SAE Media Group’s inaugural Conference
RNA Therapeutics USA
14-15 June, 2023 | San Diego, CA, USA
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
  • 14:40 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
    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 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
  • 17:10 Chairman’s Closing Remarks and Close of Day One

    Michelle Lynn-Hall

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

    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

  • 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
  • Tae-Won Kim

    Tae-Won Kim, Vice President, Preclinical Development, Ionis Pharmaceuticals

    Christine Esau

    Christine Esau, Vice President, Arrowhead Pharmaceuticals

    Archit Rastogi

    Archit Rastogi, Assistant Director, Ionis Pharmaceuticals

    12:20 Networking Lunch

    13:20 Harnessing human genetics to target metabolic diseases with RNAi Therapeutics:

    Ho-Chou Tu

    Ho-Chou Tu, Associate Director, Alnylam Pharmaceuticals
    View Bio

  • RNAi therapeutics has demonstrated success in the clinic both in the rare and prevalent disease space
  • Human genetics is a powerful way to identify relevant drug targets with higher likelihood of success
  • Introducing early-stage GalNAc-siRNA programs targeting prevalent metabolic diseases (eg. T2D and NASH) based on human
  • 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

    Michelle Lynn-Hall

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


    Senior Principal Scientist
    Stoke Therapeutics Inc
    SVP Head of Discovery & Translation
    CAMP4 Therapeutics
    Assistant Director
    Ionis Pharmaceuticals
    Vice President
    Arrowhead Pharmaceuticals
    Biologist Officially / Vaccine Product Coordination Team Lead Unofficially
    Biomedical Advanced Research & Development Authority (BARDA)
    Former Vice President Formulation & Drug Delivery
    Associate Director
    Alnylam Pharmaceuticals
    Researcher and Entrepreneur
    Stanford University
    Senior Director, Technology Innovation
    Sirnaomics, Inc
    CTO & Co-Founder
    Associate Vice President, Genetic Nanomedicine
    Eli Lilly
    Vice President Chemistry
    Avidity Biosciences Inc.
    Vice President, Chemistry
    Avidity Biosciences
    Expert Scientist
    HAYA Therapeutics
    Senior Director, Head of Neuromuscular Research
    Dyne Therapeutics
    HDT Bio
    Vice President, Preclinical Development
    Ionis Pharmaceuticals
    Vice President, Global Head of RNA and Targeted Therapeutics
    Janssen Pharmaceutical Companies of Johnson & Johnson
    Associate Director





    Horton Grand Hotel

    San Diego, USA

    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 SMi Group. SMi 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.

    Preliminary Attendee List





    Eclipsebio has developed multiple world-class RNA genomics products used by biopharma and academic customers around the world to accelerate the development of tomorrow’s key RNA genomics discoveries and medicines.

    Our innovative standard and customizable solutions bring new and deeper insights to the RNA interactome by providing more accurate, reproducible and useable data for our customers.

    Our products:

    - Identify new RNA regulatory and epigenetic mechanisms

    - Spur exciting new insights into mRNA

    - RNA binding protein and miRNA biology

    - Map new RNA drug targets to accelerate the development of safe and effective RNA medicines




    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. Additionally, 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. ChemGenes remains devoted to providing you with invaluable customer service and comprehensive technical support.



    Established in 2007, Lexogen is a global leading company in transcriptomics, next-generation sequencing, RNA analysis and bioinformatics. Our mission is to empower our customers with innovative top quality RNA analysis solutions & support, in order to improve health and well-being for everyone and our planet. Lexogen, an ISO 9001 certified company, is the leader in 3’ mRNA sequencing, a technology proven for its efficiency, its robustness, and its sensitivity.

    Lexogen’s portfolio includes innovative kits developed and produced in Vienna, Austria for:

    • true single-cell as well as bulk RNA sequencing,

    • RNA extraction,

    • RNA extraction,

    • spike-in RNA variant controls, and

    • nascent RNA labeling for transcriptome-wide analysis of RNA kinetics.

    Lexogen also provides first-class, fully integrated, customizable RNA sequencing services, from experimental design consulting to sample processing and tailored bioinformatics analytical reporting.

    Lexogen is a privately held company headquartered in Vienna, Austria, with a subsidiary in New Hampshire, USA.

    Media Partners



    Founded in 2020, 1STRAND sets to showcase San Diego County as the premier location for RNA therapeutics R&D, provide a forum to highlight the positive impact of RNA medicines on public health, and to connect professionals in the space and grow the local talent pool of qualified individuals.


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    Horton Grand Hotel


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    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.


    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.


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

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    ‘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.


    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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