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Development of RNA therapeutics & vaccines

Wednesday 16th March 2022
(All times in CET)


The bright future of RNA


08:30 Opening remarks


08:40 The technology that saved the world: how a once-dismissed idea led the fight against COVID-19

Prior to 2020 a vaccine had never been developed in less than four years. This session explores the remarkable story behind the development of mRNA vaccines for COVID-19, and what their success means for the future of RNA medicines.

  • How to take an unrealised idea and apply it to a new disease area
  • Lessons learned from the rapid development of an experimental drug
  • What does the success of mRNA vaccines mean for the future of RNA medicines?

Melissa Moore, CSO, Moderna

Melissa Moore, CSO, Moderna


09:10 Keynote panel – What will the next five years of RNA medicines look like?

After a decade of promise, RNA medicines are coming of age. This session presents the perspectives of RNA thought-leaders on the bright future of RNA, including where they see the most exciting growth and what continues to keep them awake at night.

  • What lessons have been learned from clinical progress to date?
  • Which new technologies offer exciting contributions to the field, and where are the major technology gaps still to be filled?
  • What are the major challenges that the field still needs to solve to truly realise the potential of RNA medicines?

Melissa Moore, CSO, Moderna
Douglas Fambrough, CEO, Dicerna Pharmaceuticals
Brett Monia, CEO, Ionis Pharmaceuticals
Bill Haney, CEO & Chairman, Skyhawk Therapeutics
Alexandra Bause, Co-Founder, Investment Director & Head of VentureLabs, Apollo Health Ventures

Melissa Moore, CSO, Moderna   Douglas Fambrough, CEO, Dicerna Pharmaceuticals    Brett Monia, CEO, Ionis Pharmaceuticals    Bill Haney, CEO & Chairman, Skyhawk Therapeutics    Alexandra Bause, Co-Founder, Investment Director & Head of VentureLabs, Apollo Health Ventures


09:50 Keynote panel – Where does RNA sit for Big Pharma?

Big Pharma’s relationship with RNA has been fractious, but a handful of thought-leaders are driving internal and external programs to build RNA capabilities. This session explores the Big Pharma commitment to RNA medicines including partnership opportunities and research priorities for the next five years.

  • Which RNA technologies are of the most interest and why?
  • Assorted drug development strategies including internal R&D, partnership programs and external innovation
  • What is the vision for this field in five to ten years?

Tim Luker, VP, Search & Evaluation – Emerging Technology & Innovation, Corporate Business Development,Eli Lilly
Shalini Andersson, Chief Scientist, New Therapeutic Modalities and Head of Oligonucleotide Discovery, AstraZeneca
Sylke Poehling, Senior Vice President and Global Head, Therapeutic Modalities, Roche
Sudha Chivukula, Head of RNA Technology, Research & External Innovation, Sanofi Pasteur

Tim Luker, VP Venture Science, Corporate BD, Eli Lilly    Shalini Andersson, Chief Scientist, New Therapeutic Modalities and Head of Oligonucleotide Discovery, AstraZeneca    Sylke Poehling, Senior Vice President and Global Head, Therapeutic Modalities, Roche    Sudha Chivukula, Head of RNA Technology, Research & External Innovation, Sanofi Pasteur


10:30 Morning Break and Oligonetworking Roundtables

Are you new to the field? Or would you like to meet some new players?

Join the Oligonetworking Roundtables to meet with other RNA leaders over a coffee.

A relaxed structured networking session for those who want to build new business relationships in this growing field.


Innovation Rapid Fire Session

The field of RNA is ripe for tackling new targets, modalities, technologies and delivery methods. This session presents a snapshot of some of the most exciting new work in early development from the next generation of RNA Leaders.


11:30 A new and druggable means to reverse cell senescence through modulation of RNA splicing

Levels of splicing factors change during ageing, compromising our ability to carry out “fine tuning” of gene expression. Senisca is using oligonucleotide-based therapeutic approach to reset splicing factor levels and reverse senescence.

