Archives for category: Cell Therapy and Regenerative Medicine

Last week we held the latest in our series of BIA member webinars, which focused on cell and gene therapy, with guest speakers Dr Sven Kili and Dr Emily Culme-Seymour from GSK, Michael Hunt of ReNeuron and Dr Damian Marshall from the Cell and Gene Therapy Catapult.

With emerging technologies and new clinical trials being developed and launched, the webinar examined just a few of the advances and evolution in the development of cell and gene therapies over the last couple of years, as well as business models and commercial milestones.

Joined by almost 100 participants on the webinar, BIA CEO Steve Bates noted the keen interest inspired by this sector.

Dr Sven Kili kicked things off with an introduction to the BIA’s Cell and Gene Therapy Advisory Committee, on which he holds the position of Chair. As a key area of focus for the BIA, the Committee’s expert input helps to inform BIA positions on relevant policy issues – such as the current Select Committee inquiry into Regenerative Medicine and, when it is published, the Accelerated Access Review.

Alongside the growing interest in cell and gene therapy witnessed in recent years, the area has also seen a growth in clinical development and likewise, investment. GSK is investing heavily in cell and gene therapy, and during the webinar Dr Emily Culme-Seymour outlined some of their ongoing projects of interest, including in GSK’s rare diseases portfolio. As for the future, the cell and gene therapy environment continues to evolve and Emily highlighted the benefits of utilising a more collaborative approach to the development of novel therapies, such as partnerships with SMEs and academia.

Next up was Michael Hunt, Chief Financial Officer of ReNeuron. Having been AIM-listed since 2005, Michael observed that the funding landscape has evolved over recent years. Michael gave an outline of ReNeuron’s therapeutic pipeline, covering treatments across the diverse areas of retinitis pigmentosa, motor disability from stroke, and critical limb ischaemia.

Finally, Dr Damian Marshall provided an overview of the Cell and Gene Therapy Catapult’s analytical platform for characterisation of cell therapy products, which allows data to be integrated from multiple platforms to analyse the biological, chemical and physical properties of cell products.

Watch the webinar in full below.

Watch this week’s video to see what the Cell and Gene Therapy Manufacturing Centre, currently under construction in Stevenage, will look like once it is opened in 2017.

To find out more about the centre visit the website.

Do you have a video you would like the sector to see? Contact us.

As people come back from the ‘holidays’ the Knowledge Transfer Network (KTN) is checking in that you are exploring the Innovate UK funding calls currently available where ATMPs are in scope – and that you can contact the KTN for support.

These include BioMedical Catalyst (14 September deadline, up to £10 million to develop innovative ideas that will help solve healthcare challenges, our sector in scope particularly reference to ‘tailored treatments that either change the underlying disease or offer potential cures’.

Also current is the £15 million Open Competition, though cross sector this includes any technology, engineering or industrial area, including Innovate UK’s 4 priority sectors for growth- e.g. health and life sciences, total eligible project costs of £25,000 up to £1 million, deadline 7 September.

Forthcoming for 2016 is the Health and Life Sciences call, planned is the cell therapy manufacturing call (both mentioned on page 17 of Innovate UK delivery plan).

KTN are also exploring how companies might use the Global Cooperation Feasibility Studies competition – where business can apply for a share of £1.2 million to carry out short, international feasibility studies – the competition brief mentions that they will fund SMEs to support the costs for their involvement in activities such as:

  • meetings with partners in the UK or other countries
  • travel and subsistence, including travelling abroad to meet partners, attend brokerage or exhibition events and gain a better understanding of market opportunities and user/customer needs
  • due diligence work on partners and markets
  • staff time for proof of market or concept work
  • sub-contracting services (limited to up to 10% of costs)

So this might be useful if exploring a collaboration partner through attending a key event in Q1 2017, or an EU proposal?

The recent Treasury announcement makes it clear that EU H2020 opportunities are very much open to UK business – such as the SME instrument Cell technologies in medical applications and especially calls like the single stage SC1-PM-11-2016-2017 Clinical Research on regenerative medicine.

