that it has completed the acquisition of Flatiron Health, a privately held healthcare technology and services company headquartered in New York City, US. Flatiron Health is a market leader in oncology-specific electronic health record (EHR) software as well as in the curation and development of real-world evidence for cancer research. Under the terms of the agreement, the transaction value for the acquisition of Flatiron Health was USD 1.9 billion on a fully diluted basis, subject to certain adjustments. Flatiron Health will continue its operations as a separate legal entity and will maintain its current business model, network of partnerships and overall objectives. The integrity of patient protected health information will be preserved, as will dedicated sales and marketing, providerfacing and life science business activities.
Source: Roche Media Release, 6 April, 2018.

Boehringer Ingelheim and OSE launch up-to$1.4B+ immunooncology partnership

Boehringer Ingelheim has committed a potential $1.4 billion-plus toward cancer immunotherapy by partnering with OSE Immunotherapeutics to develop its latepreclinicalstage candidate OSE-172 (Effi-DEM), a novel checkpoint inhibitor antibody designed to treat solid tumours.
Source:, April 5, 2018.

Kite founder’s new firm inks off-the-shelf CAR T deal with Pfizer

Newly formed company Allogene Therapeutics has teamed with Pfizer to develop allogeneic CAR T-cell therapies. The asset contribution agreement will see Allogene take over the development of Pfizer’s CAR-T Cell therapy portfolio, including UCART19, the lead candidate in its pact with Cellectis and 16 preclinical assets.
Source:, April 3, 2018.

Fujifilm acquiring Irvine Scientific and IS Japan for $800M, with cell culture media growth in mind

Fujifilm said it intends to expand its ability to develop and manufacture biopharmaceuticals and regenerative medicine treatments by acquiring a pair of cell culture media businesses owned by Japan-based JXTG Holdings for a combined $800M. Fujifilm plans to acquire all outstanding shares of Irvine Scientific (ISUS) and IS Japan (ISJ) and incorporate the businesses into its existing contract development manufacturing organization (CDMO) unit Fujifilm Diosynth Biotechnologies.
Source:, March 29, 2018.

Novartis: ‘Time is right’ for $13bn consumer health sale to GSK

Novartis will sell its stake in a consumer healthcare joint venture to partner GSK and plans to invest the $13bn into its core pharmaceutical businesses. As part of an asset exchange in 2014, Novartis and GSK combined their over-the-counter drugs and nutritional businesses to form a consumer health joint venture. Four years on, GSK agreed to Pay $13B to buyout Novartis 36% stake in the venture.
Source:, March 27, 2018.

Mundipharma goes up against MSD with Herceptin biosimilar in Europe

Mundipharma said it is not concerned at arriving second to market with a trastuzumab biosimilar after inking a deal to commercialise Celltrion’s Herzuma in seven European countries.
Source: March 20, 2018.

Lundbeck to acquire Prexton Therapeutics for up-to-$1.1B

Lundbeck has agreed to acquire Prexton Therapeutics for up to $1.1B the companies said, in a deal that adds a Phase II Parkinson’s disease candidate to the buyer’s pipeline focused on neurological as well as psychiatric disorders.
Source:, March 16, 2018.

Pfenex hunting partners to advance Lucentis and Neulasta biosimilar candidates

Pfenex said it is actively looking for partners for its Lucentis and Neulasta biosimilar programmes, which are presently on hold.
Source:, March 16, 2018.

Flexible friend: Sanofi talks capacity benefits of Lonza joint venture

Sharing a facility with a CMO gives Sanofi the flexibility to adjust biomanufacturing capacity according to its needs, said its senior vice-president for biologics. Sanofi plans to construct a large scale mammalian cell culture manufacturing site in Visp, Switzerland in partnership with the CMO Lonza.
Source:, March 16, 2018.

Biogen buys Pfizer neuropsychiatry candidate for up-to-$590M+

Biogen said it will expand its neuroscience pipeline into neuropsychiatry by agreeing to acquire from Pfizer the Phase II-ready cognitive impairment associated with schizophrenia (CIAS) candidate PF-04958242 for up to $590 million-plus.
PF-04958242 is an α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptor potentiator designed to facilitate neurotransmission. AMPA receptors mediate fast excitatory synaptic transmission in the central nervous system, a process that can be disrupted in schizophrenia and other neurological and psychiatric diseases. Biogen said it plans to launch a Phase IIb trial of PF-04958242 in the second half of 2018.
Source:, March 12, 2018.

First Herceptin biosimilar launched in UK

Merck & Co. will take on Roche as Ontruzant becomes the first biosimilar of cancer drug Herceptin (trastuzumab) to be launched in Europe.
Source:, March 9, 2018.

Merck & Co. and Eisai launch up-to-$5.76B cancer partnership

Merck & Co. will partner with Eisai to codevelop its marketed cancer treatment Lenvima® (lenvatinib mesylate) for additional oncology indications, both alone and in combination with Merck’s Keytruda® (pembrolizumab), through a collaboration that could generate up to $5.76B for Eisai, the companies said today.
The value of the collaboration represents potentially the second most valuable immuno-oncology partnership ever launched the second time in as many months that has occurred. The announcement comes two months after the companies won the FDA’s Breakthrough Therapy designation for a Lenvima/Keytruda combination in advanced and/or metastatic renal cell carcinoma (RCC).
Source:, March 8, 2018.

Roche teams with Emulate for organon-chip testing tech

Roche will use Emulate’s Human Emulation System to better predict the safety and efficacy of its drug candidates. Emulate has previously forged deals with other big pharma firms over its preclinical platform, including Merk & Co., J&J, Takeda and Pfizer.
Source:, Feb 27, 2018.

AbbVie, Voyager launch up-to-$1.1B+gene-therapy collaboration to treat Alzheimer’s, neurodegenerative diseases

AbbVie will partner with Voyager Therapeutics to develop and commercialize gene therapies for Alzheimer’s disease and other neurodegenerative diseases, through a collaboration that could generate more than $1.1B-plus for Voyager, the companies said. The collaboration is intended to create one-time treatments by combining AbbVie’s monoclonal antibody expertise, global clinical development and commercial capabilities with Voyager’s gene therapy platform. That platform is designed to generate adeno-associated viral (AAV) vectors that deliver monoclonal antibodies directed against accumulations of tau protein within the brain associated with impaired brain function and neuronal cell loss.
Source:, Feb 20, 2018.