Lorna Harries, Co-Founder and CSO, Senisca

Lorna Harries, Co-Founder and CSO, Senisca


11:40 Enabling mRNA therapies for pulmonary disease indications

mRNA therapies can help to replace or augment missing or non-functional proteins or to introduce new proteins to modulate the course of a disease. Ethris are developing a pipeline of immune-modulation and mRNA-based protein replacement therapies.

Carsten Rudolph, Co-Founder and CEO, Ethris

Carsten Rudolph, Co-Founder and CEO, Ethris


11:50 Targeting long non-coding RNAs for tissue-specific treatment of fibrotic diseases

Specific long non-coding RNAs (lncRNAs) are important regulators of the resting fibroblast to myofibroblast conversion. HAYA is utilising modified ASOs to target and inhibit proprietary lncRNAs to prevent and reverse fibrosis.

Samir Ounzain, CEO and Scientific Co-Founder, HAYA Therapeutics

Samir Ounzain, CEO and Scientific Co-Founder, HAYA Therapeutics


12:00 Regeneration of urethral muscle in Stress Urinary Incontinence using engineered mRNA

Engineered IGF-I-encoding mRNA following injection in the urinary sphincter resulted in secretion of therapeutically relevant protein levels which activated muscle regeneration. Treatment of stress urinary incontinence in an animal model resulted in functional and morphological regeneration, supporting its therapeutic potential.

Klaas Zuideveld, CEO, Versameb

Klaas Zuideveld CEO, Versameb


12:10 Prophylaxis 2.0 via RNAi

The first generation of SARS-CoV-2 prophylaxis, namely mRNA-based vaccines, has been a remarkable scientific triumph. Yet, respiratory viruses continue to pose formidable challenges to all vaccine modalities. Eleven Therapeutics is developing Prophylaxis 2.0 via intranasal administration of RNAi therapeutics. It circumvents viral escape mutants, uniquely protects from inter-individual transmission, and shields at-risk populations such as immunocompromised conditions.

Speaker to be advised, Eleven Therapeutics

Shadow 300x


12:20 Talk topic to be advised

Speaker to be advised, Nanovation Therapeutics

Shadow 300x


12:30 Lunch


Delivery and distribution


13:50 New advances in RNA-targeted therapeutics

A leader in antisense technology, Ionis have experience in delivering therapies by nearly all routes of tissue-specific administration. This session covers learnings and new advances in RNA-targeted therapeutics.

  • Advances in drug delivery methods
  • Addressing intra-cellular trafficking
  • What is next? Exploring new chemistries and technology advancements

Brett Monia, CEO, Ionis Pharmaceuticals

Brett Monia, CEO, Ionis Pharmaceuticals


14:10 Advancing extra-hepatic delivery of RNAi

With an advanced pipeline of RNAi therapies, Dicerna are making strides in the delivery of tissues beyond the liver. This session covers new leanings and gains in this field.

  • Update on clinical pipeline including IND process
  • Advances in hepatic and extra-hepatic delivery methods
  • Presentation of extra-hepatic delivery data

Bob Brown, CSO, Dicerna Pharmaceuticals

Bob Brown, CSO, Dicerna Pharmaceuticals


14:30 RNA therapeutics enable expansion of disease target space

RNA therapeutics are a rapidly expanding class of drugs that enable new disease targets and a new class of personalised medicines. This session looks at advances in targeted delivery and other work driving the field forward.

  • Advances in targeted delivery of oligonucleotides beyond the liver
  • Novel endosomal escape enhancers to improve oligonucleotide efficacy

Shalini Andersson, Chief Scientist, New Therapeutic Modalities and Head of Oligonucleotide Discovery, AstraZeneca

Shalini Andersson, Chief Scientist, New Therapeutic Modalities and Head of Oligonucleotide Discovery, AstraZeneca


14:50 Harnessing tumor-targeting iRGD peptides to effectively target and deliver RNA-based therapeutics into solid tumors

While RNA-based drugs have reached market to help patients with a range of diseases, progress with this class of medicines for treatment of solid tumor cancers has been disappointing. The tumor stroma serves as a primary impediment to effective solid tumor delivery for many classes of drugs and particularly for RNA-based medicines. Cend Therapeutics is advancing a unique approach to convert the stroma from an impediment to a conduit for effective drug delivery.

