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SAE Media Group’s inaugural Conference
RNA Therapeutics USA
14-15 June, 2023 | San Diego, CA, USA
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SAE Media Group is proud to announce the inaugural RNA Therapeutics USA Conference taking place in San Diego on the 14 – 15th June 2023.

As part of SAE Media Group’s leading portfolio of Drug Discovery and the long-standing RNA Therapeutics Europe conferences, we are delighted to introduce RNA Therapeutics USA.

RNA therapies are leading the way in the field of genetic medicine, with the market expected to reach 1.81 billion USD by 2025, growing at a CAGR of 7.5%. This in large can be attributed to the topical advancements in mRNA vaccines, paving the way for global regulatory successes.

We currently see the RNA Therapeutic field focus on the challenges associated with delivery of RNA molecules extrahepatically, with a particular drive to overcome barriers of delivery to the Central Nervous System. In addition, RNA technologies continue to lead the way in revolutionising immunotherapies and the vaccine landscape, along with exceptional potential to treat a wide variety of chronic, previously undruggable diseases. The 2023 agenda will address these latest challenges along with recent advancements in RNAi and continue to drive innovation in the field of RNA Therapeutics.

Don't miss this opportunity to network with the pharma and biotech community of global experts in the UK and Europe; the two-day agenda offers you peer-to-peer networking with Directors of novel RNA therapies, CEOs, Senior and expert scientists, Chief Medical Officers, Senior Vice Presidents and many more.

 

 

 

  • Learn how RNA Therapies are revolutionising treatment for chronic diseases of the liver, heart, CNS and musculoskeletal system.
  • Investigate the latest trends in RNA immunotherapies as a tool for designing novel RNA based vaccines
  • Enhance your knowledge of the latest innovations in RNA application and RNA delivery techniques with case studies from leading industry experts
  • Engage in the growing advances and development of novel RNAi technologies in the treatment of chronic diseases
     

 

Key Job Titles include:

Research & Development Directors/Managers/Scientists
Heads of Clinical Development
Heads of Pre-Clinical Development
Head of RNA Biology
Head of RNA Formulation and Drug Delivery
Head of Pre-clinical R&D
Head of New Modalities
Head of Discovery
Senior Scientists
Chief Scientific Officers
Chief Medical Officers

Global Presence:

Attendees from all over the world attend our events, gain hands on insight from representatives from UK, Europe and USA.


 

 

Advanced Cell Diagnostics, A Bio-Techne Brand; Aldevron; Aldevron Llc; AMBR Consulting Ltd; AmpTec GmbH; Asahi Kasei Corporation; Astrazeneca; Bachem; BianoGMP; BioNTech RNA Pharmaceuticals; Bio-Techne Ltd; Chiesi Farmaceutici spa; eTheRNA; Ethris GmbH; F.Hoffmann-La Roche AG; Galapagos; GE Healthcare; Genevant Sciences; Genmab A/S; GSK; himed; Imperial College London; Inabata France S.A.S.; Independent; InteRNA Technologies BV; Intertek; Intertek Pharmaceutical Services Manchester; JSC R-PHARM; MHRA; Miescher Pharma GmbH; MiNA Therapeutics; MiNa Therapeutics Ltd; moderna Therapeutics; Nanogenics Ltd; New England Biolabs; NHS Blood and Transplant; NHS Blood and Transplant Authority; Nippon Fine Chemical; Nitto Avecia; Nitto Denko Avecia; Norinnova Technology Transfer AS; Pantherna Therapeutics GmbH; Polyplus-Transfection; Roche Diagnostics Deutschland; Roche Diagnostics Deutschland GmbH; Roche Innovation Center; Royal College of Surgeons in Ireland; Sanofi; Sense Biopharma AS; Symbiosis Pharmaceutical Services Limited; Thermo Fisher Scientific; TranSINE Therapeutics; Univeristy of Gent – Gene Therapy; University Gent; Vaccitech Ltd;

 

Conference programme

8:00 Registration & Coffee

9:00 Chairman's Opening Remarks

Michelle Lynn-Hall

Michelle Lynn-Hall, Associate Vice President, Genetic Nanomedicine, Eli Lilly
View Bio

9:10 Genetic (Nano)Medicine: State of the Art, Frontiers and Current Research

Michelle Lynn-Hall

Michelle Lynn-Hall, Associate Vice President, Genetic Nanomedicine, Eli Lilly
View Bio