Astellas adding ‘off-the-shelf’ stem cell tech in $102.5M acquisition

Japan’s Astellas Pharma will bolster its regenerative medicine arm through the acquisition of pluripotent stem cell maker Universal Cells. The Seattle-based stem cell maker will become a subsidiary operating under Astellas’s institute for Regenerative Medicine (AIRM). Astellas will have direct access to Universal Cells’ donor technology, used to create cell therapy products that do not require Human Leukocyte Antigen (HLA) matching which the firm says dramatically reduces the risk of rejection.
Source:, Feb 20, 2018.

Roche expands in personalized medicine and oncology with $1.9B purchase of Flatiron Health

Roche expanded its presence in personalized medicine and oncology by the $1.9B acquisition of Flatiron Health, a healthcare technology and services company whose offerings are designed to support cancer care providers and life science companies. Flatiron Health has developed the OncologyCloud™ software suite, whose components include OncoAnalytics® for deep clinical and business insights, OncoEMR® for electronic medical record and workflow software, OncoBilling® for claims filing, and SeeYourChart® for sharing laboratory tests and other data directly with patients.
Source:, Feb 16, 2018.

Cellectis wins patents for CRISPR gene editing in T-cells

The French allogeneic T-cell developer said CRISPR/Cas9 technology is an “easy-to-use and cheap tool” for gene editing and plans to license the patents to others. The firm plans to make the newly granted patents available for licensing to companies that want either to use CRISPR technologies for engineering allogeneic chimeric antigen receptor (CAR) T-cells while suppressing genes involved in checkpoint inhibitors, or to insert a DNA CAR construct by gene targeting a specific locus in the genome of T-cells.
Source:, Feb 14, 2018.

BMS and Nektar Ink up-to-$3.6B immuno-oncology combination therapy collaboration

Bristol-Myers Squibb (BMS) will partner with Nektar Therapeutics on a combination cancer immunotherapy collaboration that could generate up to $3.6B-plus for Nektar, potentially the second-most-valuable immuno-oncology partnership ever launched. The companies agreed to co-develop Nektar’s lead immuno-oncology program NKTR-214 in combination with BMS’ marketed cancer immunotherapy Opdivo® (nivolumab), as well as a combination of NKTR-214 with both Opdivo and BMS’ other marketed cancer immunotherapy blockbuster drug, Yervoy® (ipilimumab), in more than 20 indications across nine tumour types.
Source:, Feb 14, 2018.

Short-acting to extended half-life, Ireland signs exclusive haemophilia deal

The Republic of Ireland will move its population from short-acting to extended half-life haemophilia therapies (Elocta), under an exclusive two-year deal between the Health Services Executive (HSE) and Sobi. The deal follows an earlier contract agreement for the supply of Alprolix indicated to treat haemophilia B.
Source:, Feb 14, 2018.

Charles River to buy MPI Research for $800m

Charles River Laboratories International said it is en route to ‘double its size in five years’ with the acquisition of non-clinical CRO MPI Research. The $800M acquisition will add drug safety, testing and discovery services to Charles River offerings as well as a 1 million sq. ft. facility in Michigan, US.
Source:, Feb 13, 2018.

Denali signs Lonza to make biologics in first CMO deal

Lonza has inked a biologics deal with Denali to make neurodegenerative disease therapies, offering the Californian firm opportunity to have production “on their back doorstep.” The partnership covers all stages of development manufacturing for clinical trials, for diseases such as Alzheimer’s, Parkinson’s and Amyotrophic Lateral Sclerosis (ALS). Lonza Group will also be responsible for delivering finished products for patients, overseen by its in-house Drug Product Services in Basel, Switzerland.
Source:, Feb 12, 2018.

UK collaboration to manufacture commercial viral vectors

Cobra Biologics and Symbiosis have received a grant to establish a commercial viral vector manufacturing facility in the UK. The $2.5M innovate UK grant was awarded as part of a collaboration aiming to support the development of gene therapies in the UK.
Source:, Feb 9, 2018.

Charles River gains access to AstraZeneca’s lead discovery infrastructure

Charles River has been granted commercial access to AstraZeneca’s high-throughput screening (HTS) and compound management infrastructure, an investment that will help the company “deliver optimal benefit” to its customers. As part of the agreement, Charles River scientists-inresidence will perform HTS programs for clients at AstraZeneca’s Centre for Lead Discovery in Cambridge, UK.
Source:, Feb 5, 2018.

Oncosec tech to deliver genetically engineered proteins directly into tumours

Avacta said a research collaboration using Oncosec’s gene delivery technology could lead to the development of gene delivered Affimer immunotherapies. The partnership teams Avacta’s Affimer protein platform, based on a small human protein that can be engineered to bind with high specificity to a wide range of protein targets, with Oncosec’s ImmunoPulse technology, to deliver DNA encoded proteins directly into animal model tumours. The collaboration includes Avacta’s PD-L1 inhibitor (AVA04).
Source:, Jan 29, 2018.

Sanofi to buy Ablynx for €3.9B, leaving Novo Nordisk in the cold

Sanofi agreed to acquire its antibodyfragment tech partner Ablynx for €3.9B. The deal is designed to expand the buyer’s R&D pipeline and rare blood disorders portfolio with a late-stage candidate and its underlying technology based on therapeutic proteins or “Nanobodies.”
Source:, Jan 29, 2018.

J&J Q4: pricing pressures drive 9% drop in US Remicade sales

J&J has reported its largest quarterly decline in US Remicade (infliximab) sales, but said demand remains stable amid biosimilar competition. While total pharmaceutical sales grew 17.6% year-on-year to $9.7B, the firm reported another quarter of declining sales of one-time-top-selling biologic Remicade (infliximab). In the US, J&J has faced over a year biosimilar competition from Pfizer’s Inflectra, and saw a second infliximab biosimilar (Renflexis) marketed by Merck & Co. enter the market in July 2017.
Source:, Jan 26, 2018.

