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Oligonucleotide Therapies- Overcoming the Challenges of Delivery
20 September 2022
Oligonucleotide Therapies- Overcoming the Challenges of Delivery

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.

 

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

Arpan Desai

Managing Director, Nanovation Therapeutics
Arpan Desai

Arpan is Managing Director of Nanovation Therapeutics, he has over a decade of experience leading project and platform initiatives in the RNA therapeutics space. He has a track record of creating innovative delivery approaches and progressing LNP-based projects from discovery into the clinic across multiple therapeutic areas. Before joining Nanovation he served as a director at AstraZeneca, where he was been involved in leading projects and platform initiatives related to ASOs, anti-miRNA, mRNA for gene editing, vaccines and protein replacement. This has included playing key roles in collaborations with Moderna, Ionis, VaxEquity and Regulus. He established and led a global matrix team of RNA delivery scientists with the mission of creating new delivery technologies for mRNA. Much of the key infrastructure for the design and optimization of LNPs was achieved under his leadership. Moreover, several key advances in LNP platform science were obtained, such as the development of a proteomic based screening method for in vivo LNP assessment, evaluation of higher throughput methods for DMTA of LNPs and evaluation of almost 100 external technologies to name a few. His work on understanding how LNPs interact with different tumour cells facilitated the selection of preclinical models to assess LNP performance. This ultimately resulted in AZs first LNP based mRNA project progressing to the clinic – MEDI1191 (partnered with Moderna Therapeutics)

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

Pete comes to NATA with over 25 years’ experience in cell and molecular biology. After a degree in Biochemistry and a PhD in Genetics, he led projects studying new models of neurological disorders at the University of Oxford, before becoming a group leader at the Department of Physiology, Anatomy and Genetics in 2012 funded by an ERC Consolidator Award. His group’s work in rare disease modelling using the mouse resulted in a new position as an MRC Programme Leader at the MRC Mammalian Genetics Unit in 2017. Pete joined NATA in February 2022, bringing an understanding of multi-disciplinary approaches to translational research. Pete remains a visiting academic member of the University of Oxford and is an active co-investigator in the MRC National Mouse Genetics Network.

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.

Workshop agenda

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8:30

Registration & Coffee

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9:00

Opening Remarks

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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, MRC Investigator, MRC Toxcology Unit, University Of Cambridge

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

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

    Arpan Desai, Managing Director, Nanovation Therapeutics

    clock

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

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

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

    Arpan Desai, Managing Director, Nanovation Therapeutics

    clock

    10:30

    Morning Coffee

    clock

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

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

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

    Arpan Desai, Managing Director, Nanovation Therapeutics

    clock

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

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

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

    Arpan Desai, Managing Director, Nanovation Therapeutics

    clock

    12:20

    Closing Remarks


    Managing Director
    Nanovation Therapeutics
    Executive Director
    UKRI/MRC Nucleic Acid Therapy (NATA)
    Head of Biology
    UKRI/MRC Nucleic Acid Therapy (NATA)
    MRC Investigator, MRC Toxcology Unit
    University Of Cambridge

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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.

     

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