Benefits of Single-use Technologies

Demand for worldwide bioprocess suppliers to develop technologies for producing disease treatments and vaccines has increased dramatically. To meet the growing needs for new biologics in a reliable, fast paced and cost efficient manner, it has resulted in a steep rise in the uptake of single-use technologies. We take a look at the benefits of it here.

Craig E. Smith from Thermo Fisher Scientific will be talking on: “Meeting the Challenges of a Rapidly Changing Bioproduction Industry with Single Use Technology – Past, Present and Future” at BioProcess International European Summit on 12th April.

Benefits of Single-use technologies

FDA Breakthrough Therapy Designation List 2016-2017

With new FDA Breakthrough Therapy Designations constantly being granted, we keep an up-to-date list of every approval. Check back for all the latest news and information.

(Last Updated: 2 February 2017)

Continue reading “FDA Breakthrough Therapy Designation List 2016-2017”

FDA Breakthrough Designation Approval: Challenges & Tips from Roche’s Dr. Niklas Engler

Since being signed into law on 9th July 2012, the FDA’s Breakthrough Designation has changed the face of drug development. In November 2013 Roche’s monoclonal antibody, Gazyva, became the first medicine designated as a Breakthrough Therapy and in the next two years a further 37 approvals were granted. This increasing trend has continued into 2016, with a raft of new drugs given approval in the first two months of the new year.

Roche continue to be at the forefront of the industry and have had a hand in more than one of these new approvals. In January their drug venetoclax (ABT-199), developed in partnership with AbbVie, received a third Breakthrough Therapy Designation for use in combination with hypomethylating agents, to treat patients with first-line acute myeloid leukemia (AML) who cannot receive high-dose chemotherapy. Then just this week it was announced that ocrelizumab (OCREVUS) has been given BTD approval for treatment of patients with primary progressive multiple sclerosis (PPMS).

Early last year we spoke to Dr. Niklas Engler, Head of European Technical Development for Biologics at Roche, about the company’s success with the FDA’s Breakthrough Designation and the challenges it creates.


“The FDA’s Breakthrough Designation has created a direct dialogue between sponsors and the agency. For Gazyva, which attacks targeted cells both directly and together with the body’s immune system, we had weekly exchanges; the speed in which the FDA was working with us was really impressive.”

Technical Support Teams

“The big challenges we face at the moment aren’t necessarily protein production, that is fairly well understood, it’s how technical teams support variations in clinical trials, whether it’s an accelerated programme such as FDA Breakthrough Designation, or heavily gated programmes where you don’t do any technical support for a long time and then have to catch up very quickly.”

Cost and Resources

“Obviously, the financial and personnel investments are huge for such as product so you have to make sure you have the right product in place and all the departments are aligned – technical, clinical and non-clinical development. Because once you jump on that train you accelerate very quickly. If you are not prepared you can really slow down any accelerated advantage you once had.”

Supplying the market

“If you want to go for a large indication like the PD-1 and PDL-1 markets, you need to make sure everything is in place to supply. This is a big topic for the FDA, they don’t want difficulties in supply.”

Want to find out more about this topic? Gargi Maheshwari (Executive Director, Biologics Process Development and Commercialisation, MSD) will be talking on Merck product Keytruda’s FDA Breakthrough Designation at BioProcess International European Summit in Vienna. Her session – “Keytruda – Acceleration of a Breakthrough Therapy…What to do when CMC is on the Critical Path” – is at 9.25am 11th April. Find out more and see the full agenda here.

3D tissue printing breakthrough sends excitement through regenerative medicine industry

On a day that has seen the UK’s newspapers run front page stories on the benefits and successes of science’s golden child – the CAR-T cell, other news has surfaced of another equally successful breakthrough in the field of regenerative medicine.

First published in Nature Biotechnology, a team at Wake Forest Baptist Medical Centre has developed a new technique that 3D-prints living body parts, which when implanted into animals function normally.

Although similar techniques where biodegradable scaffolding is built and then ‘soaked’ in cells are already in use for human patients, the science of tissue regeneration in the past has been limited by the enormous challenge of keeping the cells alive. Generally speaking once the tissues become thicker than 0.2 millimetres the cells become starved of nutrients and oxygen.

To combat this limitation the team, led by Professor Anthony Atala, has developed a ground breaking new technique that creates tissues laden with micro-channels more akin to a sponge, which allows the nutrients to be delivered and penetrate the tissue.

In a joint publication with Hyun-Wook Kang, Sang Jin Lee, In Kap Ko, Carlos Kengla and James J Yoo, Professor Atala writes that the challenge of producing “3D, vascularised cellular constructs of clinically relevant size, shape and structural integrity” can be overcome by their “integrated tissue-organ printer (ITOP)”.

ITOP can create stable tissue constructs of any shape at a human-scale. Explaining how the technology works, the paper details:

“Mechanical stability is achieved by printing cell-laden hydrogels together with biodegradable polymers in integrated patterns and anchored on sacrificial hydrogels. The correct shape of the tissue construct is achieved by representing clinical imaging data as a computer model of the anatomical defect and translating the model into a program that controls the motions of the printer nozzles, which dispense cells to discrete locations.”

The real genius though is the addition of microchannels into the tissue that facilitates the diffusion of nutrients to printed cells (the sponge like quality) thus overcoming the 0.2 mm thickness limit of tissue constructs.