It will also be interesting to see what opportunities might come out of the Innovative Medicines Initiative (IMI) Stakeholder Forum at end September where there is a dedicated advanced therapies discussion.

KTN continues to work alongside the National Contact Points (NCPs) for Health and the SME instrument, Innovate UK and Enterprise Europe Network (EEN) on how best to support businesses in applying for these calls. We also work closely with the Catapults key to this space.

Contact me or a colleague directly and we’ll help you to make an efficient decision on which call(s) might give you a non-diluting funding stream to drive forward your innovation, and business. We know that often this is a balance between current business strategy and precious time to develop proposals and collaborations.

Sarah Goulding and Mark Bustard

Sarah.goulding@ktn-uk.org   mark.bustard@ktn-uk.org

Our 2nd Annual Committee Summit takes place in London today, where we’ll be hearing from each of our eight Advisory Committees on their 2015 objectives and key issues and priorities for 2016. Here we take a look at our Committees focussing on the fields of synthetic biology and cell therapy and regenerative medicine, two areas identified by policy makers as technologies in which the UK is set to be a global leader – part of the “Eight Great Technologies” initiative. Find out more in these infographics: synthetic biology and regenerative medicine.

Cell Therapy and Regenerative Medicine Advisory Committee (CTRMAC)

CTRMAC is one of our most popular committees with over 30 member companies taking part, many of which are involved in the R&D of cell and gene therapies. In 2015 the Committee welcomed Cell Medica and Cellular Therapeutics into the fold.

The Committee maintains close links to the wider cell therapy and regenerative medicine sector – the newly renamed Cell and Gene Therapy Catapult hosts the majority of CTRMAC meetings and through the year we’ve had guest speakers from the ATMP Manufacturing Community (amc) and UK Regenerative Medicine Platform (UKRMP).

In 2016 the Committee will be working in alignment with our Regulatory Affairs Advisory Committee priorities in reviewing NICE’s forthcoming mock appraisal of a CAR-T cell therapy, and will align with manufacturing issues and the Manufacturing Advisory Committee regarding the supply chain of viral vectors for gene therapy. Once the Accelerated Access Review report is released this spring, CTRMAC will focus on the implications for cell and gene therapies, particularly on pricing and reimbursement of advanced therapy medicinal products (ATMPs).

RegenMed_infographic

Synthetic Biology Advisory Committee (SBAC)

In 2015 we were pleased to welcome SynbiCITE, the Innovation and Knowledge Centre for Synthetic Biology, as a new member of both BIA and SBAC. For the third year running, a number of SBAC members (this year Synpromics, Synthace, Touchlight Genetics and ZuvaSyntha) were represented on UK trade mission to San Francisco, organised and funded by SynbiCITE with UKTI support.

Following on from the 2012 UK Roadmap for Synthetic Biology, the Synthetic Biology Leadership Council, with ministerial level input, has this week published the next phase of strategic direction for this growing technology area – Biodesign for the Bioeconomy: UK Synthetic Biology Strategic Plan 2016.

Speaking at the launch of the plan yesterday, Life Sciences Minister George Freeman MP outlined a vision for the growth of this innovative sector in the UK, ensuring the UK maintains its position as a global leader. In conjunction with this, SBAC’s core aims in 2016 include to continue to showcase strengths in the UK sector, which is likely to include company engagement at the popular SynBioBeta conferences in London (6 – 8 April 2016) and San Francisco, and to increase investor awareness of and engagement with UK synthetic biology.

SynBio_infographic

Find out more about our eight Advisory Committees on the website, or read our recent blogs on the Finance and Tax, Intellectual Property, and the Communications and People Committees.

blog_reportThroughout the year the BIA advocates for our members in the UK life science industry, engaging with decision makers and influencers in Westminster, Whitehall and across the science, health and business sectors, as well as with regulators in the UK and Europe. Our latest quarterly report, ‘Influencing and shaping our sector: BIA update July – October 2015’, sets out what we’ve been up to in the last few months.