  • Key challenges in genetic medicine
  • Case studies in nucleic acid API innovation
  • Case studies in delivery innovation
  • Future outlook
  • 9:50 An antisense oligonucleotide-based approach for the treatment of autosomal dominant optic atrophy

    Aditya Venkatesh

    Aditya Venkatesh, Senior Principal Scientist, Stoke Therapeutics Inc
    View Bio

  • Introduction to Stoke’s proprietary research platform TANGO
  • Application of TANGO for the treatment of autosomal dominant optic atrophy (ADOA)
  • Pre-clinical pharmacology data for STK-002, Stoke’s clinical candidate for ADOA
     
  • 10:30 Morning Coffee

    11:00 AI/ML developed RNA therapeutics for neurodegenerative diseases

    Martin Akerman

    Martin Akerman, CTO & Co-Founder, Envisagenics
    View Bio

  • Overcoming in-silico modeling challenges for neurodegenerative diseases
  • How Envisagenics’ SpliceCore platform can be used in designing antisense oligonucleotides
  • Translational R&D from in-silico to in-vitro for potential ASO candidates in neurodegenerative disease
  • 11:40 A new era of programmable therapeutics to upregulate genes

    Alfica Sehgal

    Alfica Sehgal, SVP Head of Discovery & Translation, CAMP4 Therapeutics
    View Bio

  • Targeting regRNA to increase gene expression and the use of RNA actuators to increase regRNA activity and restore levels of mRNA  expression
  • Powerful tool to upregulate endogenous gene expression to treat diseases
  • Understanding the mechanism of action for regRNA mediated gene regulation
  • Current pipeline programs in CNS eg Dravet syndrome and liver metabolic diseases, eg urea cycle disorders
  • 12:20 Networking Lunch

    13:20 Targeted RNA therapeutics approaches for developing transformational medicines for patients

    Tamar Grossman

    Tamar Grossman, Vice President, Global Head of RNA and Targeted Therapeutics, Janssen Pharmaceutical Companies of Johnson & Johnson
    View Bio

  •  RNA modalities for therapeutics and vaccine development
  • Novel carriers for improved extra-hepatic targeting
  • CNS delivery of siRNA for neurological disease therapies
  • 14:00 The FORCETM platform overcomes barriers of oligonucleotide delivery to muscle and corrects myotonic dystrophy featuresin preclinical models

    Stefano Zanotti

    Stefano Zanotti, Senior Director, Head of Neuromuscular Research, Dyne Therapeutics
    View Bio

  • Introduction to the FORCE platform
  • DYNE-101: application of the FORCE platform for the treatment of myotonic dystrophy type I
  • Insight into DYNE-101 delivery in NHP
  • 14:40 Afternoon Tea

    15:10 Advancing RNA therapeutics through the development of long non-coding RNA therapies

    Samir Ounzain

    Samir Ounzain, CEO, HAYA Therapeutics
    View Bio

  •  Exploring new directions in the identifi cation of novel lncRNA targets
  • Reviewing antisense oligonucleotides in the targeting of disease generating lncRNAs
  • Case study: targeting the lncRNA Wisp
  • 15:50 Antibody Oligonucleotide Conjugates for the Treatment of Muscle Disorders

    Ramana Doppalapudi

    Ramana Doppalapudi, Vice President Chemistry, Avidity Biosciences Inc.
    View Bio

  • Challenges with oligonucleotide delivery
  • Development of antibody oligonucleotide conjugates (AOC) for the delivery of oligonucleotides
  • Application of AOC technology for the treatment of rare muscle genetic disorders
     
  • 16:30 Chairman’s Closing Remarks and Close of Day One

    8:30 Registration & Coffee

    9:00 Chairman's Opening Remarks

    Michelle Lynn-Hall

    Michelle Lynn-Hall, Associate Vice President, Genetic Nanomedicine, Eli Lilly
    View Bio

    9:10 Emulsion adjuvants for pandemic preparedness, what can we do better?

