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The genomic revolution heralded by the completion of the human genome project is providing unprecedented knowledge of the underlying genetic basis of disease. This understanding has provided an opportunity to directly treat the genetic causes of disease using Nucleic Acid therapies (NATs), which offer the opportunity to manipulate the human genome through replacement of damaged or absent genes, suppression of deleterious genes or direct editing of genetic codes in vivo. These therapies offer precision genomic treatments with wide application in areas of high unmet medical need with critical importance to society, particularly degenerative disease, rare diseases and cancer. While there have been a number of recent high-profile nucleic acid therapy drug approvals (e.g. Eteplirsen FDA approval 2016, Nusinersen FDA approval 2016, Patisiran FDA approval 2018), success has been modest and confined mainly to liver and local delivery indications. It is widely accepted that for nucleic acid medicines to truly effect a paradigm shift in healthcare the major scientific and industrial challenge of effective nucleic acid drug delivery must be overcome. To date, for example, siRNA delivery in humans has reliably been demonstrated only in liver hepatocytes – but work in primates has already shown encouraging outputs for delivery into kidney, lung, heart, skeletal muscle and leukocyte populations. Further development of NA delivery systems will allow more accurate dosing and targeting of these therapeutics to a broader range of specific cells and tissues, reducing or eliminating toxicity.

Given the high risk and cost of developing nucleic acid drug delivery technologies, it is not feasible for this work to proceed as part of any individual therapy development programme and the UK Government investment in the Nucleic Acid Therapy Accelerator is therefore essential in developing critical mass, bringing together key stakeholders and incentivising collaboration, catalysing and de-risking development of next generation nucleic acid medicines.

 

  • Learn about advances in delivery mechanisms for nucleic acid therapies
  • Become part of a growing network of experts in nucleic acid therapies within the UK and internationally
  • Hear about partnering opportunities with UKRI/MRC Nucleic Acid Therapy Accelerator and other key stakeholders
  • Develop your understanding of taking NATs from bench to bedside

Executives, Directors, VPs, Heads, Principals, Managers of:

RNA Biology/Discovery
Novel Therapeutic Modalities
Innovation Technologies
Formulation and Drug Delivery
Clinical Research and Development
Cell Biology

 

Accanis Therapeutics; Advanced Cell Diagnostics – A Bio-Techne Brand; Aescap 2.0; Alnylam Pharmaceuticals UK & Ireland; AmpTec GmbH; Aseptic Technologies SA; AstraZeneca; Bachem AG; BianoGMP; BioNTech IMFS GmbH; BioNTech RNA Pharmaceuticals; Boehringer Ingelheim Pharma GmbH & Co. KG; Calithera Biosciences; CEPI - Coalition for Epimemic Prepardness Innovation; Chemgenes Corporation; Curevac Ag; Dicerna Therapeutics.; Duke University School of Medicine; Ethris GmbH; Exicure Inc.; F.Hoffmann-La Roche AG; GSK; Horizon Discovery; InteRNA Technologies BV; Intertek; Lipocalyx; MedImmune; MHRA; MiNA Therapeutics; N4 Pharma UK Limited; New England Biolabs; NHS Blood and Transplant Authority; Nitto Avecia; NOF Europe GmbH; Nouscom SRL; ProQR Therapeutics; RCSI; RCSI Royal College of Surgeons in Ireland,; RNA Medco; ROCHE; Roche Diagnostics Deutschland GmbH; Roche Innovation Center; Russell Publishing Ltd; Sanofi; Sanofi-Aventis Deutschland GmbH; Takeda Development Centre Europe Ltd.; TCR Solutions; The Italian Institute of Technology; The RNA Medicines Company; Thermo Fisher Scientific; University Medicine Mainz Hospital; University Of Portsmouth; University Of Surrey; Yuria Pharma;

Workshop programme

8:30 Registration & Coffee

9:00 Opening Remarks

9:10 Encapsulation of Cargos for Effective Delivery

  • Discuss the pros on cons of a variety of encapsulation approaches, from lipid nanoparticles, exosomes and polymers
  • Methods to achieve preferential uptake
  • Cell biology of uptake: what we know and how it aligns with the current and future encapsulation approaches.
  • Models for in vivo testing of encapsulation approaches: IV/ CSF/ IM delivery
  • Safety concerns around encapsulation
     
  • Ritwick Sawakar

    Ritwick Sawakar, MRC Investigator, MRC Toxcology Unit, University Of Cambridge
    View Bio

    Nick Lench

    Nick Lench, Executive Director, UKRI/MRC Nucleic Acid Therapy (NATA)
    View Bio

    Peter Oliver

    Peter Oliver, Head of Biology, UKRI/MRC Nucleic Acid Therapy (NATA)

    9:50 Targeting Oligos with Conjugations (Pt 1- Antibodies and Nanobodies)

  • What are the challenges in delivery of nucleic acid therapies via antibody or nanobody?
  • Identifying and targeting tissue and cell receptors to achieve specific delivery
  • Does the cost outweigh the benefit for antibody-oligo conjugate drugs?
  • Discovery of new antibody-conjugates for reaching recalcitrant tissues
  • Local/ systemic immune system activation with antibody conjugates
  • Nick Lench