You can download the report in full here. Highlights include:

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  • Our work ahead of the Government’s Spending Review on 25 November to illustrate why public funding for science and innovation is so vital. We’ve undertaken a range of activities on behalf of industry and in collaboration with the wider life science community. If you want to add your voice please do contact us!
  • Our activities across blog_labour tablethis autumn’s party conference season, including our joint roundtable discussions as part of Life Sciences UK (ABHI, ABPI, BIA and BIVDA) at the Labour and Conservative party conferences. We also review the new Labour landscape following the installation of Jeremey Corbyn as new Labour leader and his shadow ministerial team.
  • An update on Patent Box developments. Following the completion of talks at an international level by the G20 and the OECD on the Base Erosion and Profit Shifting (BEPS) project, HM Treasury has issued its consultation on the implementation of the reforms into the UK. Find out more about the changes and our efforts to demystify the policy and potential business implications, starting with the publication of an overview paper – Changes to the UK Patent Box: An Overview, October 2015 – which was prepared in partnership with Confluence Tax.
  • Details on the recently announced PRIME scheme – a new scheme, developed by the European Medicines Agency (EMA) and the EU Regulatory Network, to support development of priority medicines addressing major public health needs. The BIA will be inputting to the public consultation issued on 26 October on the key elements of the scheme, which is scheduled to launch in Q1 2016.

We’re always keen to hear from our members. Which policy areas do you care about; what’s impacting your business and R&D? Do you have a story that would help us to make the case for continuity in what works and to change what doesn’t?

To learn more about BIA’s policy, public affairs and regulatory affairs work, see our previous quarterly policy updates, our consultation responses, or email Zoë with any questions.

Earlier this week, the Regenerative Medicine Expert Group (RMEG) published their report ‘Building on our own potential: a UK pathway for regenerative medicine’. The report is the culmination of work by the RMEG, in consultation with other stakeholders, to develop a strategy and action plan to make the NHS ready to deliver regenerative medicines. In the post below, Helen Cline, Legal Director, Pinsent Masons takes a closer look at the report.

Complex regulation must be streamlined and commercial incentives improved for the UK to remain a hub for regenerative medicine.

The UK Government has identified regenerative medicine as one of the UK’s “Eight Great Technologies”, due to its “huge opportunities for technological advance” and potential economic benefit. The RMEG report rightly concludes that the UK has the “industrial base, the academic excellence and the clinical know-how that is necessary” as well as previous experiences of success in advanced medicine and treatments to “consolidate and build upon its position as a world leader in regenerative medicine”. Even so, challenges remain.

The RMEG is tasked with developing an NHS strategy for regenerative medicine, which it defines as “methods that replace or regenerate human cells, tissues or organs in order to restore or establish normal function”. The report sets out what additional steps are necessary to help researchers develop, and clinicians utilise, technologies that improve the human body’s ability to repair itself.

Regenerative medicine is the “future of medicine” and the proposals in the RMEG report to make navigating the regulatory environment easier are welcome.  Regulation in the area of regenerative medicines is complex, reflecting the nature of the technologies and their risks. For example, in the UK there are four regulatory bodies, each regulating distinct aspects of the development of regenerative medicine under different pieces of legislation, as well as a range of other agencies with an oversight role. One of the practical problems for researchers in regenerative medicine is the division of regulatory responsibility between these different bodies. There have already been steps taken towards the streamlining of regulatory advice, with a single point of access, for advice on regenerative medicine rules, available from the Innovation Office within the Medicines and Healthcare Products Regulatory Agency. Regulators have also collaborated on providing guidance on regenerative medicines issues, including developing the UK Stem Cell Tool Kit  to provide clear guidance on the regulatory pathways that must be followed in developing a regenerative product derived from stem cells. However, the RMEG’s report correctly highlights that “further steps are needed to ensure that standardisation of processes, and streamlined regulation, are guiding principles in advancing regenerative medicine”.