  • Validation of lead investigational drug, CEND-1, in clinical trials for the treatment of pancreatic cancer – a tumor type particularly noted for its desmoplastic stroma
  • CEND-1 is an internalizing RGD (iRGD) dual-acting cyclic peptide that enhances receptor-mediated endocytosis and transcytosis to penetrate all layers of the tumor
  • Application of the same tumor-targeted tissue penetrating technology to enable effective delivery of RNA-based drugs within tumor-penetrating nanocomplexes (TPNs)
  • Preliminary results for delivery on the enhanced iRGD-guided TPN delivery of RNA-based therapies.

David Slack, CEO, Cend Therapeutics

David Slack CEO, Cend Therapeutics


15:10 Overcoming the Blood–Brain Barrier for RNA therapeutics

Biorchestra is developing disease-modifying therapies for neurodegenerative diseases using RNA therapeutics and a proprietary polyion-complex-micelle-based delivery system, BDDS(TM).

  • How to deliver RNA therapeutics to the brain and brain cells 
  • Tackling issues of deep brain delivery via intravenous injection
  • Introduction of our lead program adopting BDDS(TM)

Branden Ryu, CEO, Biorchestra

Branden Ryu,CEO,Biorchestra


15:30 Afternoon Break and Services Clinics

Do you need specialised delivery technology? Or to build relationships with GMP providers? Or to get some IP advice?

In the Services Clinics participants are offered the chance to pre-book at 10-minute meeting with exhibitors and build business relationships with some of the leading service providers in the field.


Delivery and durability


16:00 Putting patients at the heart of an saRNA drug delivery strategy

RNA delivery, gene up-regulation with acceptable clinical outcome are not easy to achieve. This session takes on drug delivery and durability from a “patient-first” perspective, through the lens of RNA activation.

  • Using RNA activation to restore normal function to cells
  • Increasing duration of the drug, and why it matters
  • RNA gene activation in the brain
  • How to predict clinical response with small activation RNAs

Nagy Habib, Founder and Head of R&D, MiNA Therapeutics

Nagy Habib, Founder and Head of R&D, MiNA Therapeutics


16:20 CARTs - a new vehicle for nucleic acid delivery with a unique controllable release mechanism

CARTs are biodegradable, amphiphilic oligomers suitable for the formulation and delivery of nucleic acids. The session introduces this novel platform and gives an overview about the underlying concepts and application data.

  • CARTs are based on a polycarbonate-b-polyalphaaminoester backbone and self-assemble with nucleic acids into nanoparticles
  • CARTs lead to a complementary biodistribution of RNA expression when compared to LNPs
  • CARTs exhibit high transfection efficiency in various cell lines and are also active in hard to transfect cells such as primary T cells

Philipp Heller, Projektmanager Innovation, Evonik

Projektmanager Innovation, Evonik


16:40 Talk topic to be advised

Angele Maki, SVP & Head of Business Development, ReCode Therapeutics

Angele Maki, SVP, Business Development, ReCode Therapeutics


17:00 Talk topic to be advised

Suzanne Saffie-Siebert, Chairwoman and Chief Executive Officer, sisaf

Suzanne Saffie-Siebert CEO at SiSaf


17:20 Advances in RNA-targeted delivery approaches to access previously undruggable targets

With the advancement of RNA-targeted therapeutics tries are being made in assorted delivery approaches. This session explores the development and application of several innovative approaches.

  • Addressing limitations of current delivery platforms
  • A look at innovative new approaches including antibody-olio conjugates, peptide-mediated delivery and exome-based nanotechnology
  • Update on recent research and applications

Matthew Wood, Professor of Neuroscience and Deputy Head, Medical Sciences Division, University of Oxford

Matthew Wood, Professor of Neuroscience and Deputy Head, Medical Sciences Division, University of Oxford


17:40 Improving responses of mRNA using self-replicating RNAs

Self-replicating RNAs have shown enhanced protein expression at lower doses than conventional mRNA vaccines, suggesting potential importance drug candidates. This session explores advancements in and applications of the technology.