    Rushit N Lodaya

    Rushit N Lodaya, Expert Scientist, GSK

  • Why  emulsion adjuvants are a tool of choice during Pandemic
  • Advances made in understanding the mechanism of action of these adjuvants
  • Learnings from the Covid-19 Pandemic
  • Perspective for future adjuvant development
  • 9:50 Utilising self-amplifying RNA technology

    Steven Reed

    Steven Reed, CEO, HDT Bio
    View Bio

  • Delving into model informed vaccine development for novel vaccines including self-amplifying RNA technology
  • Insight into HDT’s AMPLIFY vaccine platform which includes self-amplifying RNA
  • Enabling simplifi ed manufacturing processes, enhanced temperature stability and improved safety
  • Next steps: assessing the current fi eld and future work required or approval of novel vaccines
  • 10:30 Morning Coffee

    11:00 Accelerating the Development of Emerging Infectious Diseases: COVID Case study

    Chuong Huynh

    Chuong Huynh, Biologist Officially / Vaccine Product Coordination Team Lead Unofficially, Biomedical Advanced Research & Development Authority (BARDA)

  • Introduction to the BARDA’s vaccine strategy and approaches towards novel vaccines
  • Insight into BARDA’s COVID-19 Medical Countermeasure Portfolio
  • Strategies for developing successful COVID vaccine development contracts
  • 11:40 Panel Discussion: Reviewing RNA therapeutics as the next generation of genetic medicines

    Archit Rastogi

    Archit Rastogi, Assistant Director, Preclinical Development , Ionis Pharmaceuticals Inc.

  • Discussing current drug like properties of RNA modalities
  • Advancing RNA medicines beyond current vaccine products
  • Dissecting the challenges associated with targeted delivery of RNA based therapies
  • Understanding the future direction of RNA based drugs, with a focus on emerging, novel RNA modalities
  • 12:20 Networking Lunch

    13:20 Tailoring delivery systems for next generation mRNA therapeutics

    Heinrich Haas

    Heinrich Haas, Former Vice President Formulation & Drug Delivery, BioNTech
    View Bio

  •  Nanoparticle technologies for nucleotide and drug delivery systems
  • Structural and functional coherencies within selected delivery  systems 
  • Novel approaches and tailored solutions for future mRNA  therapeutics
  • 14:00 Developing siRNAs as the next generation of RNA therapies in Ocular Diseases

    Ying Yu

    Ying Yu, Associate Director, AbbVie
    View Bio

  • Establish siRNA-based target validation and therapeutic platform for ocular disease
  • Developing in vitro and in vivo assays that can effectively screen siRNAs lacking immunostimulatory properties.
  • Developing analytical approaches to study the pharmacokinetics of siRNA in the eye
  • A Case study: investigate the correlation between the pharmacokinetics and effi cacy of siRNA in rat retina
  • 14:40 Afternoon Tea

    15:10 GalAhead™: a novel therapeutic GalNAc-RNAi platform to downregulate single and multiple genes

    Jimmy Weterings

    Jimmy Weterings, Senior Director, Technology Innovation, Sirnaomics, Inc
    View Bio

  • Introduction to GalAhead, Sirnaomics’ GalNAc-RNAi therapeutic platform
  • Validation of technology in vivo and in vitro
  • Progress report on GalAhead-based programs
  • 15:50 The Utility of RNA Cocktail Approaches and New Solutions for Combinational Therapy Distribution

    Jay Sarkar

    Jay Sarkar, Researcher and Entrepreneur, Stanford University
    View Bio

  • Combinational Logic In Gene Networks
  • Exploitation of these Networks using Exogenous RNA
  • Demonstrated Examples and Domain of Use Cases
  • Complexities of Cocktail Delivery and New Solutions to Accommodate
  • 16:30 Chairman’s Closing Remarks and Close of Day Two

    +

    FEATURED SPEAKERS

    Aditya Venkatesh

    Aditya Venkatesh

    Senior Principal Scientist, Stoke Therapeutics Inc
    Alfica Sehgal

    Alfica Sehgal

    SVP Head of Discovery & Translation, CAMP4 Therapeutics
    Archit Rastogi

    Archit Rastogi

    Assistant Director, Preclinical Development , Ionis Pharmaceuticals Inc.
    Chuong Huynh

    Chuong Huynh

    Biologist Officially / Vaccine Product Coordination Team Lead Unofficially, Biomedical Advanced Research & Development Authority (BARDA)
    Heinrich Haas