The RMEG, like the House of Lords’ Science and Technology Committee in their 2013 report,  also identifies funding issues as a barrier to the growth of regenerative medicines, and calls on the UK government and industry to come together to develop “an innovative business model that supports the early adoption of regenerative medicines”. It also calls for reviews to be carried out on “the funding for excess treatment costs for cell therapy trials”, with the aim of finding “a mechanism … to ensure that meeting of these costs is not a barrier to clinical trials or the early adoption of technologies”.

While debate in the past has been on the legality of funding research, at least in the US, debate today is on how best to regulate the clinical translation and support the commercialisation of these innovative technologies.

The nature of regenerative medicine research involves creating personalised precision therapies that may provide a cure rather than merely alleviating symptoms. There are, in many cases, high up-front costs associated with these individualised therapies that raise challenges around evaluation, funding and adoption. Unnecessary, excessively complex or burdensome regulation, together with unclear evaluation and funding pathways, could stifle the promise of regenerative medicine. That said, moves to streamline regulation to enable innovation must not put patients at risk of harm and damage the reputation of this field of research at a crucial point in its history.

Ethical and patent issues have also created challenges in the development of regenerative medicines. A ruling by the Court of Justice of the EU last year on the patentability of inventions that emerge from research on human embryonic stem cells highlights some of the divergent opinions on the issues.

Research in the field of regenerative medicine is emotive, especially when it concerns human embryonic stem cells. The debates around regulation and commercialisation include a diversity of opinion on the many ethical, regulatory and legal considerations; one example being patient consent in clinical trials, as the line between research and treatment is blurred in this area of medicine.

The UK Government has demonstrated its commitment to the growth of regenerative medicines research in the UK. Centres of excellence around cutting-edge technology are being assembled with a view to combining research and catapulting it forward; examples include the Cell Therapy Catapult and the new Precision Medicines Catapult. However, for regenerative medicine to flourish, and for the UK to retain its strong position in Europe and globally, a strict but enabling legislative and regulatory framework that facilitates innovation is essential. The current approach to regulation of medicines poses particular challenges in the era of ‘stratified medicines’. There are practical challenges in building the clinical evidence base necessary for approval under current regulatory mechanisms where patient populations are very small. The current regulatory and evaluation processes and policies need to evolve in response to, and in anticipation of, scientific developments that will be critical for the development of the UK’s regenerative medicines industry and the move to a stratified and more personalised approach to developing precision medicines.

The original article is available from Out-Law.com, legal news and guidance from Pinsent Masons.

Read the BIA press release welcoming the publication of the report here.

The testing of new medicines can be a lengthy and arduous process, delaying access to promising innovative treatments. Today’s video features a TED talk from bio researcher Geraldine Hamilton, who creates ‘organs-on-a-chip’ – engineering a ‘home away from home’ for the cells which more accurately recreates the dynamic environment in which they exist in the body.

Watch to find out how the chips are created and how they are being utilised for the testing of new medicines, including in the burgeoning field of personalised medicine.

Do you have a video you would like the sector to see? Contact us.

Following on from last year’s event in Brussels, Thursday 13 November saw key opinion leaders, life science investors, analysts and advanced therapy companies gather in central London for the 2nd Annual EU Advanced Therapies Investor Day. Hosted by the Alliance for Regenerative Medicine (ARM) in cooperation with GE Healthcare Life Sciences, Bryan Garnier & Co and Cell Therapy Catapult, the one day event provided attendees with an insight into the latest technologies and therapies for a variety of indications.

Kieran Murphy, CEO, GE Healthcare

Kieran Murphy, President and CEO, GE Healthcare Life Sciences

Morrie Ruffin, Managing Director, ARM opened the event followed by a keynote presentation from Kieran Murphy, President and CEO, GE Healthcare Life Sciences. In his presentation, Kieran outlined the huge potential of the field due to the sheer number of patients affected by diseases that advanced therapies have the ability to treat, such as cancer and diabetes. He described how ‘bringing regenerative medicine to the mainstream’ will require more than biopharmaceutical companies alone – including global academic collaborations, government input, gaining the confidence of regulators, investment and sophisticated novel supply chain processes.

Throughout the day there were presentations from 14 advanced therapy companies, including BIA member companies Oxford BioMedica and ReNeuron.

