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Oligonucleotide Therapeutics and Delivery
9 September - 10 September 2024
Oligonucleotide Therapeutics and Delivery

 

Step into the forefront of innovation at SAE's 4th annual Oligonucleotide Therapeutics and Delivery conference!

Deep dive into the latest breakthroughs in oligonucleotide development, regulatory compliance, and CMC considerations. Discover cutting-edge strategies to elevate oligonucleotide discovery and precision targeting. Explore optimized delivery methodologies for oligonucleotide-based therapies, propelling the frontier of oligonucleotide drug development forward.

Don't miss your chance to be part of this transformative event on 9 – 10 September.

Join us and shape the future of therapeutic advancement!

Why should you attend?

  • Benchmark against leading big pharma case studies to advance your manufacturing and analytical strategies for advanced oligonucleotide synthesis
  • Explore the potential of industry cross collaboration with insights from working groups, regulatory bodies and senior big pharma and biotech experts leading the way in driving advancements for genetic medicine
  • Learn how AI is propelling drug discovery forward for sequence design and unlocking undruggable proteins and how this can translate to your pipelines
  • Engage in the latest drug delivery technologies, optimising target specificity for RNA therapeutics and vaccines
     

 

FEATURED SPEAKERS

David Tew

David Tew

Senior Scientific Director, Enzyme Engineering and Biocatalysts (E2B), GSK
Nagy Habib

Nagy Habib

Professor of Surgery, Imperial College London, Co-Founder, MiNA, Apterna and Dawn Therapeutics
Peter Smith

Peter Smith

Director, New Modalities Chemistry, AstraZeneca
Samir Ounzain

Samir Ounzain

Senior Chief Executive Officer and Scientific Co-Founder, HAYA Therapeutics
Thierry Dorval

Thierry Dorval

Head of Data Sciences & Data Management, Servier Pharmaceuticals
Thomas Thum

Thomas Thum

Founder, Chief Scientific Officer and Chief Medical Officer, Cardior Pharmaceuticals GmbH

Alessandro Bonetti

Principal Scientist, R&D Biopharmaceuticals, AstraZeneca
Alessandro Bonetti

Dr. Alessandro Bonetti obtained his Master degree in Medical Biotechnologies at the University of Naples in 2001 and moved to Helsinki for his PhD studies where he investigated the genetic prediposition to multiple sclerosis in Nordic populations. He then moved to RIKEN Yokohama to study the biological role of long non-coding. During that time he developed numerous technologies to identify the functional relevance of non-coding transcripts in physiological and pathological conditions. Subsequently he joined AstraZeneca to lead the transcriptomic studies in the Oligonucleotide platform. In October 2022 he was appointed Principal Scientist to lead the non-coding initiative in AZ.

Daniel Elbaum

Chief Scientific Officer, QurAlis
Daniel Elbaum

Daniel (Dan) Elbaum, PhD, is a drug discovery and development leader with nearly three decades of experience. He thrives on making new discoveries and connections and has made significant contributions in the fields of oncology, inflammation, and orphan diseases. He has worked in large organizations including Pfizer and Amgen, as well as several start-up companies including Kinetix Pharmaceuticals and FoldRx Pharmaceuticals (acquired by Pfizer). He was senior director, chemistry and portfolio management, and co-head of the research and non-clinical development group at Retrophin. To keep abreast of cutting-edge research, he actively maintains close ties with the academic and start-up communities.

Dan earned his PhD and undergraduate degrees in chemistry from Harvard University, and conducted postdoctoral research at the University of Pennsylvania. An inventor on more than 25 patents and an author on more than 15 peer-reviewed publications, Dan is a mentor in the Kindle Mentors Program at Boston University and a longstanding member of the American Chemical Society and the Association for the Advancement of Science.