    Heinrich Haas

    Former Vice President Formulation & Drug Delivery, BioNTech
    Jay Sarkar

    Jay Sarkar

    Researcher and Entrepreneur, Stanford University
    Jimmy Weterings

    Jimmy Weterings

    Senior Director, Technology Innovation, Sirnaomics, Inc
    Martin Akerman

    Martin Akerman

    CTO & Co-Founder, Envisagenics
    Michelle Lynn-Hall

    Michelle Lynn-Hall

    Associate Vice President, Genetic Nanomedicine, Eli Lilly
    Ramana Doppalapudi

    Ramana Doppalapudi

    Vice President Chemistry, Avidity Biosciences Inc.
    Rushit N Lodaya

    Rushit N Lodaya

    Expert Scientist, GSK
    Stefano Zanotti

    Stefano Zanotti

    Senior Director, Head of Neuromuscular Research, Dyne Therapeutics
    Steven Reed

    Steven Reed

    CEO, HDT Bio
    Tamar Grossman

    Tamar Grossman

    Vice President, Global Head of RNA and Targeted Therapeutics, Janssen Pharmaceutical Companies of Johnson & Johnson
    Ying Yu

    Ying Yu

    Associate Director, AbbVie

    Aditya Venkatesh

    Senior Principal Scientist, Stoke Therapeutics Inc
    Aditya Venkatesh

    Aditya Venkatesh combines over six years of experience in oligonucleotide therapeutics research with a strong background in retinal biology and ocular diseases. He is currently an Senior Principal Scientist at Stoke Therapeutics, where he has been involved in developing an antisense oligonucleotide-based approach to treat autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. Prior to Stoke, he worked at Idera Pharmaceuticals validating an antisense oligonucleotide platform for rare disease targets. He received his Ph.D. in Biomedical Sciences from the laboratory of Claudio Punzo at UMass Chan Medical School. His doctoral work elucidated the role of the mTOR pathway in promoting cone photoreceptor cell survival and visual function in Retinitis Pigmentosa, a genetic disease that leads to blindness.

    Alfica Sehgal

    SVP Head of Discovery & Translation, CAMP4 Therapeutics
    Alfica Sehgal

    Alfica Sehgal
    Alfica is VP at CAMP4, heading the discovery, preclinical and translational research. Before CAMP4, she was at Alnylam Pharmaceuticals, where she held multiple roles. Alfica was the program leader for Alpha-1 antitrypsin deficiency related liver disease, driving the program from bench to Phase 1, as well as the program research lead for the fitusiran program for the treatment of haemophilia and rare bleeding disorders. She was also a member of the platform team and next wave team responsible for generating and triaging new pipeline programs.
    Alfica received her post-doctoral training at Johns Hopkins University and Yale University, studying lipid uptake, cholesterol and oxygen homeostasis in parasites, mammalian cells and mice. She received her doctoral degree from the Tata Institute of Fundamental Research (India) in naturally acquired immunity against malarial parasites. She has published more than 30 articles and book chapters in peer-reviewed journals and has authored 17 patents and PCT applications.
     

    Archit Rastogi

    Assistant Director, Preclinical Development , Ionis Pharmaceuticals Inc.
    Archit Rastogi

    Chuong Huynh

    Biologist Officially / Vaccine Product Coordination Team Lead Unofficially, Biomedical Advanced Research & Development Authority (BARDA)
    Chuong Huynh

    Heinrich Haas

    Former Vice President Formulation & Drug Delivery, BioNTech
    Heinrich Haas

    Heinrich has his professional focus on development of pharmaceutical products in the field of nanotechnology and drug & RNA delivery. In his Ph.D. in the group of Prof. Dr. Helmuth Möhwald at Johannes-Gutenberg Universität Mainz and academic career in Italy and Brazil he researched lipid membranes and organized bio-molecular systems. Having moved to pharmaceutical industry he developed nanoparticle products with application in cancer, inflammatory diseases autoimmune diseases and other indications. At BioNTech, he is the scientific lead of the formulation and analytics department, which develops RNA therapeutics based on a broad scope of delivery technologies..
     