  • New developments in self-replicating RNA technology
  • Applications for vaccines and mRNA therapies
  • Role of self-replication in improving delivery and durability

Nathaniel Wang, CEO, Replicate Bioscience

Nathaniel Wang, CEO, Replicate Bioscience


18:00 Famous Pairs Party

RNA Leaders is all about partnerships! Celebrate famous partnerships throughout history at the Famous Pairs Party and enjoy a drink with prospective business partners at the same time. Most importantly, can you find your own famous counterpart at the party?

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Thursday 17th March 2022
(All times in CET)


RNAi advancements


 

09:00 Lessons learned from preclinical and clinical development of microRNA therapies

Dysregulated microRNA expression is a key factor in many complex multi-factorial diseases. This session explores Regulus’s current pipeline of therapies in development including lessons during clinical progression.

  • Update on preclinical and clinical pipeline
  • Investigating new disease areas including delivery methods
  • Regulatory challenges and key lessons for development of RNA therapies

Denis Drygin, CSO, Regulus Therapeutics

Denis Drygin, CSO, Regulus Therapeutics


09:20 A new modality to improve distribution, durability and potency of RNAi

The need to treat neurodegenerative diseases is urgent and currently unsolved. This session explores a breakthrough therapeutic approach using RNAi to halt neurodegenerative conditions at their source.

  • Understanding the modality including impact on distribution, durability and potency
  • Update on pre-clinical development data
  • Tackling delivery and distribution to the deep brain structure

Aimee Jackson, CSO, Atalanta Therapeutics

Aimee Jackson, CSO, Atalanta Therapeutics
 


09:40 Harnessing new chemistries and molecular design to silence disease-associated genes

By harnessing the body’s natural RNA interference mechanism, it is possible to create precision medicines that silence disease-associated genes. This session looks at new chemistries and molecular design to tackle a wide range of diseases.

  • Putting patients at the heart of new discoveries
  • Update on R&D pipeline and presentation of data

Marie Wikstrom Lindholm, SVP and Head of Molecular Design, Silence Therapeutics

Marie Wikstrom Lindholm, SVP and Head of Molecular Design, Silence Therapeutics


10:00 Discovering RNA therapeutics with Abzu’s explainable AI

Two of the most promising advances in healthcare technology – RNA therapeutics and explainable AI – are fundamentally transforming what’s possible in the field of therapeutic development. Abzu’s pioneering bioinformatics platform effortlessly integrates heterogeneous data, such as sequences and multi-omics data, and reveals intricate nonlinear relationships – even on wide datasets.

The core technology, the QLattice, is the engine that generates a veritably infinite number of mathematical models to explain fundamental interactions – and yet identifying and understanding interactions is easy with straightforward and simple models. By using the QLattice, scientists gain a deeper understanding of biological and chemical mechanisms to create new drugs and modalities and increase their success rate, reducing the time required for drug discovery.

This presentation will show how Abzu’s pioneering bioinformatics platform is being used to predict active siRNAs and how the explainable models uncover the underlying mechanisms in siRNA.

Lykke Pedersen, Senior Bioinformatician, Abzu

Lykke Pedersen, Senior Bioinformatician, Abzu. png


10:20 Morning Break and Oligonetworking Roundtables

Are you new to the field? Or would you like to meet some new players?

Join the Oligonetworking Roundtables to meet with other RNA leaders over a coffee.

A relaxed structured networking session for those who want to build new business relationships in this growing field.


mRNA therapeutics

 


11:00 Talk topic to be advised

Speaker to be advised, Providence Therapeutics


11:20 Talk topic to be advised

Speaker to be advised, Cytiva


Antisense approaches

 


11:40 Talk topic to be advised

Speaker to be advised, Axolabs

Axolabs


12:00 Upregulation of protein expression via ASO therapy

Preclinical studies have shown that TANGO ASOs can be used to reduce the synthesis of non-productive mRNA and increase the synthesis of productive mRNA. This session explores the utility of the approach in developing mutation-independent therapies for autosomal dominant haploinsufficiency diseases.

  • Understanding the technology
  • Presentation of pre-clinical and clinical data
  • Future activities and challenges

Gene Liau, CSO, Stoke Therapeutics

Gene Liau, CSO, Stoke Therapeutics
 


12:10 Drugging the genome to address base causality with a novel synthetic genetic medicine

The use of a synthetic ASO platform offers the opportunity to practice genetic medicine at scale. The session explores how to combine the specificity of nucleotide engagement with the intracellular penetration and broad organ distribution capabilities of small molecules to target the genome or transcriptome.