David Evans

CSO, Sirnaomics, Inc
David Evans

David M. Evans, Ph.D, Co-founder and CSO, Sirnaomics Inc. Gaithersburg, MD. Dr. Evans has over 25 years experience in drug target and small molecule discovery for oncology, infectious diseases and CNS therapeutics. He has been involved in the RNAi field since 2003. As Head of Drug Discovery for the Cancer Drug Development Lab for TGEN and as Sr. Director, RNAi Discovery and Therapeutic Services group, Dharmacon group, Thermo Fisher Scientific (TFS), he built HTS operations to identify targets that synergized with known oncology therapeutics. In 2008 Davidwas a cofounder of Sirnaomics and has led the R&D group with afocus on using siRNAs as therapeutics in their own right. Sirnaomics now has several candidates in clinical testing and a
rich pipeline of preclinical products. David received his BSc(1983) and PhD (1987) from Imperial College, London and has published a number of peer reviewed papers and review articles.
 

David Tew

Senior Scientific Director, Enzyme Engineering and Biocatalysts (E2B), GSK
David Tew

David Tew is a Senior Scientific Director at GSK. David has 35 years of experience in the pharmaceutical industry including early drug discovery, mechanistic enzymology, assay development, reagent generation and the application of Synthetic Biology. Most recently David has been leading a project to apply Synthetic Biology to redesign the manufacture of therapeutic oligonucleotides. This has resulted in a platform approach to therapeutic oligonucleotide synthesis and manufacture that is both scalable and more sustainable than current approaches.
In 2018 David was appointed as The Royal Society Entrepreneur in Residence at Bristol University working with BrisSynBio and the Bristol Biodesign Institute. David held this position until 2022.
During the SARS-CoV-2 pandemic, David lead the innovation workstream for the Cambridge COVID-19 Testing Centre, a collaboration between AstraZeneca, GlaxoSmithKline and the University of Cambridge.
 

Heinrich Haas

Chief Technology Officer, NeoVac
Heinrich Haas

Joanna Hemming Taylor

Associate Principal Scientist, AstraZeneca
Joanna Hemming Taylor

Joanna Hemming Taylor graduated with a MChem in Chemistry with industrial experience from the University of Manchester in 2009, followed by a PhD in biophysical and computational chemistry from University College London with an emphasis on characterisation of lipids and peptides at the air-liquid interface. In 2014 Joanna joined AstraZeneca and has worked as an analytical project lead on multiple early phase clinical projects, from small molecule therapeutics to larger synthetics such as peptides and drug-dendrimer conjugates. Most recently, Joanna has been responsible for leading the analytical development of siRNA projects within Early Chemical Development.

Nagy Habib

Professor of Surgery, Imperial College London, Co-Founder, MiNA, Apterna and Dawn Therapeutics
Nagy Habib

For over three decades Nagy has been at the forefront of clinical research and clinical practise in cancer. He pioneered the first clinical trial in the use of adenovirus and plasmid for the treatment of liver cancer, as well as the use of plasmid gene therapy in hydrodynamic gene therapy delivery. He was the first in Europe to perform a clinical trial with the use of adult CD34+ stem cells in patients with liver failure and stroke.
Nagy is a founder and was the Head of R&D of Mina therapeutics whilst at Mina he was driving the development of saRNA drug (a new class of medicines) which is currently being trialled in patients with liver cancer in eight UK centres, and sites in Singapore and Taiwan (OUTREACH study, Clinical Trials.gov ID NCT02716012) and in a second trial in patients with solid tumours (TIMEPOINT study, ClinicalTrials.gov ID NCT 04105335) in the UK, USA, Europe, Singapore and Taiwan.
He has published widely in gene therapy, stem cell therapy, oligonucleotides, endoscopy and surgery. Currently he is the CEO of Apterna Limited, a company focussed on novel oligonucleotide delivery and Dawn Therapeutics specialising in gene therapy. Previously Nagy was founder and Chairman of EMcision Limited (acquired by Boston Scientific Inc in 2018).
Nagy is Lead Clinician and Head of the Department of HPB Surgery at Imperial College London.

Peter Smith

Director, New Modalities Chemistry, AstraZeneca
Peter Smith

Peter Smith is currently Director, New Modalities Chemistry within AstraZeneca’s Early Chemical Development group based in Macclesfield, UK. Peter has a background in synthetic organic chemistry (BSc Hons Glasgow, PhD Heriot-Watt, PDRA Sussex) and has worked at AstraZeneca for over 20 years in a variety of chemistry roles. During 2016, Peter took the position of early-phase Oligonucleotide chemistry lead, responsible for chemical development and drug substance manufacturing of AZ’s early-phase oligonucleotide clinical portfolio, and since then has progressed to his current role where he has accountability across the fields of oligonucleotides, peptides, ADC’s and ionizable lipids.

