Abstracts and speaker bios

John is Regius Professor of Medicine at Oxford University. His research interests are in autoimmune diseases and immunology, where he has contributed to our understanding of immune activation in autoimmune diseases. He served as President of the Academy of Medical Sciences from 2006 to 2011 and chaired the Office for the Strategic Coordination of Health Research until 2017. In 1993 he founded the Wellcome Trust Centre for Human Genetics. In 2001 he was appointed non-executive director of Roche Holding AG and in 2008 he joined the Gates Foundation Global Health Advisory Board, which he has chaired since 2012. In 2011 Sir John was appointed a UK Life Sciences Champions by the PM. He sits on the board of Genomics England Limited and chairs its Science Advisory Committee. He was knighted (GBE) in the 2015 New Year Honours for services to medicine, medical research and the life science industry. 

Sofía is Full Professor of Bioethics at Universidad del Desarrollo, Santiago, Chile (https://medicina.udd.cl/centro-bioetica/persona/sofia-salas/). This center has recently been appointed as a PAHO/WHO Collaborative Centre for Bioethics (https://medicina.udd.cl/centro-bioetica/centro-colaborador-opsoms/). She is a member of the Faculty of Medicine Research Ethics Committee (https://medicina.udd.cl/centro-bioetica/sobre-el-centro/comite-de-etica/), a member of the Department of Ethics, Chilean Medical Association and President of the Advisory Committee on Research Ethics for the Chilean Ministry of Health.

Abstract

In the developed world we have access to a myriad of medicines and whilst we may have to wait a year or two post launch for bodies such as NICE to approve funding, there is relatively little delay in being able to access new medicine. What happens in the developing world can be very different. Lack of healthcare infrastructure, bureaucratic and lengthy review processes and lack of affordability make access to life-saving medicines difficult, costly and slow. Many pharma companies have found ways round many of these issues, including licensing regional pharma companies to enable them to manufacture and supply branded medicines at more affordable prices. Partnerships with NGOs also serve to enable faster access and deliver essential drugs to where they are most needed.

Laurence is founder and CEO of xim, developers of Lifelight software which enables standard smartphones to perform contactless measurement of blood pressure and other vital signs with clinical grade accuracy. Lifelight has been developed over five years and is currently undergoing regulatory approval.

Laurence has a long background in healthcare informatics strategy and implementation across Europe, USA and the Middle East. He is passionate about the human application of technology in terms of user experience, computer vision, machine learning and connected digital health. With a background in computer science and an MBA, he is an experienced entrepreneur and active mentor.

Abstract

The world of medical diagnostics and digital health is evolving rapidly and looks set to support clinicians to make even better clinical decisions and revolutionise patient interactions and engagement over the coming decades. This seminar will look at specific examples of new and emerging technologies, and examine their role in drug development programmes

Mene is the Executive Vice-President, R&D at AstraZeneca (AZ). Prior to this, he served as Executive Vice-President of AZ’s IMED Biotech Unit and Global Business Development, and held senior R&D roles at Wyeth and GSK. He led the transformation of R&D productivity at AZ through the development of the “5R” framework, resulting in a greater than four-fold increase in success rates compared to industry averages. He champions an open approach to working with academic and other external partners, transforming academic-industry collaboration.

Mene is a Fellow of, amongst others, the Academy of Medical Sciences and the Royal Society of Biology. He sits on the Council of the MRC, co-chairs the UK Life Sciences Council Expert Group on Innovation, Clinical Research and Data and is on the Boards of The Francis Crick Institute, Cambridge University and Dizal Pharma. In 2020 Mene was awarded a Knighthood for his services to UK science.

Abstract

The future treatment for many of today’s complex diseases lies in discovering new biology, understanding and challenging scientific hypotheses and evolving the way we design and conduct our clinical trials. Improving R&D productivity is critical to the success of future drug discovery and development, with failure rates from pre-clinical development to launch often in excess of 90 percent.

The advent of data science, artificial intelligence and digital technologies, coupled with ever more sophisticated platform technologies has the potential to increase the probability of success, and to discover new medicines more efficiently and deliver them more effectively.

Our ambition is to transform the lives of patients with improved outcomes and a better quality of life, through more effective treatment and prevention, ultimately working toward a cure for some of the world’s most complex diseases.

Georgina is the founder and Chairperson of ArchAngel MLD Trust. Having spent almost 20 years as a television producer, her life and career changed dramatically in 2013 when her daughter Ava was diagnosed with a rare lysosomal storage disorder, Metachromatic Leukodystrophy. When Ava was accepted onto a clinical trial of Gene Therapy for MLD in 2014, the experience led Georgina to form a charity, in order to support medical teams around the world working on treatments for MLD and to offer grants to affected families. ArchAngel is also currently spearheading a campaign to have all UK babies screened for MLD (and other rare diseases) at birth.

Abstract

The diagnosis of a rare disease can be a thunderbolt and the subsequent realisation that the condition has no effective treatment a direct lightening strike. However, thanks to increasing investment in orphan drug development projects, the world of rare diseases is beginning to become a less devastating place to find yourself. This lecture will outline one family’s hopeful journey through Gene Therapy for Metachromatic Leukodystrophy; from diagnosis and the challenges of accessing a clinical trial, to the inspiration which has led to the formation of a charity and a campaign calling for review and expansion of the entire UK New-born Screening system.