Biogen looking to up its stake in Samsung biosimilar joint venture

Biogen reported record biosimilar revenues and said it intends to increase its share in Samsung Bioepis. For the Q4 2017, Biogen generated $122M in sales of biosimilar products, up 21% on Q3 and up 130% on the same period last year. For 2017 biosimilar sales stood at $380M, a 277% increase on 2016. This was due to the recent approvals and launches of biosimilar products by Samsung Bioepis, a joint venture between Biogen and Korean manufacturer Samsung BioLogics formed in 2012. Biogen owns approx. 9% of Samsung Bioepis, but can increase its share to 49.9% under terms of the deal.
Source:, Jan 26, 2018.

Eurofins adds radiolabelling service with CRO acquisition

Eurofins Scientific has incorporated radiolabelling services and boosted drug discovery capabilities following the acquisition of UK CRO Selcia. Under the agreement, the analytical testing firm will take on Selcia’s 2,500m2 research and development laboratories in Ongar, UK, where it houses its medicinal chemistry and peptidyl prolyl isomerease (PPIase) inhibitor screening platform. Eurofins will also integrate the contract research organization’s radiolabelling capabilities to test compounds metabolism and utilization.
Source:, Jan 25, 2018.

Sanofi signs with IONTAS to discover antibodies using mammalian display tech

Sanofi and IONTAS have signed an agreement through which the company will use IONTAS’ proprietary technology, which aims to bridge the gap between a “great lead” and “poor developability”. IONTAS generates antibodies with favourable blinding and expression properties using its Mammalian Display Antibody Discovery Platform. The platform enables the construction of cell line libraries, where each display a different IgG-formatted antibody on the cell surface.
Source:, Jan 25, 2018.

Janssen expands multi-year hit identification deal with CRO ChemDiv

Janssen and ChemDiv have expanded their multi-year hit identification collaboration to find potent anti-infective drug candidates. The companies have expanded the discovery chemistry assets available to the collaboration to include a number of novel compounds built on the basis of recent ChemDiv MedChem and synthetic design research in the anti-infective arena.
Source:, Jan 25, 2018.

Shortages and soaring generic prices prompt US hospitals to form not-forprofit drug business

Drug shortages and rising medicine prices in the US have driven a group of hospitals to form a not-for-profit generic drug manufacturing firm. Five associations representing more than 450 hospitals in the US include Intermountain Healthcare, Asension, SSM Heath and Trinity Health, in consultation with the US department of Veterans Affairs. The associations will consider what high-capacity contract manufacturing organisation capacity is available and where, when determining whether the not-for-profit will make generic drugs in-house or outsource manufacturing to a third party.
Source:, Jan 25, 2018.

GE wins another FlexFactory fit out in ‘big market’ China

Clover Biopharmaceuticals will adopt GE Healthcare’s centrally automated FlexFactory platform at its facility in Zhejiang, China where it plans to make a biosimilar to Amgen’s Enbrel. The site biomanufacturing technologies will include two 200L lines. The facility is expected to be operational in the second half of 2018.
Source:, Jan 24, 2018.

Sanofi to pay $11.6B for Bioverativ for haemophilia biz and commercial network

Sanofi agreed to buy Bioverativ Inc. for $11.6B for haemophilia business and to help develop its candidate RNAi therapeutic fitusiran. Bioverativ split from Biogen Inc. last year with the idea the new firm would focus on discovering, researching, developing and commercializing treatments for haemophilia and other blood disorders.
Source:, Jan 23, 2018.

Celgene buying T-cell tech partner Juno for $9B

Celgene will add chimeric antigen receptor (CAR) and T-cell receptor (TCR) technologies through the acquisition of its immune-oncology partner Juno Therapeutics. The firms have been working together since July 2015 when they entered into a 10-year immunotherapy partnership, valued at around $1B.
Source:, Jan 23, 2018.

European Patent Office intends to grant BioInvent patent relevant to its F.I.R.S.T.TM platform

BioInvent International AB announced that the European Patent Office, has communicated its intention to grant the company a patent relevant to its unique, function-based F.I.R.S.T.TM platform. More precisely, the patent builds on earlier F.I.R.S.T.TM patents, extending protection to combined use of differential biopanning and high throughput sequencing, such as NGS in identification of antibodies to low expressed cell surface antigens. The European patent should be granted once all remaining administrative actions have been completed by BioInvent.
Source: BioInvent press release, Jan 17, 2018.

Google Ventures and others back universal flu vaccine firm Vaccitech

Vector-based vaccine tech firm Vaccitech has secured $27.1M in venture capital with investors Google Ventures, Sequoia China, Neptune Ventures and Oxford Sciences Innovation. The Oxford University spin-out explained the funding will be used to develop its portfolio of vaccines, which includes a universal influenza vaccine in Phase II development.
Source:, Jan 17, 2018.

Sweden’s Xbrane eyes partnership to bring Lucentis biosimilar to China

Swedish drug maker Xbrane Biopharma said it is teaming with CR Pharma to bring its Lucentis (ranibizumab) biosimilar to China. Xbrane has signed a non-binding letter of intent with the Chinese pharmaceutical firm CR Pharma for the development of Xlucane, its biosimilar candidate to Roche’s blockbuster macular degeneration (AMD) drug Lucentis (ranibizumab).
Source:, Jan 16, 2018.

Takeda to Acquire TiGenix for €520M

Takeda Pharmaceutical said it has agreed to acquire stem cell therapy developer TiGenix for up to €520M in a deal designed to strengthen the buyer’s late-stage gastroenterology pipeline and presence in the U.S. specialty care market. The deal builds upon a partnership by the companies, through which TiGenix sold to Takeda ex-U.S. licensing rights to develop and commercialize Cx601 (darvadstrocel), an up-to-€39M deal signed in July 2016.
Cx601 is a suspension of allogeneic expanded adipose-derived stem cells. Last month, Cx601 became the first allogenic stem cell therapy to receive a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). That recommendation is expected to be the basis for a marketing authorization by the EC that is expected in the first half of 2018.
Source:, Jan 5, 2018.

Thermo Fisher and Juno sign sevenyear CAR-T tech deal

Juno Therapeutics will use Thermo Fisher Scientific’s Cell Therapy Systems (CTS) technology to develop CAR T therapies for cancer treatments. The agreement will supply Juno with CTS Dynabeads CD3/CD28 for 7 years in a nonexclusive licensing deal. The magnetic beads activate and expand T cells specially designed to identify and combat cancer cells in patients.
Source:, Jan 3, 2018.