Priya Karmali

Chief Technology Officer, Capstan Therapeutics
Priya Karmali

Priya Karmali, PhD. is Chief Technology Officer at Capstan Therapeutics and leads all aspects of technology development. Priya has over 20 years of experience in the field of lipid nanoparticle delivered nucleic acid therapeutics from discovery through development. Prior to Capstan, Priya served as Vice President of Technology Innovation and Development at Arcturus Therapeutics. Previously, Priya held positions of increasing responsibility at Regulus Therapeutics and Nitto. Priya has a PhD in Chemistry from Indian Institute of Chemical Technology (India) where her doctoral research was focused on development of novel liposomal nucleic acid delivery systems for use in non-viral gene therapy and DNA vaccination. She pursued her postdoctoral research at Sanford-Burnham-Preby’s-Medical Discovery Institute, La Jolla, CA focusing on developing targeted nanoparticles for drug delivery. Priya is an author and co-inventor on over 70 publications and patents in the field.

Rabia Khan

Founder & CEO, Serna.bio
Rabia Khan

Rabia Khan, PhD (Immuno Genetics, MBA) is the founder and CEO of Serna Bio, a YC-backed biotech building the world's first map of the druggable transcriptome. She is a member of the Board of Trustees for the UK Dementia Research Institute, and an advisor to No Label Ventures, a fund investing in immigrant founders in the UK and EU.

Prior to founding Serna Bio, Rabia was Managing Director, Discovery Sciences at Sensyne Health plc (now - Arctoris Data) where she established the scientific strategy, built the data science and discovery teams by recruiting and leading a team of 50 machine learning and clinical researchers, delivering on a number of significant pharma partnerships including Bayer, BMS, Roche and Alexion.

She also held senior roles at BenevolentAI and Meta (acquired by Chan Zuckerberg BioHub). At Meta (previously Sciencescape), she was pivotal in the partnership with the Intelligence Advanced Research Projects Activity (IARPA) to acquire horizon-scanning technology that used NLP to research the biomedical corpus. At BenevolentAI, she helped shape the discovery strategy for a number of programs, led the Age-Related Macular Degeneration and Glioblastoma drug discovery programmes and served as the interface between the technical and biological teams under the mentorship of Prof. Jackie Hunter.

Born and raised in Pakistan, Rabia has a passion for supporting diversity in technology, and supporting initiatives focused on improving access to care for Schizophrenia and Dementia.
 

Samir Ounzain

Senior Chief Executive Officer and Scientific Co-Founder, HAYA Therapeutics
Samir Ounzain

Samir Ounzain is a molecular biologist with over 15 years of experience exploring the dark matter of the genome and its roles in development and disease.
Prior to founding HAYA Therapeutics, Samir was a Project Leader and Research Fellow at the Lausanne University Hospital (CHUV), where his research efforts directly led to the discovery of hundreds of novel heart-enriched lncRNAs, most notably the lncRNAs CARMEN, Meteor and Wisper.
 

Session Reserved for

, BioPhorum
Session Reserved for

Stefan Rathjen

Senior Scientist Drug Delivery, Silence Therapeutics GmbH
Stefan Rathjen

Stefan Rathjen studied biochemistry with majors in bioorganic chemistry and immunology at the university of Greifswald, Germany. Afterwards, Stefan did his PhD in molecular and cell biology at the Curie Institute and the “Université Paris-Saclay”, France, in which he studied endocytosis and intracellular processing of Shiga toxins. Since 2018, Stefan joined Silence Therapeutics in Berlin, Germany, where he works on improving Silence’s GOLD-platform, enabling extra-hepatic delivery of siRNA-conjugates, and the establishment of new experimental methodologies.