Laura will be joined by Cecilia Chisholm, Mahesh Kudari and Marc Watson to talk about careers and career paths through pharmaceutical medicine.

We have three speakers who are giving their experiences of different roles within pharma, as a springboard for discussions for those who may be interested in exploring a different role.

Areas covered:

• Clinical development
• Safety/Pharmacovigilance
• Regulatory
• Medical Affairs

Munir is David Weatherall Chair in Medicine at the University of Liverpool, and a Consultant Physician at the Royal Liverpool University Hospital. He is Director of the MRC Centre for Drug Safety Sciences, and Director of the Wolfson Centre for Personalised Medicine. He is an inaugural NIHR Senior Investigator, Fellow of the Academy of Medical Sciences in the UK, Commissioner on Human Medicines and is a non-executive director of NHS England and has been appointed as President of British Pharmacological Society. He was awarded a Knights Bachelor in the Queen’s Birthday Honours in 2015. His research focuses on personalised medicine, clinical pharmacology and drug safety.

Abstract

A key issue for the genomic revolution is how it will help in improving treatment for diseases.  As the number of people globally who have their genomes sequenced increases, the depth and breadth of information available will allow us to progress personalised or precision medicine, to ensure that patients get the right treatment at the right dose and at the right time.  This will be crucial in ensuring that we optimise the benefit-risk ratio of all therapies (new and old) maximizing efficacy and minimizing safety.  In some cases, genomic information will be directly relevant to the development of new therapies – this is an appropriate strategy given that drugs which are developed on the basis of genetic information seem to have a higher success rate to go to market.  Furthermore, we also need to consider how we can use genomics to help in stratifying early phase trials so that not only the correct drug, but also the correct dose can be identified.  Genomic information can also provide insights that may lead to re-purposing of medicines for diseases for which there is unmet medical need.  Genomic information may also be important in improving the use of drugs which have been available over many years to (a) change the intensity of therapy; and (b) improve the benefit-risk profile.

In summary, the genomic revolution offers many opportunities for therapeutics – the key issue will be to develop the evidence base which allows the genomic findings to be translated into patient care.

Bob Holland

Bob is highly experienced across all phases of Drug Discovery and Development, especially in the CNS and Oncology Therapy Areas.

A career-long interest in the utility of biomarkers and a desire to ensure that patients receive drugs with the mechanisms most likely to provide benefit led Bob to establish and lead AstraZeneca’s first Department of Personalised Healthcare & Biomarkers.  Subsequently, interested in the tremendous potential of next-generation sequencing and the practicalities of converting biomarkers into medically useful products, Bob became CMO of the molecular diagnostics company Oxford Gene Technology (now part of Sysmex).  Currently, Bob works as Senior Clinical Fellow at Sosei Heptares and is a member of the FPM Medical Devices and Diagnostics Expert Group.

Petar Gjorgiev

Petar worked as a general practitioner in Bulgaria before moving to the UK where he has pursued a career in pharmaceutical medicine.  Petar has worked as a licenced physician in the pharmaceutical industry since 2013 and has experience in all phases of clinical trials as a research physician and principal investigator. In 2016 he joined Amgen in his first role as a safety physician. Currently Petar works as a Global Safety Physician at AstraZeneca in the oncology therapeutic area.

Abstract

Advances in Medical Devices and In Vitro Diagnostics have the potential to generate improvements in healthcare of a magnitude at least comparable to those generated by new medicines.  However, delivering these improvements requires that the devices and diagnostics actually work as intended and provide the results claimed without adding risks of injury by the device or incorrect treatment through a wrong diagnostic result.

In this session we will briefly review the evolving regulatory landscapes (with a focus on diagnostics), and proposals and requirements for safety vigilance (especially for devices).  Important lessons must be drawn from the UK’s response to the Covid 19 pandemic and some of the difficulties we have experienced in the implementation of very advanced science will be used to stimulate discussion.

Sally-Anne Tsangarides

Sally-Anne has spent almost 35 years in the pharmaceutical industry, beginning in manufacturing and supply chain in ‘big pharma’. She soon transitioned to commercial roles and leadership, moving into areas of increasingly specialised medicine and, latterly, has led launches of five innovative products to address high unmet needs in paediatric rare conditions at three different companies. She believes it is an immense privilege to be responsible to launch the first potentially transformative AAV9 gene therapy from Novartis Gene Therapies into the UK and works with stakeholders in the clinic, patient organisations, the NHS and policymakers to deliver for affected families.

Abstract

The concept of gene therapy was introduced in the 1970s and the first gene therapy trial began in 1990 in a four-year-old girl with adenosine deaminase (ADA) deficiency. However, it took until 2012 for the European Medicines Agency to award the first ever licence for gene therapy in Europe, for the treatment of an ultra-rare blood disorder. There are now over 2,600 gene therapy clinical trials completed, ongoing or approved worldwide and horizon scanning indicates that NICE is due to assess between 27 to 33 advanced therapeutic medicinal products by 2023. The NHS provided its first gene therapy, to treat blindness, under routine commissioning early in 2020.