European Commission News

EU Commission launches newvaccination proposals

The proposals aim to boost cooperation among member states in the vaccines field, reduce vaccine-preventable diseases which claim the lives of up to 3 million people every year, and combat the rise of fake news and disinformation against vaccination. With the new vaccination proposals, scientists have a good opportunity to join forces and use IT tools to explain their scientific opinions to people, EU Health Commissioner Vytenis Andriukaitis told in an interview.
Source: EuropaBio Newsletter 21-27 April, 2018.

Lilly wins EU approval for psoriatic arthritis mAb

Eli Lilly’s arthritis monoclonal antibody Taltz (ixekizumab) has been approved for a second indication in Europe. The EC granted marketing authorisation for Taltz a treatment for active psoriatic arthritis in adult patients who are intolerant to diseasemodifying anti-rheumatic drug therapies.
Source:, Jan 30, 2018.

EC publishes draft revision of sterile drug GMP guidelines

The EC has published a draft revision of its GMP guidelines on the production of sterile drugs with tweaks to recommendations on clean rooms, sealing technologies and single-use systems. The document addresses a number of issues that have emerged in recent years e.g. it provides information on the qualification of cleanroom that take revisions to ISO 14644 standards into account. For the draft revision see:
Source:, Jan 3, 2018.

Regulatory News

International collaboration on GMP inspections reduces burden on API makers, says JM

The European Medicines Agency (EMA) issued a report in conjunction with national authorities of 9 European and non-European countries (i.e., US, Canada, Japan and Australia) as well as the European Directorate for the Quality of Medicines (EDQM) and the World Health Organisation (WHO). The report contains results from a six-year international active pharmaceutical ingredient (API) inspection programme that the ingredient Johnson Matthey (JM) says benefits companies and authorities.
For the report see:
Source:, April 25, 2018.

Positively Europe: Thumbs up for Amgen & Sandoz biosimilars

The EMA has recommended the approvals of a third Herceptin (trastuzumab) biosimilar ABP980 made by Amgen and Sandoz’s version of Remicade (infliximab). If the EC approves ABP980 it will become the third biosimilar version of Roche’s bestselling monoclonal antibody.
Source:, March 27, 2018.

Takeda’s off-the-shelf stem cell therapy approved in Europe

Alofisel (darvadstrocel) has become the first allogeneic stem cell product to be approved in Europe. The EC granted approval of Alofisel, previously known as Cx601, for the treatment of complex perianal fistulas in adult patients with Chrohn’s disease.
Source:, March 27, 2018.

Biosimilar buffer? EU thumbs up for Amgen’s Neulasta delivery device

The EMA has recommended approval of Amgen’s on-body injector device to be used with its biosimilar-threatened Neulasta (pegfilgrastim).
Source:, March 7, 2018.

US budget boost for FDA and generic competition

An FDA advocacy group says an extra $473M of funding in the US 2019 budget request represents “an enormous vote of confidence” in the agency. The budget also looks to tackle high drug pricing in the US through a series of measures, including by giving the FDA greater ability to bring genetics to the market faster by incentivizing more competition among manufacturers.
Source:, Feb 15, 2018.

IQVIA awarded FDA contract to expand biologic safety monitoring

IQVIA has been contracted to expand the safety and efficacy monitoring of biologic products as part of a novel alliance with the US Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER). As part of the agreement, IQVIA will monitor and assist the evaluation of safety and effectiveness of CBER-regulated vaccines, blood products and other biologics. The agreement also marks the launch of CBER’s Effectiveness and Safety (BEST) initiative.
Source:, Feb 7, 2018.

US FDA to take risk-based approach to biomanufacturing inspections

A risk-based approach to inspection regulations could result in increased audits of more complex manufacturing sites, the US FDA said. The final rule coming into force on June 2018 will affect how often the FDA is inspecting certain facilities and will implement a risk-based schedule replacing the biennial inspection requirement for drug and biological product establishments required in the Federal Food Drug and Cosmetic Act and aligning with the Food and Drug Administration Safety and Innovation Act.
Source:, Jan 26, 2018.

Benchmark for tracking industry efforts to fight antimicrobial resistance launched at WEF in Davos

The Access to Medicines Foundation has launched a ‘benchmark’ that maps drug industry efforts to stem the spread of drug resistance at the World Economic Forum in Davos, Switzerland. The Antimicrobial Resistance Benchmark was developed as an independent and public tool for measuring how pharmaceutical companies are responding to antimicrobial resistance. For the benchmark see:
Source:, Jan 23, 2018.

Success for Amgen as Europe welcomes first Avastin biosimilar

The EC has approved Mvasi, Amgen and Allergan’s biosimilar version of Roche’s cancer drug Avastin (bevacizumab). The biosimilar was co-developed by Allergan and Amgen, but will be marketed in the EU by Amgen.
Source:, Jan 22, 2017.

Room for another: Novartis files Humira biosimilar in US

Novartis subsidiary Sandoz announced that it has submitted for US FDA review its biosimilar to Abbvie’s bestselling Humira (adalimumab). If successful it would be the third biosimilar approval for Sandoz following Zarxio, a version of Amgen’s Neupogen approved in 2015, and its Enbrel biosimilar Erelzi, approved in 2016. It would also become the third biosimilar approved for Humira after Amgen’s Amjevita and Boehringer-Ingelheim’s Cyltezo. Due to patent issues no adalimumab is available in the US and a long-standing legal battle between Amgen and Abbvie concluded with a January 2023 launch date for Amjevita. By then there could be many more adalimumab biosimilars approved.
Source:, Jan 16, 2018.

Roche’s OCREVUS (ocrelizumab) approved in the European Union for relapsing forms of multiple sclerosis and primary progressive multiple sclerosis

Roche announced that the EC has granted marketing authorisation for OCREVUS® (ocrelizumab) for patients with active relapsing forms of multiple sclerosis defined by clinical or imaging features and for patients with early primary progressive multiple sclerosis in terms of disease duration and level of disability, and with imaging features characteristic of inflammatory activity.
Source: Roche press release, Jan 12, 2018.

Infliximab monograph adopted by European Pharmacopoeia Commission

The European Pharmacopoeia Commission has adopted the first “Infliximab concentrated solution” monograph for a monoclonal antibody. The newly adopted Infliximab monograph will be included in the European Pharmacopoeia Supplement 9.6 and will become effective in January 2019.
Source:, Jan 10, 2018.