Thierry Dorval

Head of Data Sciences & Data Management, Servier Pharmaceuticals
Thierry Dorval

Thierry Dorval received a B.S. degree in theoretical physic and obtained a Ph.D. in image processing and artificial intelligence at Pierre & Marie Curie University, Paris, France. He then joined the Institut Pasteur Korea in 2005 first as researcher in biological image analysis then as a group leader specialized in High Content Screening applied to cellular differentiation as well as toxicity prediction. In 2012 he joined AstraZeneca, UK, where he was leading the Image and Data Analytics team. His activities were about developing and advising on quantitative image and data analysis solutions in support of high content phenotypic screens.
In 2015 he joined Servier, France, first as leader of the High Content Screening group within CentEX CPCB and then as Head of Data Science Lab, working on phenotypic approaches to improve drug discovery pipeline efficiency using high content and machine learning strategies.
 

Thomas Thum

Founder, Chief Scientific Officer and Chief Medical Officer, Cardior Pharmaceuticals GmbH
Thomas Thum

Prof. Dr. Dr. med. Thomas Thum, Founder, managing director and CSO/CMO of Cardior Pharmaceuticals GmbH, is a key opinion leader in cardiac diseases and the development of RNA-based therapeutics. He has received numerous awards for his work. Thomas is a member of the editorial boards of the world’s most prestigious journals for cardiovascular research and an executive member of national and international research committees in the cardiovascular field. Since 2009 Thomas is Director of the Institute of Molecular and Translational Therapeutic Strategies (IMTTS) at Hanover Medical School (MHH) and visiting professor at the National Heart and Lung Institute at Imperial College London since 2013. From 2021 to 2023 he was Director of the Fraunhofer Institute for Toxicology and Experimental Medicine (ITEM) in Hanover, Germany. Thomas Thum has authored over 500 scientific publications in renowned and field-leading journals and has filed over 50 patents, with many of them licensed and some already under clinical development.

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Conference agenda

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8:00

Registration & Coffee

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9:00

Chair's Opening Remarks

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9:10

Harnessing innate mechanisms of gene activation for RNAa therapeutics

Nagy Habib, Professor of Surgery, Imperial College London, Co-Founder, MiNA, Apterna and Dawn Therapeutics

  • An update on RNAa development programs for previously undruggable targets
  • Key finding and next steps from the latest RNA activation clinical results
  • Extrahepatic oligonucleotide delivery: how has technology and R&D progressed in recent years and what does this mean for the industry
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    9:50

    Manufacturing therapeutic oligonucleotides – the challenges of scale, cost and sustainability

    David Tew, Senior Scientific Director, Enzyme Engineering and Biocatalysts (E2B), GSK

  • Solid phase vs liquid phase methods
  • New chemistries for oligonucleotide synthesis
  • The use of enzymes in oligonucleotide synthesis
  • Thoughts on how these new methods will help to address cost and sustainability issues
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    10:30

    Morning Coffee

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    11:00

    Analytical strategies to ensure the quality of small synthetic antisense oligonucleotide and siRNA therapeutics in early development

    Joanna Hemming Taylor, Associate Principal Scientist, AstraZeneca

  • Overview of analytical strategies for synthetic oligonucleotides 
  • Highlighting the difference between strategies for single strand (ASOs) and double strand (siRNA) therapeutics
  • Challenges and opportunities for analysis of oligonucleotide related impurities
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    11:40

    The advantages and disadvantages of the various RNA delivery technologies

    Session Reserved for

    Session Reserved for, , BioPhorum

  • Current delivery systems can be unstable with sensitivity to shearing and temperature.
  • New and emerging RNA delivery systems could provide more beneficial delivery systems with more agile and efficient manufacturing methods
  • Technology could be adapted from different platforms to leverage new solutions. However, it is unclear what type of characterization should be performed, how risks are defined and what needs to be addressed for each batch.
  • Regulatory guidelines are still in development for RNA technologies and the chemistry, manufacturing and control perspective is unclear.
  • Understanding the comparative benefits and challenges across delivery systems can aid more informed decision making during delivery technology selection and development
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    12:20

    Networking Lunch

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    13:20

    Non-coding RNA-based therapeutics at the pulse of heart disease

    Thomas Thum, Founder, Chief Scientific Officer and Chief Medical Officer, Cardior Pharmaceuticals GmbH

  • Ongoing phase 2 trial in heart failure patients: How is the oligonucleotide R&D landscape accelerating forward for heart failure treatments?
  • Cardior Pharmaceuticals/pharma company interaction: What this means for strengthened RNA-based pipelines for chronic populations
  • Delving into further drug discovery pipelines in heart failure subgroups, where is research and funding still needed?
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    14:00