    Jay Sarkar

    Researcher and Entrepreneur, Stanford University
    Jay Sarkar

    Dr. Sarkar is a researcher and entrepreneur with a background in applied physics and electrical engineering, which he utilizes to develop systems biology solutions for medicine. He built his first company off his foundational PhD work at Stanford University, where he pioneered the use the mRNA modality for transiently reprogramming cell epigenetics to specifically reset cellular age. The approach has grown as one of the hottest in the longevity community, with some of the largest startup investments in biotech history. He has now progressed to studying the broader problem of new modality integration and distribution in a variety of tissues.

    Jimmy Weterings

    Senior Director, Technology Innovation, Sirnaomics, Inc
    Jimmy Weterings

    Jim has had a tight bond with the development of Oligonucleotide Therapeutics since 2002, performing research on Peptide Nucleic Acid (PNA) in the van Boom group at Leiden University and targeted SSO conjugates at Prosensa. During his Ph.D at Leiden University, the Netherlands he generated and studied CpG Oligonucleotide and other Toll-like receptor targeting ligand containing conjugates. At Cenix BioScience, he developed siRNA conjugates for targeted CNS delivery. And at Cristal Therapeutics he led various projects on the nanomedicine delivery of oligonucleotide in oncology. In 2019, Jim joined AstraZeneca, Discovery Sciences in Sweden, to further advance the development of (targeted) oligonucleotide therapeutics. Recently Jim joined Sirnaomics, USA as Senior Director, Head of Technology Innovation with focus on again targeted delivery of oligonucleotides. Jimmy (co-)authored 13 articles and holds several patents for drug delivery enabling technologies, including the most recent one on TMTHSI, a superior conjugation compound to generate oligonucleotide and antibody conjugates. TMTHSI has recently been marketed as CliCr® with Iris Biotech and is licensed to Synaffix as well as part of a collaboration with Lonza.

    Martin Akerman

    CTO & Co-Founder, Envisagenics
    Martin Akerman

    Dr. Martin Akerman is the inventor of SpliceCore®, Envisagenics’ flagship platform born of his vision of applying machine learning to RNA information and discovering new drug targets in areas of unmet need. Martin trained as a postdoctoral fellow with Dr. Adrian Krainer at Cold Spring Harbor Laboratory, where he helped in the development of Spinraza®, the first FDA-approved RNA therapeutic for treating Spinal Muscular Atrophy. Dr. Akerman received his PhD in Bioinformatics from Technion, Israel Institute of Technology, where he studied how RNA splicing can boost functionality of the human genome and trigger diseases.

    Michelle Lynn-Hall

    Associate Vice President, Genetic Nanomedicine, Eli Lilly
    Michelle Lynn-Hall

    Michelle Lynn Hall is currently the Senior Director of New Therapeutic Modalities at Eli Lilly, where she leads a team of engineers and scientists in the discovery of nucleic-acid-based therapies. Immediately prior to joining Lilly, Michelle worked at Moderna, where she led a team of modelers studying mRNA therapeutics design and lipid nanoparticle delivery. She received her Ph.D. from Columbia University where she developed a novel method to predict chemical reactivity and applied these to skills to a diverse set of problems ranging from materials science to biocatalysis. She then completed a joint postdoc at Novartis Institutes for Biomedical Research and Yale University, where she studied computer-aided drug design. She subsequently worked at Schrödinger where she focused on molecular simulations, machine learning, and chemical biology. She is passionate about equity, diversity, inclusion, and awareness in science, technology, engineering and math (STEM) and has served as a mentor, champion and advocate for women and minorities throughout her career. As a native Texan, she has intolerably strong opinions about BBQ.

    Ramana Doppalapudi

    Vice President Chemistry, Avidity Biosciences Inc.
    Ramana Doppalapudi

    Dr. Ramana Doppalapudi is a scientist with experience in the discovery and development of a variety of drugs including peptides, antibodies, oligonucleotides, and antibody drug conjugates. Over the last 23 years in the pharma and biotech industry, Dr. Doppalapudi developed multiple clinical candidates for the treatment of cancer, muscular diseases, and wet AMD. Dr. Doppalapudi is currently serving as Vice President, Chemistry at Avidity Biosciences in La Jolla, CA, USA where he is responsible for discovery chemistry and CMC.

    Rushit N Lodaya

    Expert Scientist, GSK
    Rushit N Lodaya

    Samir Ounzain

    CEO, 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.