A closer look at orphan medicines in Europe

The European Medicines Agency’s new factsheet provides an updated overview of the EU’s orphan designation programme. In more concrete terms, it explains how the programme works, what a rare disease is, and what incentives are made available to developers. For the factsheet see:
Source: EuropaBio Newsletter 16 Dec-5 Jan, 2018.

US FDA approves first gene therapy for inherited disease

US FDA has approved Spark Therapeutics’ gene therapy Luxturna™ (voretigene neparvovecrzyl) for treating children and adults with the rare inherited blindness disorder biallelic RPE65 mutationassociated retinal dystrophy. Approval of the one-time adeno-associated virus vector (AAV)-delivered gene therapy, which FDA Commissioner S. Gottlieb described in a statement as “a milestone that reinforces the potential of this breakthrough approach in treating a wide-range of challenging disease,” marks the first gene therapy approval for a genetic disease. Spark has not confirmed the cost of treatment, although some commentators are anticipating a $1M per patient price tag. Luxturna is currently under review by the European Medicines Agency.
Source:, Dec 20, 2017.

Clinical Trial News

Entire clinical research model at risk: ‘Call to action has to be now’

The industry’s current research model is failing patients who live in a mobile world and will not sustain if clinical trials continue to take years to recruit, says industry expert. Many oncology trials are recruiting at a number of half a patient per site per month and cost $2-3B to get to the market. Compounding recruitment challenges, 30% of the millennial generation doesn’t have a primary care physician and they are expected to overtake Baby boomers as America’s largest generation by 2019. Instead, the digitally generation received health care through ambulatory clinicians or telehealth. As such these patients are lost to participate in clinical trials as they don’t fit into the industries current system.
Source:, April 18, 2018.

PharmaCyte looks to revolutionize cancer treatment with planned Ph. IIb trials

PharmaCyte’s live-cell encapsulation technology could change the way solid tumour cancers are treated, as the biotech moves forward with CRO selection ahead of its Ph IIb clinical trial which is expected to cost between $15 and $17M. By encapsulating genetically engineered human cells that activate an inactive chemotherapy drug, a conversion that normally occurs in the liver, the company hopes it can provide targeted cancer treatment with few or no treatment side effects. The capsules are implanted as close as possible to the patient’s tumour site, where they work as little factories to activate the chemotherapy drug.
Source:, April 12, 2018.

Pfizer joins the TriNetX network to ‘harness’ real world data

Pfizer will recruit patients with rare diseases and identify clinical trial sites with support from the TriNetX global health research network, which is becoming “the de facto platform” for research. TriNetX recently announced a new algorithm designed to further support clinical patient identification. Currently the TriNetX is working with more than 25 of the top pharmaceutical companies.
Source:, April 10, 2018.

Novartis: Decentralized trials could expand enterprise around the world

Novartis hopes to eventually recruit ‘a significant portion’ of patients into a decentralized clinical trial model that has the potential to increase the company’s global reach. Novartis and Science 37 announced an expanded alliance through which the companies will launch up to 10 decentralized clinical trials across the US.
Source:, March 12, 2018.

Researchers ‘very surprised’ by scale of errors in clinical trial registries

Nearly one-sixth of clinical trials registered in both the US and EU have discrepancies in their completion status, errors which undermine trust in the industry and medicine, say researchers. Researchers examined 10,492 clinical trials registered on both and the EU Clinical trial register (EUCTR) for completion and reporting status discrepancies. According to the study 16.2% of dual registered trials had discrepant completion status and 33.9% of trials listed as ongoing on EUCTR were listed as completed on
Source:, March 7, 2018.

New guidelines look to improve the use of patient feedback in clinical trials

Researchers have published new international guidelines for the inclusion of patient-reported outcomes in clinical trial protocols with the goal of providing high quality evidence and enhancing patient care. The new international guidelines SPIRIT-PRO checklist were developed by a team of researchers from the University of Birmingham and the University of Sydney, in collaboration with various international stakeholders, including patients. For the guidelines see:
Source:, Feb 20, 2018.

Boehringer Ingelheim joins SCRS as Global Impact Partner

Boehringer Ingelheim has joined the Society for Clinical Research Sites as a Global Impact Partner (GIP) to help identify new approaches to working with sites. SCRS is a global trade organization representing more than 9000 clinical research sites in 47 countries. The organizations GIP platform provides a platform for sponsors, contract research organizations and vendors to engage with SCRS and its members.
Source:, Feb 19, 2018.

Framework outlines consistent approach to managing R&D outsourcing

IAOCR and PCMG have released a new set of standards to establish a consistent approach to managing clinical outsourcing to ensure clinical trials have a better chance of running optimally, said co-founder. The standards provide a competency framework for clinical outsourcing managers and were introduced to ensure those in this role have the abilities to do their job competently.
Source:, Feb 12, 2018.

New trial design guidelines address ‘inappropriate’ patient exclusion

Exclusion of brain metastasis patients from clinical trials of anti-cancer drugs could remove up to two-thirds of the stage IV population said researchers who have published new guidelines aimed at bringing attention to opportunities for improvement. The new guidelines published in Lancet Oncology were developed by the response assessment in neuro-oncology brain metastases (RANO-BM) group and describe how to address cancer patients with CNS involvements in clinical trials of anti-cancer drugs. For the guidelines see:
Source:, Jan 15, 2018.

Research News

Human brain organoids thrive inmouse brains

Scientists report a new approach that can develop more sophisticated organoid models by ensuring they receive sufficient oxygen and other nutrients via transplantation into rodents. The work could yield insights into the development of cures for brain disorders, speed up the testing of drugs, and even pave the way for someday transplanting healthy populations of human cells into people’s brains to replace damaged or dysfunctional tissue. By transplanting human brain organoids into mouse brains, scientists based at the Salk Institute hope to develop models that better represent the complexity of the brain. And the scientists have already achieved a dramatic first: an organoid that is both infiltrated by blood vessels and is also nourished by the blood flowing through these vessels.
The Salk scientists sought to replicate a more supportive physiological environment by grafting human stem cell–based organoids into a blood vessel–rich area of the mouse brain. The grafted human organoids integrated into the host environment, formed both neurons and astrocytes, and were surveyed by immune cells. Significantly, the team saw not only native blood vessels, but vessels with blood flowing through them. The team divided each organoid in half before transplantation, and the scientists maintained one of the halves in culture, so they could directly compare the benefit of both environments. They found that the cultured halves were filled with dying cells after a few months, while the age-matched organoids in the rodents were healthy. To find out if the transplanted organoids were functional as well as healthy, the team conducted calcium imaging tests, in which neurons produce a dye when they fire. And, indeed, the neurons within the organoids were firing in a synchronized way. The work was published in Nature Biotechnology.
Source:, April 17, 2018.