    Reviewing industry progress and R&D for advanced oligonucleotide modalities

  • Antisense oligonucleotides – the next chapter: How have we seen R&D evolve in recent years with more funding in genetic medicine and increased regulatory approvals
  • Technologies driving drug discovery: exploring how AI is being utilized to unlock drug discovery of oligonucleotide therapeutics
  • From proof of concept to oligonucleotide-based therapies: how are CMC practices developing to match the acceleration in R&D
  • Nagy Habib, Professor of Surgery, Imperial College London, Co-Founder, MiNA, Apterna and Dawn Therapeutics

    Peter Smith, Director, New Modalities Chemistry, AstraZeneca

    Thierry Dorval, Head of Data Sciences & Data Management, Servier Pharmaceuticals

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    14:40

    Afternoon Tea

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    15:10

    Mapping the Druggable Transcriptome – Exploring the Intersection of AI, synthetic Biology, and Drug Discovery

    Rabia Khan, Founder & CEO, Serna.bio

  • Why RNA? Limits of protein-focused drug discovery
  • The druggable transcriptome: mapping structure-functional relationships at scale
  • Leveraging the new RNA world for drug discovery – unlocking classically undruggable proteins and unexplored potential therapeutic targets
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    15:50

    Data Driven Optimization of Oligonucleotide Sequence Design

    Thierry Dorval, Head of Data Sciences & Data Management, Servier Pharmaceuticals

  • Expanding on the latest breakthroughs of Servier’s work in oligonucleotide sequence design:
  • Optimising drug discovery using computational and knowledge graph approaches
  • New case studies from Servier’s ASOS design approach
  • Ensuring off target effect identification using AI approaches
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    16:30

    In Vivo tLNP Platform – Transforming Cell Therapy

    Priya Karmali, Chief Technology Officer, Capstan Therapeutics

  • Using LNP for in vivo CAR-T and cell reprogramming
  • Payload considerations and platform approaches
  • The untapped potentials of LNP-CAR for curative immunotherapies including fibrosis, autoimmune etc
  • Scaling this up into a proven and viable platform
  • clock

    17:10

    Chair’s Closing Remarks and Close of Day One

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    8:30

    Registration & Coffee

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    9:00

    Chairman's Opening Remarks

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    9:10

    Novel Lipid Technology Platforms for RNA Therapeutics and Vaccines

    Heinrich Haas

    Heinrich Haas, Chief Technology Officer, NeoVac

  • To fully exploit the potential of nucleotide-based pharmaceuticals, tailored delivery systems are required. 
  • Key tasks comprise control of activity, targeting selectivity and immunogenicity.
  • Careful selection of lipids and lipid compositions allows to design nanoparticles for a given purpose
  • In-depth analytics and control strategies are of great importance for successful development 
  • Here we present examples for controlled assembly for improved mRNA nanoparticle formats
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    9:50

    Identification and validation of non-coding pharmacological targets

    Alessandro Bonetti, Principal Scientist, R&D Biopharmaceuticals, AstraZeneca

  • Overview of the non-coding field
  • Non-coding elements as novel pharmacological targets
  • Developing a technological pipeline for identificaiton and validation of non-coding pharmacological targets
  • RNA therapeuthics targeting non-coding elements
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    10:30

    Morning Coffee

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    11:40

    Improving target specificity and delivery technologies for oligonucleotide therapeutics

  • Updates in LNP technologies and viral vectors for targeted oligonucleotide delivery
  • Extrahepatic delivery: how far have we come and what is still needed to meet industry acceleration
  • Looking to the future: what lies ahead for technologies to improve target specificity of ASO delivery?
  • Heinrich Haas