The technology has been demonstrated in mandible and calvarial bone, cartilage and skeletal muscle, with the future aims to produce tissues for human complex tissues and solid organs.

Speaking to the BBC, Professor Atala explains how this works in the real world,

“Let’s say a patient presented with an injury to their jaw bone and there’s a segment missing. We’d bring the patient in, do the imaging and then we would take the imaging data and transfer it through our software to drive the printer to create a piece of jawbone that would fit precisely in the patient.”

Although this breakthrough has got many in the field excited, Professor Atala has cautioned that additional research is needed before ITOP tissue constructs can be used in patients, but mused that “it will be less than a decade before surgeons like me are trialling customised printed organs and tissues. I can’t wait!”

Image Credit: Nature BioTechnology

Francis Crick Institute Scientists Granted License to Edit Genes in Human Embryos – What will the Initial Impacts be?

In a global first, the UK’s Human Fertility and Embryology Authority (HFEA) have granted permission to a group of London-based scientist to edit the genomes of human embryos for research. Announced on 1st February 2016, the endorsement is the first of its kind by any national regulatory authority.

The approved research application from the Francis Crick Institute led by Dr Kathy Niakan is aimed at identifying and understanding the gene human embryos need to develop successfully. The research team will look at the first seven days of a fertilised egg’s development in the hope of providing important knowledge for understanding how a healthy human embryo develops.

Dr Niakan will be using CRISPR-Cas9 technology which enables the scientists to modify, identify, or replace genetic defects.

Commenting on the news, Paul Nurse, Director of the Crick, explained: “I am delighted that the HFEA has approved Dr Niakan’s application. Dr Niakan’s proposed research is important for understanding how a healthy human embryo develops and will enhance our understanding of IVF success rates, by looking at the very earliest stage of human development.”

So what will be the initial impact of this authorisation?


With a topic such as genetically modified human genomes, controversy will not be far behind, with those opposed to the authorisation believing that this will open the door to ‘GM babies’.  It must though be made clear that this authorisation specifically states that it will be illegal for the scientists to implant the modified embryos into women.

Speaking on the ethical implications of the decision, Dr Sarah Chan from the University of Edinburgh said:

“The use of genome editing technologies in embryo research touches on some sensitive issues, therefore it is appropriate that this research and its ethical implications have been carefully considered by the HFEA before being given approval to proceed.”

“We should feel confident that our regulatory system in this area is functioning well to keep science aligned with social interests.”


Moving away from the controversy, it is good news for those receiving IVF treatment. With the increased knowledge that will be gained from the research, IVF success rates should dramatically improve thanks to the focus on miscarriage and fertility. Speaking to the BBC, Dr Kathy Niakan has said that the reason why she applied for the approval to edit human embryos was that “we would really like to understand the gene needed for a human embryo to develop successfully into a healthy baby”.

Dr Niakan went further to explain that “the reason why it is so important is because miscarriages and infertility are extremely common, but they’re not very well understood”.

Ethical Approval

The research is still in need of ethical approval before the programme can begin, but it is looking likely that this will be granted and the research will begin in the next few months.

With the research likely to add to the basic scientific knowledge needed for assisting infertile couples becoming parents, and to reduce the heartbreak of miscarriage, this is but one practical positive that will hopefully come from the work of Dr Niakan and her team.

Not only does the research have the potential to greatly assist in IVF treatment, it is another display of the excellent position the UK holds in the genome editing field of research and the advances being made by UK scientists.

At Cell Therapy Manufacturing & Gene Therapy Congress on 2nd – 5th February in Brussels, industry leaders will discuss technical and commercial strategies to deliver gene therapy products to patients. See the full agenda and book tickets at

The Prime Medical Group: The Value of Medical Communications for Gene Therapy

With Cell Therapy Manufacturing & Gene Therapy Congress less than a week away, we spoke to one of the exhibitors presenting in Brussels:

“The Prime Medical Group is delighted to be presenting at next week’s Cell Therapy Manufacturing and Gene Therapy Congress. The congress will provide a forum for delegates to discuss technical and commercial strategies for cell and gene therapies.

The Prime Medical Group has worked extensively in this area, providing strategic communications support for several orphan drugs and rare diseases, as well as developing and implementing the communications programme for the only gene therapy approved in Europe.  Howard Sinclair (Strategic Director – Rare Diseases and Gene Therapy) and Andrew Jobson, PhD (Scientific Director – Rare Diseases and Gene Therapy) will present on Tuesday 3 February at 12:10pm. The presentation will focus on the importance of an effective medical communications strategy and plan, as well as the key considerations when bringing an orphan drug or gene therapy to the market to ensure optimal return on clinical studies investment.

‘This is an exciting opportunity for The Prime Medical Group to share our experience and expertise with some of the leading figures in the industry,’ Howard commented. ‘We have successfully partnered with pharmaceutical and biotech companies over the years and understand the challenges faced when developing an impactful communications programme for orphan drugs and rare diseases as well as gene therapies.’

In addition to presenting, The Prime Medical Group will also have an exhibition booth at the congress allowing delegates to discuss their specific communication needs with the team in more detail.”

Cell Therapy Manufacturing & Gene Therapy Congress is being held in Brussels on 2nd – 5th February 2016. See the full agenda and book tickets at  

The Prime Medical Group: Established in 1997 The Prime Medical Group is a leading medical education and communications agency, committed to creating outstanding medical education and communication programmes with global, regional or national implementation. To learn more their therapeutic experience or services visit