The ‘gold standard’ of preclinical research yields ‘highly unreliable’ results, study finds

Standardization in preclinical animal studies may be a cause of poor reproducibility making translation to humans in clinical trials difficult, if unlikely, say researchers. The aim of the study was to examine the extent to which standardization may be a problem in preclinical animal research and whether a multi-laboratory study could be a solution: For the publication see:
Source:, Feb 27, 2018.

Hepatitis B infects organ-on-a-chip platform

For the first time, a real pathogen has instigated a real disease process in an artificial organ. As far as scientists are concerned, the whole point of an artificial organ in this case, a liver-on-a-chip platform is to simulate a real organ’s physiology, and that includes pathophysiology. By using an artificial liver to model the interactions between human tissue and an infectious agent in this case, hepatitis B scientists based at Imperial College London have opened a new front in disease research and, potentially, the development of new drugs. Hepatitis B virus (HBV) is currently incurable and affects over 257 million people worldwide. Development of a cure has been slow because there is no model system in which to test potential therapies. However, the Imperial team showed that liver-on-achip technology originally developed at MIT, the University of Oxford, and biotechnology company CN Bio Innovations could be infected with hepatitis B virus.
Essentially, the scientists demonstrated that their 3D microfluidic primary human hepatocyte system could be infected with HBV at physiological levels, and that it responded to the virus much like a real human liver, exhibiting immune cell activation and other markers of infection. Details of the work appeared in the journal Nature Communications.
Source:, Feb 15, 2018.

Huntington siRNA highly toxic to cancer cells

There is no upside to Huntington’s disease (HD), as the genetic illness is fatal and causes the breakdown of nerve cells in the brain. However, if one were searching for some positive aspect, HD patients are an anomaly with respect to contracting cancer with 80% fewer cases than the general population. A team of investigators at Northwestern University Feinberg School of Medicine has discovered why HD is so toxic to cancer cells and are harnessing it as a novel approach to treat cancer.
HD is caused by an overabundance of a certain type of repeating RNA sequences in one gene, huntingtin, present in every cell. The defect that causes the disease is also highly toxic to tumour cells. These repeating sequences in the form of so-called small interfering RNAs (siRNAs) attack genes in the cell that are critical for survival. Nerve cells in the brain are vulnerable to this form of cell death. However, cancer cells appear to be much more susceptible.
In order to test the “super-assassin” molecule in a treatment scenario, the Northwestern team delivered the molecule in nanoparticles to mice with human ovarian cancer. The treatment significantly reduced the tumour growth with no toxicity to the mice. More importantly, the tumours did not develop resistance to this form of cancer treatment. Additionally, the research team used the siRNA molecule to treat human and mouse ovarian, breast, prostate, liver, brain, lung, skin, and colon cancer cell lines. They found that the molecule killed all cancer cells in both species. The study was published in EMBO Reports.
Source:, Feb 12, 2018.

Shields up for new cancer genetherapy virus

The promise of gene therapy to treat cancer has always been limited by the vector used to deliver the rectified genetic payload. While off-targeting has always been a major concern, no less of an issue is the host immune system destroying the therapeutic virus before it has had a chance to complete its mission. Now, investigators at the University of Zurich have developed a vector they believe will dramatically increase the efficiency of cancer gene therapy using human adenovirus 5.
Until recently, the use of adenoviruses in tumour therapy has been very limited. They lack the ability to infect cancer cells and therefore cannot inject the genetic blueprints for the therapeutic molecules to fight the disease. Moreover, adenoviruses are efficiently neutralized by the immune system and very rapidly eliminated by the liver. This new study shows how researchers succeeded in rebuilding these viruses so that they effectively recognize and infect tumour cells. For this purpose, the investigators created molecules that act as an adapter between the virus and the tumour cell. The adapters, which cling very tightly to the coat of the virus, can depending on their version bind to different surface molecules on the tumour cell. The scientists tested adapters for several receptors such as human epidermal growth factor receptor 2 (HER2) and epidermal growth factor receptor (EGFR), which are present on several types of cancer cells. Only viruses equipped with these adapters could infect the tumour cells. The researchers subsequently hid the virus under a novel protein coat, which serves as camouflage for the virus and which protects it from the immune system. As a basis for this shield, the researchers used an existing antibody that they redesigned.
Interestingly, the shield not only protects the redesigned virus from the immune cells but also prevents the virus from being eliminated by the liver, which normally quickly removes unmodified adenoviruses from the bloodstream, often making therapeutic applications impossible. With its shield and its adapter, these viral gene shuttles efficiently infected tumour cells in laboratory animals. The findings were published in Nature Communications.
Source:, Jan 31, 2018.

NIH commits $190M to somatic geneediting tools/tech research

The NIH plans to award approximately $190M over six years to researchers developing tools and technologies designed to enable somatic cell genome editing in humans. Researchers funded through the Somatic Cell Genome Editing program are expected by the NIH to assemble the tools and tech they develop into a genome editing “toolkit” of knowledge, methods, and tools, all to be shared with the scientific community.
The program will be funded through NIH’s Common Fund, and managed by a transagency Working Group representing multiple NIH Institutes and Centers, led by the National Center for Advancing Translational Sciences. Because the program is focused on non-reproductive somatic cells, any changes to the DNA wrought by the genome editing tools and tech will not be inherited avoiding the ethical qualms raised by permanent germline editing of human genomes.
Somatic cell genome editing is among applications of CRISPR (clustered regularly interspaced short palindromic repeats) technology to be explored by The CRISPR Journal, an international, multidisciplinary peer-reviewed journal being launched this year by Mary Ann Liebert Inc.
Source:, Jan 24, 2018.