The rationale behind investment in immuotherapies was discussed in a ‘fireside chat’ between Usman (Oz) Azam, Global Head, Cell & Gene Therapies Unit, Novartis and Keith Thompson, CEO, Cell Therapy Catapult. Oz explained how compelling clinical data underpinned Novartis’ decision to create a dedicated business around cell and gene therapies and discussed the progress of their CAR T cell therapy candidate CTL019. The potential of CAR therapies to treat solid tumours was also indicated and the panel agreed that announcements such as the ‘one stop shop’ for regulation of regenerative medicine products show that regulators are receptive to these technologies.

The day continued with a session on targeted immunotherapies for oncology. Discussions covered how the ‘science is ripe’ in regenerative medicine, which will lead to big returns on investment and how scale up of processes and an enabling ecosystem will be key to success. The panel suggested that genome editing platforms such as CRISPR and zinc finger nucleases could revolutionise the cancer immunotherapy field. UCL’s Emma Morris identified funding for phase I studies from global research councils as key to advancing the regenerative medicine field.

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Steve Bates chairs panel session on therapies for ophthalmologic disorders

Steve Bates, CEO, BIA chaired a session on therapies for ophthalmologic disorders featuring panellists from Sanofi, UCL, QMUL and NightstaRx. They discussed how the eye presents an attractive target for cell therapies due to its accessibility and suggested that the ophthalmologic space has the potential for developing blockbuster therapies to treat diseases such as choroideremia, age-related macular degeneration and retinitis pigmentosa. It was questioned what expertise and infrastructure would be required to successfully implement these therapies in a clinical setting.

The final panel reviewed the use of cell and gene based therapies to treat cardiovascular disease. This brought up many interesting ideas, including the possibility of ‘cell free therapy’ – providing the proteins and substances that cells secrete at the right time and in the right quantities to treat diseases. Methods of achieving better delivery and retention of cells in cardiac tissue were also discussed.

If you’re interested in catching up on the day’s events, a webcast of all talks, panels and company presentations will be available within the next two weeks on the event website.

BIA_synbiomission_600x300Last week saw a sudden and unexpected joint proposal from the UK and Germany that would make changes to preferential intellectual property tax regimes, including the UK Patent Box, a key part of the fiscal landscape for attracting and retaining life science companies to the UK.  The proposed changes will be under discussion this week at the OECD/G20 summit in Brisbane as part of the Base Erosion and Profit Shifting (BEPS) project. BEPS is an extremely broad tax project regarding the determination on a country by country basis of the taxable profits of multinationals. Patent boxes are just part of one of 15 workstreams.

This new proposal does not appear to represent any change in the intention of the UK patent box regime, ie it does not propose abolishment of a regime providing a lower tax rate on profits  generated from patents. What it does address are the rules that underlie such schemes and how to define “substantial activity”, in the context of mobile income and assets such as IP. The OECD has suggested three approaches for consideration – value creation, transfer pricing and the nexus approach. The UK patent box scheme is at present based on a transfer pricing approach (as articulated by Treasury Minister David Gauke MP at the BIA’s UK Bioscience Forum) where substantial activity was to be determined in the context of the location of key functions. This new proposal outlines a nexus approach which permits a jurisdiction to provide benefits to income arising out of IP so long as there is a direct nexus between the income receiving benefits and the expenditures contributing to that income.

If the proposal is approved by the OECD/G20 this week the intention would be to work towards a new nexus-compliant UK patent box with effect from July 2016. Existing IP in the current regime at that date will be “grandfathered” and allowed to apply the old rules until 2021.

At this stage it is difficult to say what the precise implications could be across the sector if this proposal is adopted. The BIA is working and will continue to work with members, government and wider stakeholders on this issue and it is the top agenda point at our Finance and Tax advisory committee this week. Please get in touch with myself of Pamela Learmonth if you would like to engage directly with us on what will be an ongoing policy project.