This seminar will present perspectives on the current state of UK readiness to develop and adopt gene therapies and the potential for these new health technologies to transform outcomes for patients with genetic conditions.

Guest speakers: Eric Alton, Professor of Gene Therapy and Respiratory Medicine at Imperial College, London and Chair of the NIHR ATMP Coordinating Group. Arseniy Lavrov, Clinical Development Head EMEA, Novartis Gene Therapies. Jayne Spink, Chief Executive, Genetic Alliance UK.

Eric Alton

Eric Alton is Professor of Gene Therapy and Respiratory Medicine at Imperial College, London, an Honorary Consultant Physician at the Royal Brompton Hospital and NIHR Senior Investigator. Over the past 20 years he has coordinated the UK Cystic Fibrosis Gene Therapy Consortium, which brings together all the key centres in the UK (Edinburgh and Oxford Universities and Imperial College, London) in a programme to deliver gene therapy for these patients. He is the Chair of the NIHR ATMP Coordinating Group which has pulled together a dozen of the key stakeholders in the field to catalyse coordination and avoid duplication.

Arseniy Lavrov

Arseniy, MD, PhD, FFPM is a board-certified neurologist and a pharmaceutical physician with 9 years of clinical and academia experience. He completed a PhD research in vascular dementia and vascular cognitive impairment. Dr Lavrov has a further 15 years of pharmaceutical industry experience with a focus on drug development in Neurosciences, across all phases of development from discovery to post approval. His experience involves translational,  clinical development and marketing authorization activities of gene therapies for neurological diseases.  His main areas of interest also include: rare neurological diseases; movement disorders; dementias and cognitive impairment. Dr Lavrov has also an interest and experience in digital biomarkers and endpoints. Previously Head Unit Physician and Clinical Development Head at Neurosciences Therapy Area Unit at GSK, Dr Lavrov is currently leading EMEA Clinical Development at Novartis Gene Therapies.

Jayne Spink

Jayne is Chief Executive of Genetic Alliance UK, a national alliance with over 200 member charities. Genetic Alliance UK is home to Rare Disease UK (which Jayne chairs) and SWAN UK which provides support to families who have a child or young person with complex needs and no diagnosis.

Jayne has a BSc and a PhD in Genetics. Following a period of postdoctoral molecular genetics research, she continued her career in central government working on range of genetics related policy areas  and as scientific secretary to the UK Gene Therapy Advisory Committee. Having served as an Associate Director of the Centre for Clinical Practice at England’s HTA body NICE, Jayne moved to the third sector in 2007 to take up the post of Director of Policy and Research with the UK Multiple Sclerosis Society and, immediately prior to joining Genetic Alliance UK, held the post of Chief Executive of the Tuberous Sclerosis Association (UK).

Jayne lives in London with her husband and two children. Jayne’s elder child, her son, was diagnosed soon after birth with Ebstein’s Anomaly (which occurs in 1 per 200,000 live births).

Michael Holmes is a Pharmaceutical Medicine Specialty Trainee and Associate Professor at University of Oxford. He studied Preclinical Medicine at University of St. Andrews and graduated with distinction in Clinical Medicine from The Royal Free & University College Medical School. Following working in the NHS as an academic Foundation/Core Medical Trainee, he graduated with distinction in Epidemiology from LSHTM and undertook a PhD in Genetic Epidemiology at UCL.

Michael’s work focuses on translational applications of human genetics, identifying modifiable risk factors and potential therapeutic targets. He was awarded the prestigious John French Lecture by the British Atherosclerosis Society in 2020.

Abstract

It costs ~$1bn to bring an investigational medicinal product (IMP) to market. Fewer than 1 in 10 IMPs succeed from phase 1 to marketing authorisation.

Novel approaches to characterising the effects of therapeutic targets outside of conventional trial settings may help forestall the high failure rate.

In this presentation, I will outline how applications of human genetics can inform each stage of drug development, providing early support of causation and characterising target-mediated intended and adverse effects.

Human genetics represents a burgeoning field of opportunity for informing the development of therapies and one that the Pharmaceutical Medicine community should embrace.

Jonathan is the Director of Policy at the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK. The MHRA Policy Division, which Jonathan heads, helps the Agency carry out its public health mandate in alignment with wider UK Government priorities and requirements. The Division leads for the Agency, and the UK Government, in the management of EU and International business relating to pharmaceuticals and medical devices regulation.
Prior to joining the Agency, Jonathan held a wide variety of policy posts since joining the UK Department of Health in 1990, including secondments to the Foreign Office and to the European Commission in Brussels; he has also headed policy teams in the UK Department of Health responsible for pharmaceutical industry policy and for private sector provision of healthcare services for NHS patients; he was also the Department’s Head of European Affairs for 4 years to 2009.

Abstract

This plenary session will give the delegates a vital and timely update on how the UK regulations for medicines development and approval are being adapted/modified to be fit-for-purpose post-Brexit.