Novel biomaterials boost T-cell production

Emerging cellular therapies require effective platforms for producing clinically relevant numbers of high-quality cells. Columbia University engineers say they have developed a novel technique for improving T-cell manufacture by focusing on the bioprocessing materials.
The engineers developed a materials-based approach to improving expansion of T cells, a compelling agent for treatment of cancer and a range of other diseases. The system consists of electrospun fibers, which present activating antibodies to CD3 and CD28.
These fibers are effective in activating T cells, initiating expansion, and simplify processing of the cellular product, compared to current bead-based platforms. In addition, reducing the mechanical rigidity of these fibers enhances expansion of mixed populations of human CD4+ and CD8+ T cells, providing eightfold greater production of cells in each round of cell growth.
According to the engineers their system provided nearly an order of magnitude more cells in a single process. What’s especially exciting is that they have been able to expand cells isolated from patients undergoing treatment for leukaemia. These cells are often very difficult to activate and expand, and this has been a barrier to using cellular immunotherapy for the people who need it.
According to the researchers, other cell types can sense the mechanical stiffness of a material. For example, the rigidity of a material used to culture stem cells can direct differentiation, with a softer material promoting production of neuron, while a stiffer substrate encourages bone cell differentiation. This effect can be as strong as the chemicals normally used to direct differentiation. However, a similar effect was unexpected in T cells for activation. The approach was published in Advanced Biosystems.
Source:, Jan 18, 2018.

3D bioscaffold mimics antigenpresenting cells for improved T-cell expansion

Researchers in the U.S. have developed a 3D biomaterial scaffold that can promote much faster ex vivo expansion of functional T cells than currently available technologies. The new scaffold is designed to provide the cues that are delivered to T cells by the body’s own antigen-presenting cells (APCs), in context, and so better mimic in vivo signalling by APCs. Led by David Mooney, Ph.D., at Harvard’s Wyss Institute for Biologically Inspired Engineering and John A. Paulson School of Engineering and Applied Sciences, the team demonstrated how the technology dramatically increased the ex vivo expansion of both primary mouse and human T cells, as well as of cancer-targeting chimeric antigen receptor (CAR) T cells, which they successfully tested in a mouse model of lymphoma. The researchers project that the technology could broaden the utility of immunotherapeutics, including adoptive cell transfer (ACT). The technology was reported in Nature Biotechnology.
Source:, Jan 16, 2018.

Blood-vessel-on-a-chip sheds light onanti-inflammatory drug candidate

A collaborative team of researchers from the Division of Haemostasis and Thrombosis at Beth Israel Deaconess Medical Center and the Wyss Institute at Harvard University have discovered small molecules called parmodulins that provide anti-inflammatory and antithrombotic protection to endothelial cells without interfering with blood clotting, making them attractive new drug candidates.
Activated protein C (APC) is a naturally occurring anticoagulant protein with antiinflammatory and other protective effects that has been used medically to treat severe blood infections and wounds. However, its use is limited because its inhibition of thrombin also impacts the blood’s ability to clot, increasing bleeding risk. These newly discovered parmodulins compounds are similar in many respects to APCs. The work was enabled by leveraging the Institute’s Organ-on-a-Chip technology to model coagulation within a human blood vessel in vitro.
The discovery of an anti-inflammatory molecule that prevents endothelial thrombosis, but also preserves normal blood coagulation is a major step toward an alternative and better approach to treating inflammatory disease. The study was published in Proceedings of the National Academy of Sciences.
Source:, Jan 16, 2018.

Stem Cell News

Controllable “Swiss army knife” CAR T cells solve key efficacy and safety issues

Chimeric antigen receptor (CAR)-expressing T cells represent a promising approach to cancer therapy, but safety and efficacy are still major hurdles. Scientists at Boston University and Massachusetts Institute of Technology (MIT) have now developed a new CAR technology, called split, universal, and programmable (SUPRA) CAR, which they claim represents the “Swiss army knife of CAR” and can simultaneously address tumour resistance, prevent immune system overactivation, and enhance specificity.
The researchers claim the SUPRA CAR technology simultaneously encompasses “multiple, critical upgrades,” including the ability to switch targets without the need to re-engineer the T cells, as well as fine-tune T-cell activation strength, and sense and respond to multiple antigens. In effect the system can fine-tune T-cell activation strength to mitigate toxicity, sense and “logically respond” to multiple antigens to combat relapse, and inducibly control celltype-specific signalling.
In vitro tests with SUPRA CAR constructs demonstrated the potential of the system to increase tumour specificity and reduce CAR T-cell therapy toxicity by controlling response. In vivo tests demonstrated use of SUPRA CARs to reduce tumour burden in a mouse breast cancer xenograft model and in a blood tumour model. “The observed robust activity of the SUPRA system against different xenograft models demonstrates the potential of the SUPRA CAR system to combat many cancers. Further in vivo studies then confirmed that the SUPRA CAR system can be fine-tuned using multiple approaches to regulate cytokine release, offering the potential to manage toxicities, such as severe cytokine release syndrome that can occur with conventional CAR T-cell therapies. The work was published in the journal Cell.
Source:, April 27, 2018.

Stem cells: on your mark, get set, don’t go

Researchers have discovered a new technique to overcome one of the major challenges of stem cell therapy the tendency of cultured stem cells to differentiate prematurely. To keep stem cells from differentiating prematurely, scientists based at the University of British Columbia (UBC) have developed a drug that helps regulate the transition between activated, proliferating stem cells and differentiationprimed, poorly engrafting progenitors. The drug, which has been tested successfully in mice, inhibits the methyltransferase Setd7.
Setd7, the UBC scientists learned, plays a role in controlling stem cell growth and their maturation into muscle fibers. Using their Setd7 inhibitor, the scientists were able to prevent immature, transplantable myogenic stem cells (MuSCs) from differentiating so that these cells could continue to divide. Then the scientists implanted the stem cells into the hind leg of mice affected by a mouse model of muscular dystrophy.
Upon transplantation, both mouse and human MuSCs that were expanded with a Setd7 small-molecule inhibitor are reported to be better able to repopulate the satellite cell niche, and treated mouse MuSCs were reported to show enhanced therapeutic potential in preclinical models of muscular dystrophy. Essentially, the UBC team found that the cells fused to the muscle, regenerated the tissue, and improved the strength of the muscle. The study was published in the journal Cell Stem Cell.
Source:, Jan 29, 2018.