Later this week, UKTI plan to launch a new website which will contain a searchable database and map of UK Life Science companies, making public some of the database from which they’ve sourced the ‘Strength and Opportunity’ reports on the sector published in recent years. The draft website can be accessed here http://ukti.cargodev.co.uk and any comments should be addressed to roisin.mcclory@uktispecialist.com. The BIA has endeavoured to help BIS check their data is accurate over the years to the best of our knowledge but we do not own or administer this database. UKTI have asked us to share the following statement with members in advance of the official launch, which gives detail advising on the collection of data and the origin of it.

“The name, location and segmentation  of  companies included in the UK Life Sciences website have been drawn from the Bioscience and Health Technology Database which is owned by the Office for Life Sciences within the Department for Business, Innovation and Skills. Turnover and employment information is sourced from a third party on a commercial basis and drawn from reported figures or estimated figures (where there is no requirement for the company to submit accounts). It is presented in bands as individual company information cannot be released. The data shown for each company and location is sourced at site or location level for employment and for turnover the bands represent UK life science derived turnover estimated to arise from activity at that site.

Information on company names and addresses has been obtained from a  range of sources including web-searches and lists provided by trade bodies and life sciences networks.  The short descriptions of the companies activities have been obtained from their own public website. The segmentation of company activity has been determined broadly by the company’s main business activities in the UK using publicly available information.  No confidential information has been included in the website but there is the option for companies to opt-out if they prefer.”

The BIA has long advocated that enabling global partners to better see the strength and opportunity within companies in the UK life science ecosystem should be core to UKTI’s life science mission and we hope that this new initiative can play a key role. We would also appreciate any feedback you have about this initiative or the website.

Remaining on the topic of websites, the latest version of the Scrip antimicrobial resistance microsite is now live – and free to view. Check it out here: http://www.scripintelligence.com/amr/

Last week’s synbio trade mission brought together a delegation of representatives, pictured above, from 14 UK companies – ranging from start ups to a seed fund and including the BIS synbio entrepreneur in residence plus representatives from the SynbiCITE Innovation and Knowledge Centre and Innovate UK – to exchange knowledge and ideas over three immersive days in San Francisco while visiting local companies and the SynBioBeta conference.

A highlight for all was the opportunity to hear from keynote speaker J Craig Venter on his body of work from the last decades and where he is turning his attention to now, such as remote DNA sequencing, which he terms ‘bio-teleportation’, with a view to testing for life on Mars. Keep an eye out for our blogs from the mission which will be published shortly.

I’ve already heard some very positive feedback from delegates about the opportunities they’ll be following up as a result of the trade mission. Together with BioPartner UK we run many missions throughout the year so if you’re interested do take a look.

It was also good to see Jeremy Hunt talking about the importance of innovation in his speech at the King’s Fund, recognising the role that it plays in unlocking personalised cures for illnesses and stating “we want the NHS to spearhead a global revolution in personalised medicine based on individual genetic characteristics”. He also used the speech to announce Dame Fiona Caldicott as the new National Data Guardian.

The Alliance for Regenerative Medicine’s 2nd Annual EU Advanced Therapies Investor Day also took place last Thursday and I was pleased to be able to attend. It was a great day and fantastic to see presentations from BIA members Oxford BioMedica and ReNeuron, in addition to a keynote speech from GE Healthcare Life Sciences President and CEO, Kieran Murphy. For those of you who are interested, the webcast of all talks, panels and company presentations will be available on the event website in a couple of weeks.

Look forward  to seeing some of you at our Manchester breakfast on Wednesday and the BIA will be out in force at the FT, Jefferies, ABPI, Consillium and AMRC events through the week.

Best,

Steve

On Tuesday it was announced that a paralysed man has been able to walk again after a pioneering therapy that involved transplanting olfactory ensheathing cells (OECs) from his nose to his spinal cord. Darek Fidyka, who was paralyzed from the chest down four years ago, can now walk using a frame.

The treatment, a world first, was carried out by surgeons in Poland in collaboration with scientists from University College London. Watch this week’s video from BBC News, below, to find out more about the therapy.

Do you have a video you would like the sector to see? Contact us.