    Nick Lench, Executive Director, UKRI/MRC Nucleic Acid Therapy (NATA)
    View Bio

    Ritwick Sawakar

    Ritwick Sawakar, MRC Investigator, MRC Toxcology Unit, University Of Cambridge
    View Bio

    Peter Oliver

    Peter Oliver, Head of Biology, UKRI/MRC Nucleic Acid Therapy (NATA)

    10:30 Morning Coffee

    11:00 Targeting Oligos with Conjugations (Pt 2- Peptides, Lipids and Aptamers)

  • What are the challenges in delivery of nucleic acid therapies via antibody or nanobody?
  • Identifying and targeting tissue and cell receptors to achieve specific delivery
  • Does the cost outweigh the benefit for antibody-oligo conjugate drugs?
  • Discovery of new antibody-conjugates for reaching recalitrant tissues
  • Local/ systemic immune system activation with antibody conjugates
     
  • Nick Lench

    Nick Lench, Executive Director, UKRI/MRC Nucleic Acid Therapy (NATA)
    View Bio

    Ritwick Sawakar

    Ritwick Sawakar, MRC Investigator, MRC Toxcology Unit, University Of Cambridge
    View Bio

    Peter Oliver

    Peter Oliver, Head of Biology, UKRI/MRC Nucleic Acid Therapy (NATA)

    11:40 Design of Pre-Clinical Toxicology Studies

  • This session will explore current and future considerations for design of pre-clinical toxicology packages that are necessary and sufficient for regulatory approval.
  • We will discuss development of alternative models for toxicology eg organoids, organ-on-chip technologies
  • Standardised assays for the assessment of oligo safety;
  • Understanding and resolving the mechanisms of oligo toxicity in humans e.g. thrombocytopaenia; inflammatory responses; hybridisation-mediated off-target effects;
  • Understanding the biochemistry of oligos in cells e.g. endosomal escape; epigenetic effects.
  • Nick Lench

    Nick Lench, Executive Director, UKRI/MRC Nucleic Acid Therapy (NATA)
    View Bio

    Ritwick Sawakar

    Ritwick Sawakar, MRC Investigator, MRC Toxcology Unit, University Of Cambridge
    View Bio

    Peter Oliver

    Peter Oliver, Head of Biology, UKRI/MRC Nucleic Acid Therapy (NATA)

    12:20 Closing Remarks

    +

    FEATURED SPEAKERS

    Nick Lench

    Nick Lench

    Executive Director, UKRI/MRC Nucleic Acid Therapy (NATA)
    Peter Oliver

    Peter Oliver

    Head of Biology, UKRI/MRC Nucleic Acid Therapy (NATA)
    Ritwick Sawakar

    Ritwick Sawakar

    MRC Investigator, MRC Toxcology Unit, University Of Cambridge

    Nick Lench

    Executive Director, UKRI/MRC Nucleic Acid Therapy (NATA)
    Nick Lench

    Nick is a co-founder of Congenica Limited, a genomic medicine and digital health company based at the Wellcome Genome Campus, Cambridge and held positions of COO and CSO prior to his appointment at NATA. Nick has worked in academia, industry and the NHS and has extensive experience in personalised medicine and clinical diagnostics. Prior to Congenica, Nick was Director of the NE Thames Regional Genetics Service at Great Ormond Street Hospital for Children, London. Nick was awarded a personal chair in Medical Genetics at Cardiff University, is currently an honorary reader in Genetics and Genomic Medicine at UCL and a fellow of the Royal College of Pathologists.

     

    Peter Oliver

    Head of Biology, UKRI/MRC Nucleic Acid Therapy (NATA)
    Peter Oliver

    Ritwick Sawakar

    MRC Investigator, MRC Toxcology Unit, University Of Cambridge
    Ritwick Sawakar

    Ritwick studied Microbiology and Biochemistry in Mumbai (India) and obtained his PhD in 2010 from Indian Institute of Science, Bangalore. Ritwick then moved to the Department of Biosystems Science and Engineering of ETH-Zürich in Basel (Switzerland) as a postdoctoral fellow with Renato Paro. In 2014, Ritwick started his own independent group at the Max Planck Institute of Immunobiology and Epigenetics in Freiburg (Germany), before moving to the MRC Toxicology Unit in 2019. Ritwick received the ERC Consolidator Grant in 2018 and Alfred Tissières Young Investigator Award in 2019.

    VENUE

    Copthorne Tara Hotel

    Scarsdale Place, Kensington, London, United Kingdom

    The Copthorne Tara Hotel London Kensington is an elegant contemporary four-star hotel in prestigious Kensington, located just a two minutes walk from High Street Kensington underground station, making exploring easy. The hotel offers well-appointed and comfortable guest rooms combining Standard, Superior and Club accommodation. Club rooms offer iconic views over the city and include Club Lounge access for complimentary breakfast and refreshments. Guests can sample the authentic Singaporean, Malaysian and Chinese cuisine at Bugis Street, traditional pub fare at the Brasserie Restaurant & Bar or relax with a delicious drink at West8 Cocktail Lounge & Bar.