CRISPR activation of single genes turns skin cells to stem cells

A new way of inducing pluripotency has been demonstrated by scientists based at the Gladstone Institutes. Rather than fuss
with the usual transcription factors or druglike chemicals, the Gladstone scientists used CRISPR activation (CRISPRa). This technique, which incorporates a modified form of CRISPR gene-editing systems Cas9 enzyme, allowed to target and epigenetically remodel specific genomic locations: the Oct4 and Sox2 genes. Remodelling either gene, the scientists discovered, turned skin cells from mice into stem cells.
The scientists reported that they had better results with their CRISPRa systems the dCas9-SunTag-VP system and the dCas9-SunTag-p300core system both of which are optimized versions of the SunTag reprogramming system. “Our results show that the epigenetic remodelling of Oct4 promoter and enhancer, either through VP64 or p300core, is sufficient to trigger reprogramming toward pluripotency”. Simultaneous remodelling of the Oct4 promoter and enhancer also triggered reprogramming and authentic pluripotent cell lines were established in both cases. The CRISPRa approach not only generates iPSCs, but also sheds light on the mechanisms of cellular reprogramming. The reprogramming strategy should also work in the generation of other cell types and in other model systems, such as human cells. Findings were published in the journal Cell Stem Cell.
Source:, Jan 19, 2018.

Stem cells made into relay cells for sense of touch

For the first time, human sensory interneurons the cells that communicate the sense of touch in the central nervous system have been derived from stem cells. In clinical applications, stem cell-derived sensory interneurons could restore feeling in paralyzed patients, potentially complementing stem cell-derived motor neurons, which are being developed to restore coordinated movement.
SJ. Butler, Ph.D., and colleagues at the University of California, Los Angeles have developed a protocol that can generate sensory interneurons from either human embryonic stem cells (hESCs) or human induced pluripotent stem cells (hiPSCs).
Two developmentally relevant factors, retinoic acid in combination with bone morphogenetic protein 4, can be used to generate three classes of sensory INs [interneurons]: the proprioceptive dI1s, the dI2s, and mechanosensory dI3s. DI1 sensory interneurons give people proprioception a sense of where their body is in space and dI3 sensory interneurons enable them to feel a sense of pressure.
Whether they tried reprogramming hESCs or hiPSCs, the researchers found that their protocol yielded the same mixture of sensory interneurons. This reprogramming method creates stem cells that can create any cell type while also maintaining the genetic code of the person from which they originated. The ability to create sensory interneurons with a patient’s own reprogrammed cells with hiPSCs derived from differentiated adult cells holds significant potential for the creation of a cellbased treatment that restores the sense of touch without immune suppression.
Dr. Butler hopes to be able to create one type of interneuron at a time, which would make it easier to define the separate roles of each cell type and allow scientists to start the process of using these cells in clinical applications for people who are paralyzed. However, her research group has not yet identified how to make stem cells yield entirely dI1 or entirely dI3 cells perhaps because another signalling pathway is involved. The researchers also have yet to determine the specific recipe of growth factors that would coax stem cells to create other types of sensory interneurons. The protocol details appeared in the journal Stem Cell Reports.
Source:, Jan 16, 2018.

Functioning human skeletal muscle grown from induced pluripotent stem cells

Duke University researchers have grown the first functional human skeletal muscle tissue entirely from induced human pluripotent stem cells (hPSCs). The achievement could feasibly allow scientists to generate skeletal muscle tissue for disease modelling and drug studies, and potentially for developing hPSC-based therapies for muscle-wasting diseases.
The team cultured induced hPSCs derived from adult non-muscle tissues in the presence of Pax7. They found that the cultured cells proliferated and developed into expandable myogenic progenitors, or induced myogenic progenitor cells (iMPSCs), which resemble native adult muscle stem cells. Grown in 2D culture, the hPSC-derived iMPSC differentiated into functional myotubes and cells that expressed Pax7, which are “the two main constituents of native skeletal muscle.”
The iMPC-derived myotubes didn’t survive for long in 2D culture, however, so the team next embedded the cells in a fibrin-based hydrogel to provide support and allow the tissue to develop into 3D skeletal muscle (iSKM) bundles. Within just a few weeks, these bundles differentiated into functional muscle tissue that exhibited the characteristic sarcomeric structure of natural muscle. These iSKM bundles were also able to contract and react to electrical stimuli and biochemical signals. Encouragingly, cultured muscle tissue transplanted into immunecompromised mice survived and continued to function. The transplanted tissue became vascularised, and integrated into the animals’ own muscle. The study was published in Nature Communications.
Source:, Jan 9, 2018.

CAR-engineered blood stem cells offer new hope for long-term HIV gene therapy

Researchers in the U.S. have created chimeric antigen receptor (CAR)-engineered hematopoietic stem/progenitor cells (HSPCs), which successfully engrafted in pigtail macaques infected with simian human immunodeficiency virus (SHIV) and generated virus-targeting cytotoxic T cells that destroyed infected cells.
The researchers, from the University of California, Los Angeles, the Fred Hutchinson Cancer Research Center, and the University of Washington, said that the CAR-engineered HSPCs persisted in the animals for more than two years, suggesting that their approach could successfully be used to treat infections such as HIV that can re-emerge after many years of suppression or dormancy. “This demonstrates for the first time the safety and feasibility of HSPCbased therapy utilizing an HIV-specific CAR for suppressed HIV infection”. The study was reported in PLOS Pathogens.
Source:, Dec 29, 2018.


Labiotech Refresh
22 May 2018, Brussels, Belgium
More info:

2nd Quality Risk Management Summit
20-21 June 2018, Lisbon, Portugal
More info:

18th European Congress On Biotechnology
1-4 July 2018, Geneva, Switzerland
More info:

Nordic Life Science Days
11-12 September, 2018, Stockholm, Sweden
More info:

5th Laupheim Biotech Days
20-21 September , 2018, Laupheim, Germany
More info:

25-27 September, 2018 in Seville, Spain
More info:

European Forum for Industrial Biotech and the Bioeconomy
6-18 Oct 2018, Toulouse, France
More info:

©2020 ACTIP Privacy Policy

Log in with your credentials

Forgot your details?