    Heinrich Haas, Chief Technology Officer, NeoVac

    Priya Karmali, Chief Technology Officer, Capstan Therapeutics

    Alessandro Bonetti, Principal Scientist, R&D Biopharmaceuticals, AstraZeneca

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    12:20

    Networking Lunch

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    13:20

    RNAi Therapeutics to treat Cancer and other indications

    David Evans, CSO, Sirnaomics, Inc

  • A polypeptide nanoparticle (PNP) formulation comprised of two active siRNA (small interfering RNA) inhibitors targeting TGF-β1 and COX-2 for intravenous treatment of solid tumours and local administration for fat remodelling
  • Codelivery of TGFβ and Cox2 siRNA inhibits HCC by promoting T-cell penetration
  • Scientific rationale for further exploration using STP707 for the treatment of HCC and potentially other solid tumors
  • Additional model siRNAs will be discussed
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    14:00

    The dark genome as the next revolution in drug discovery

    Samir Ounzain, Senior Chief Executive Officer and Scientific Co-Founder, HAYA Therapeutics

  • What is the ‘dark genome’ actually about
  • Decoding ‘junk DNA’ and the potential toolbox of IncRNA-targeting therapeutics
  • The promise compared to traditional approaches
  • Drugging the dark genome for treating fibrotic diseases
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    14:40

    Afternoon Tea

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    15:10

    Recent Advances in the Development of Splice-Switching Oligonucleotides for CNS Diseases

    Daniel Elbaum, Chief Scientific Officer, QurAlis

  • PreRNA mis-splicing is a recurring theme in neurodegenerative diseases.
  • Splice switching ASOs provide a powerful modality to correct these errors. 
  • QurAlis’ FlexASO™ platform enables the construction of splice switching ASOs with enhanced potency, safety, and biodistribution properties.
  • This platform generally and in the context of our discovery programs will be discussed
  • clock

    15:50

    Chairman’s Closing Remarks and Close of Day Two


    Principal Scientist, R&D Biopharmaceuticals
    AstraZeneca
    Chief Scientific Officer
    QurAlis
    CSO
    Sirnaomics, Inc
    Senior Scientific Director, Enzyme Engineering and Biocatalysts (E2B)
    GSK
    Chief Technology Officer
    NeoVac
    Associate Principal Scientist
    AstraZeneca
    Professor of Surgery, Imperial College London, Co-Founder
    MiNA, Apterna and Dawn Therapeutics
    Director, New Modalities Chemistry
    AstraZeneca
    Chief Technology Officer
    Capstan Therapeutics
    Founder & CEO
    Serna.bio
    Senior Chief Executive Officer and Scientific Co-Founder
    HAYA Therapeutics
    BioPhorum
    Senior Scientist Drug Delivery
    Silence Therapeutics GmbH
    Head of Data Sciences & Data Management
    Servier Pharmaceuticals
    Founder, Chief Scientific Officer and Chief Medical Officer
    Cardior Pharmaceuticals GmbH

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    ChemGenes, an ISO 9001 certified company established in 1981, is the industry leader in manufacturing oligonucleotide synthesis reagents and has consistently provided the highest quality phosphoramidites and solid supports in the market. Our facility, just outside of Boston/Cambridge Massachusetts USA, is setup for bulk manufacturing of therapeutic grade phosphoramidite and solid support DNA/RNA synthesis products for GMP grade oligonucleotide manufacturing. ChemGenes carries the widest variety of modified phosphoramidites and supports currently used in oligonucleotide synthesis including Microarray Technology, Oligonucleotide Therapeutics, Oligonucleotide Based Probes and other areas of Nucleic Acid research. In addition, ChemGenes is expanding its custom oligonucleotide synthesis department specializing in the manufacture of ASOs, siRNA, gRNA, gapmers, aptamers, DNA based probes, and superior quality beads for Single Cell mRNA sequencing applications using Drop-Seq, Slide-seq, and Seq-Well methods. ChemGenes remains devoted to providing you with invaluable customer service and comprehensive technical support.


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    At the heart of the Holland Park district, our hotel is 10 minutes from Westfield London shopping center. We're blocks from Shepherd's Bush Underground station, linking to central London, and Kensington Palace and Gardens are two miles from us. Enjoy 24-hour access to our fitness center.

     
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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’

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    SAE Media Group , Ground Floor, India House, 45 Curlew Street, London, SE1 2ND, United Kingdom
    Tel: +44 (0) 20 7827 6000 Fax: +44 (0) 20 7827 6001
    Website: http://www.smgconferences.com Email: events@saemediagroup.com
    Registered in England - SMi Group Ltd trading as SAE Media Group




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