Precision Medicine Day Two


Precision 2018 Day 2

08:00 Registration opens

Opening keynotes: Precision medicine stories

Paul Peter Tak

Towards prevention of autoimmune disease: the example of rheumatoid arthritis

  • Predictors for rheumatoid arthritis (RA) development will be presented
  • PRAIRI study is the first evaluating the effects of a biopharmaceutical in subjects at risk of developing RA
  • A single infusion of 1000 mg rituximab delays the development of arthritis up to 12 months
Mene Pangalos FRSB FMedSci

Stories from AstraZeneca’s innovative medicines and early development biotech unit

  • Quality, not quantity: Improving productivity by implementation of a company-wide framework and a precision approach
  • A success story of precision medicine in oncology and moving into other therapy areas – identifying the right target, in the right patients, at the right time for the best outcome to improve patients’ lives
  • Identifying new drug targets following a genomic approach across all therapy areas
Kari Stefansson

From discovery genetics to precision medicine

10:30 Networking Break

round tables

Roundtable discussion session

This roundtable session will run as two, 30 minute rotations, enabling you to join 2 roundtables of your choice
Table 4: Cost and benefits of precision medicine
Alastair Kent OBE

Alastair Kent OBE, Ambassador, Genetic Alliance U.K.

Table 6: Diagnostics – where precision medicine will take us

Speed Networking

A fun, exciting and effective way to make a lot of initial connections (in a very different environment from the standard business networking meetings)
Panel discussion

13:00 Networking Lunch

Break out panel: Patient involvement in precision medicine
  • What are the benefits of involving the public in precision medicine
  • The patient experience with precision medicine and patient involvement
  • How clinicians can improve the patient experience
  • What are the barriers to involvement and how can they be overcome

Drug development

Precision Medicine Therapeutic Development


Applying human genetics and big data into drug discovery strategies

Adele Mitchell, Associate Director, Discovery Data Science, Eisai Inc
Precision Medicine - Oncology

Mendelian genomics and precision medicine efforts at Yale

  • Biomarker discovery for Mendelian and Cancer Genomics
  • Precision Medicine initiative/efforts at Yale
  • Present PM cases of liver and brain cancer
  • Impact of new and upcoming technologies on PM

Leveraging known causal human biology for discovering new drugs for orphan diseases

  • In many cases the underlying cause of orphan diseases is known, often being genetic nature
  • However, that the trajectory of disease causing events and a validated therapeutic intervention point are also known does not necessarily follow
  • I will discuss the key requirements and approaches for addressing diseases where the essential causative events are known as well outline how this should differ for diseases where this is not the case
Precision Medicine - Oncology

The Human Protein Atlas - implications for human biology and precision medicine

  • Knowledge of the spatial distribution of proteins is essential to understand human biology in health and disease
  • The Human Protein Atlas constitutes an open-access knowledge-based resource, focusing on an integrated omics approach for in situ detection of human proteins down to the single cell level
  • All human protein coding genes have been classified using a combination of genomics, transcriptomics, proteomics and antibody-based profiling, and used for studying global protein expression patterns

The evolution of Pharmacogenetics (PGx) as a tool to support drug development

  • What have we learned about PGx over the last 10 years
  • How best can we utilise this to support drug development going forward
  • The utility of clinical trials and PGx to support target validation
Precision Medicine - Oncology

CRUK Stratified Medicine Programme: Experiences of delivering a national precision medicine initiative

  • Overview and update of CRUK’s Stratified Medicine Programmes and National Lung Matrix Trial
  • Challenges faced in delivering a Precision Medicine initiative
  • How SMP2 has approached addressing some of these challenges
  • Next steps for SMP2 and Precision Medicine Initiatives

Implementing precision medicines: what are the challenges?

  • Sharing of case-studies/examples on challenges and solutions in precision medicine development through to integration in treatment pathways, including;
  • Biomarker discovery
  • Clinical trial design and delivery
  • Drug-diagnostic co-development
  • Precision Medicine launch and delivery to the patient
Precision Medicine - Oncology

Precision medicine in oncology: what is it exactly and where are we?