    Stefano Zanotti

    Senior Director, Head of Neuromuscular Research, Dyne Therapeutics
    Stefano Zanotti

    Stefano Zanotti is Head of Neuromuscular Research at Dyne Therapeutics, where he oversees preclinical development of treatments for genetically-driven muscular diseases. Stefano dedicated his career to rare genetic disorders. Prior to joining Dyne, Stefano led pre-clinical research programs in multiple rare diseases as Director of Rare Renal and Musculoskeletal Diseases at Sanofi. Previously, Stefano was an Assistant Professor at the UCONN School of Medicine, where he conducted NIH-funded research on genetic disorders of the skeletal system.
    Stefano has authored more than 50 research articles and reviews in major scientific journals and received multiple awards for his contributions to skeletal biology.

    Steven Reed

    CEO, HDT Bio
    Steven Reed

    Steve Reed is the Founder, President, and CEO of the Infectious Disease Research Institute (IDRI), based in Seattle, USA. His academic appointments include Professor of Medicine at Cornell University Medical College in New York and Research Professor of Pathobiology at the University of Washington.

    Dr. Reed’s dedication to global heath began during his undergraduate studies, and were the focus of this graduate studies at the University of Montana where he began work on adjuvants with Dr. Edgar Ribi. Dr. Reed received a PhD in Microbiology and Immunology from the University of Montana in 1979. That year he was appointed as Scientist of the National Institute of Amazon Research in Manaus, Brazil, where he directed research on tropical diseases.

    Dr. Reed joined Cornell University Medical College in 1980 as Assistant Professor of Medicine, continuing to work in Brazil as manager of the Cornell-Bahia program in International Medicine. He joined the Seattle Biomedical Research Institute in 1984 where he worked until founding IDRI in 1993. He has received over $100 million in grants from the US NIH, BARDA, DARPA, and the Gates Foundation. In 1994 he co-founded Corixa Corporation (which was later sold to GlaxoSmithKline, GSK) where he served as Chief Scientific Officer until leaving in 2004. He also founded Dharma Therapeutics, a transdermal patch company, where he served as President from 2005-2008. In 2008 Dr. Reed, together with Rick Klausner, David Baltimore, and Ralph Steinman founded Immune Design Corp. (IMDZ, NASDAQ), a cancer therapeutics company, where he served as CEO until 2011.

    Dr. Reed’s research interests have focused on the immunology of intracellular infections, and on the development of vaccines and diagnostics for both cancer and infectious diseases. In partnership with GSK, he led the team that developed the first defined tuberculosis vaccine, now in advanced clinical development. He also developed the first defined vaccines for leishmaniasis, as well as the K39-based diagnostic tests currently licensed for leishmaniasis. He has more than 350 original publications, 35 book chapters and reviews, and 105 issued patents on diagnostics, vaccines, and therapeutics of adjuvants, cancer, and infectious diseases.
     

    Tamar Grossman

    Vice President, Global Head of RNA and Targeted Therapeutics, Janssen Pharmaceutical Companies of Johnson & Johnson
    Tamar Grossman

    Dr. Tamar Grossman is a VP and Global Head of RNA and Targeted Therapeutics at Johnson and Johnson. Tamar is responsible for the delivery of RNA based development candidates and ADCs across all therapeutic areas. Previously, she was a Senior Director and Head of Rare Diseases at Arcturus Therapeutics where she developed RNA therapeutics across different RNA modalities. Prior to Arcturus, Tamar was the Director of Translational Medicine at Ionis Pharmaceuticals where she led various antisense oligonucleotide research programs to clinical development. She received her PhD in Biochemistry from Tel-Aviv University and complete postdoctoral research training at the UCSD.

    Ying Yu

    Associate Director, AbbVie
    Ying Yu


    Over 25 years of research experiences in pharmaceutical companies and academic institutions with focus on RNA therapeutics, ophthalmic drug discovery and vascular physiology.

    Expertise in small interference RNA (siRNA)-based drug target validation and RNA therapeutics. including 1) Selection of siRNAs lacking immunostimulatory properties; 2) in vivo siRNA-mediated cleavage assay development; 3) in vivo siRNA efficacy evaluation and 4) in vivo siRNA PK/PD assay development.
    Two patents on novel gene discovery and siRNA delivery, awarded 4-year research grant from American heart association, two book chapters, several national awards, and over 30 papers in peer-reviewed journals including Nature, PNAS, Circulation and J Immunology.
     

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    San Diego, USA

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