    The Copthorne Tara Hotel boasts 745 square meters of flexible meeting space, consisting of the Shannon Suite and the Liffey Suite, ideal for hosting conferences, weddings and social events. Facilities include access to the business centre 24 hours a day, fully equipped fitness room, gift shop, theatre desk and Bureau de Change. With ample onsite parking outside the London congestion charge zone and excellent transport links via Heathrow Airport, the hotel is the perfect location for business or leisure stays. The hotel is within close proximity to the shops of High Street Kensington, Knightsbridge and Westfield London, Olympia Conference Centre, Royal Albert Hall, Kensington Palace and Hyde Park.

     

    A number of our clients have been approached by third party organisations offering to book hotel rooms. We would advise that you do not book through them as they are not representing the SAE Media Group. SAE Media Group books all hotel rooms directly. If you are approached by a third party organisation then please contact us before making any bookings. If you have already booked a hotel room using a third party organisation, we would highly recommend contacting the hotel you were booked into to ensure a booking has been made for you. We would also advise you to please check the terms and conditions of the booking carefully.
    HOTEL BOOKING FORM

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    Copthorne Tara Hotel

    Scarsdale Place
    Kensington
    London W8 5SR
    United Kingdom

    Copthorne Tara Hotel

    The Copthorne Tara Hotel London Kensington is an elegant contemporary four-star hotel in prestigious Kensington, located just a two minutes walk from High Street Kensington underground station, making exploring easy. The hotel offers well-appointed and comfortable guest rooms combining Standard, Superior and Club accommodation. Club rooms offer iconic views over the city and include Club Lounge access for complimentary breakfast and refreshments. Guests can sample the authentic Singaporean, Malaysian and Chinese cuisine at Bugis Street, traditional pub fare at the Brasserie Restaurant & Bar or relax with a delicious drink at West8 Cocktail Lounge & Bar.

    The Copthorne Tara Hotel boasts 745 square meters of flexible meeting space, consisting of the Shannon Suite and the Liffey Suite, ideal for hosting conferences, weddings and social events. Facilities include access to the business centre 24 hours a day, fully equipped fitness room, gift shop, theatre desk and Bureau de Change. With ample onsite parking outside the London congestion charge zone and excellent transport links via Heathrow Airport, the hotel is the perfect location for business or leisure stays. The hotel is within close proximity to the shops of High Street Kensington, Knightsbridge and Westfield London, Olympia Conference Centre, Royal Albert Hall, Kensington Palace and Hyde Park.

     

    HOTEL BOOKING FORM

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    WHAT IS CPD?

    CPD stands for Continuing Professional Development’. It is essentially a philosophy, which maintains that in order to be effective, learning should be organised and structured. The most common definition is:

    ‘A commitment to structured skills and knowledge enhancement for Personal or Professional competence’

    CPD is a common requirement of individual membership with professional bodies and Institutes. Increasingly, employers also expect their staff to undertake regular CPD activities.

    Undertaken over a period of time, CPD ensures that educational qualifications do not become obsolete, and allows for best practice and professional standards to be upheld.

    CPD can be undertaken through a variety of learning activities including instructor led training courses, seminars and conferences, e:learning modules or structured reading.

    CPD AND PROFESSIONAL INSTITUTES

    There are approximately 470 institutes in the UK across all industry sectors, with a collective membership of circa 4 million professionals, and they all expect their members to undertake CPD.

    For some institutes undertaking CPD is mandatory e.g. accountancy and law, and linked to a licence to practice, for others it’s obligatory. By ensuring that their members undertake CPD, the professional bodies seek to ensure that professional standards, legislative awareness and ethical practices are maintained.

    CPD Schemes often run over the period of a year and the institutes generally provide online tools for their members to record and reflect on their CPD activities.

    TYPICAL CPD SCHEMES AND RECORDING OF CPD (CPD points and hours)

    Professional bodies and Institutes CPD schemes are either structured as ‘Input’ or ‘Output’ based.

    ‘Input’ based schemes list a precise number of CPD hours that individuals must achieve within a given time period. These schemes can also use different ‘currencies’ such as points, merits, units or credits, where an individual must accumulate the number required. These currencies are usually based on time i.e. 1 CPD point = 1 hour of learning.

    ‘Output’ based schemes are learner centred. They require individuals to set learning goals that align to professional competencies, or personal development objectives. These schemes also list different ways to achieve the learning goals e.g. training courses, seminars or e:learning, which enables an individual to complete their CPD through their preferred mode of learning.

    The majority of Input and Output based schemes actively encourage individuals to seek appropriate CPD activities independently.

    As a formal provider of CPD certified activities, SAE Media Group can provide an indication of the learning benefit gained and the typical completion. However, it is ultimately the responsibility of the delegate to evaluate their learning, and record it correctly in line with their professional body’s or employers requirements.

    GLOBAL CPD

    Increasingly, international and emerging markets are ‘professionalising’ their workforces and looking to the UK to benchmark educational standards. The undertaking of CPD is now increasingly expected of any individual employed within today’s global marketplace.

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    We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@saemediagroup.com

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