  • A better understanding of cancer biology has opened the door of precision medicine in oncology
  • The identification of specific molecular alterations in some tumour types has translated into major therapeutic achievements
  • High throughput technologies have allowed the elaboration of prognostic signatures that led to treatment de-intensification in breast cancer
  • However, it has not been demonstrated to date that the use of high throughput technologies to guide therapy improved patients’ outcomes
Vivienne Parry

15:50 Networking break

Breakout Panel: Inside the ethics committee – the ethics behind using precision medicine
  • Panel exploring the various ethical implications surrounding precision medicine
  • Big data and patient data usage
  • Best practice for ethical decisions

Drug development

Precision Medicine Therapeutic Development


“The Center for Accelerated Drug Discovery – A strategic business partnership between the American Heart Association and Lawrence Livermore National Laboratory”

  • The two organizations will leverage one of the world’s most powerful supercomputers to accelerate drug discovery
  • Lawrence Livermore National Laboratory Scientists and engineers will create a simulated environment that rapidly and precisely predicts how drugs bind to their target proteins to generate a robust drug pipeline of new and targeted therapies
  • This partnership is part of the newly formed Center for Accelerated Drug Discovery, within the American Heart Association Institute for Precision Cardiovascular Medicine
Precision Medicine - Rare Disease

Precision and recall oncology: combining multiple gene mutations for improved identification of drug-sensitive tumours

  • There is a need for new methods able to better discriminate between sensitive and resistant tumours to a given cancer treatment from the genomic profiles of these tumours
  • Combining multiple gene mutations via machine learning results in better discrimination than that provided by the best single-gene marker in about half of the 127 analysed drugs
  • Importantly, the proportion of sensitive cell lines that are correctly predicted as sensitive (recall) of the single-gene marker was lower than that of the multi-gene marker in 93% of the drugs

Genomic scale drug design

  • First time in history we can have full genome binding affinity for molecules
  • This will shift the industry to a different design process, less risky, more precise
  • Opening of the single genome drug optimisation
Precision Medicine - Rare Disease

Precision medicine in severe asthma and its comorbidities: new kids on the block

  • Overview of the clinical problems: severe asthma and other severe "allergic" diseases (chronic spontaneous urticaria, nasal polyposis, atopic dermatitis);
  • New insights on endotypes and phenotypes of severe asthma and its comorbidities
  • From endotype to phenotypes, and back Novel biological therapies and the need of predictive biomarkers (with novel personal and literature data)
Precision Medicine - Rare Disease

Need for patient registries for rare disease clinical trials and how to integrate them into the EMR

  • Hear how Sanford Research built a registry for batten disease and how they are expanding it and implementing it into electronic health records
  • What do these registries mean for clinical trials in the rare disease field?
  • How do approaches to registries and electronic health systems differ globally?

Academic built compounds (ABC) – with the move to drug discovery platforms are we forgetting our ABCs?

  • An overview of the Oncology drug development landscape - comparing approaches and exhibiting the successes from Academia
  • MicroQuin is offering a novel approach and first-in-class treatment to ALL Breast Cancers.  This will spotlight MicroQuin’s efforts and explain why this can’t be found using a platform approach
  • Examining the Oncology pipeline: The next generation drugs and why ABC is still unbeatable
Precision Medicine - Rare Disease

The bio-inspired artificial pancreas for treatment of diabetes in the home

  • Microchip technology can create systems inspired by biology to provide control of disease
  • The Bio-inspired Artificial Pancreas has been developed and clinically validated in people with diabetes
  • Our device can improve glucose control and reduce hypoglycaemia
  • This improves quality of life in subjects with diabetes and reduces secondary complications

17:50 End of day 2: Poster viewing and networking drinks reception

last published: 26/Apr/18 12:25 GMT




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World Precision Medicine Congress


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