  • Understanding the technology including presentation of early data
  • Addressing unique changes to the scaffold, targeting and self-assembly
  • Applications and next steps

Dietrich Stephan, Founder and CEO, Neubase Therapeutics

Dietrich Stephan, Founder and CEO, Neubase Therapeutics


12:40 Lunch
 


Gene therapy and editing approaches


14:00 Delivering on the promise of genetic medicine by co-opting the body’s natural RNA editing system

KorroBio aims to address rare genetic diseases as well as common illnesses by effecting precise, disease-modifying RNA edits with a simple oligonucleotide drug product. This session explores how oligonucleotides can be designed to re-direct the enzyme ADAR to make corrective or disease-modulating A to G edits using selected targets in the liver and central nervous system as examples.

  • Understand the technology that enables precise RNA editing
  • Understand how RNA editing differs from other genetic medicine approaches
  • Illustrate the utility of the technology via presentation of pre-clinical data on targets in the liver and central nervous system

Howard Stern, CSO, Korro Bio

Howard Stern, CSO, Korro Bio


14:25 CRISPR as therapy: a non-viral approach to gene editing

Using an LNP-based delivery system it is possible to selectively knock out disease-causing genes, introduce targeted insertion of a functional gene, or both. This session explores the potential of the system to treat genetic disease.

  • Understanding the technology and its application
  • Major challenges to be addressed
  • Early clinical update and data presentation

Laura Sepp-Lorenzino, CSO, Intellia Therapeutics

Laura Sepp-Lorenzino, CSO, Intellia Therapeutics


14:50 Harnessing ADAR-mediated RNA base editing for therapy

Besides the genome, genetic information can be manipulated at the transcript level, including an approach called site-directed RNA editing. Particularly attractive for clinical translation is the harnessing of endogenous ADARs for adenosine-to-inosine RNA base editing. As inosine is biochemically interpreted as guanosine, genetic information is recoded.

  • Strategies that harness the ubiquitously expressed, endogenous ADARs either with chemically modified ASOs or genetically encoded guideRNAs
  • Compared to approaches with engineered ADARs, the latter gains advantage from providing only a guide RNA component and from very low off-target editing
  • Overall, RNA base editing represents a promising opportunity for drug discovery.

Thorsten Stafforst, Interfaculty Institute of Biochemistry, Eberhard Karls Universität Tübingen

Thorsten Stafforst, Interfaculty Institute of Biochemistry, Eberhard Karls Universität Tübingen


Targeting RNA with small molecules


15:15 Talk topic to be advised

Peter Smith, Co-Founder, President and CEO, Remix Therapeutics

Peter Smith, Co-Founder, President and CEO, Remix Therapeutics


15:40 Afternoon Break and Services Clinics

Do you need specialised delivery technology? Or to build relationships with GMP providers? Or to get some IP advice?

In the Services Clinics participants are offered the chance to pre-book at 10-minute meeting with exhibitors and build business relationships with some of the leading service providers in the field.


16:10 Drugging the undruggable: correcting the underlying genetics of disease at the RNA level

RNA mis-expression has been found to cause a growing list of diseases, from orphan and neurological conditions to major cancers. This session explores an approach to modify expression of target genes previously considered undruggable.

  • A look at small molecule therapeutics for alternative modulation of RNA
  • Update on Skyhawk’s preclinical pipeline
  • Major challenges still to be addressed

Kathleen McCarthy, Co-Founder & Chief Scientific Officer, Skyhawk Therapeutics

Kathleen McCarthy, Co-Founder & Chief Scientific Officer, Skyhawk Therapeutics


16:35 Rational approach to designing selective therapeutics targeting RNA

Talk abstract to be advised

Matthew Disney, Professor, Department of Chemistry, Scripps Research

Matthew Disney, Professor, Department of Chemistry, Scripps Research


17:00 Using small molecules for modulation of miRs relevant to cardiovascular, renal and metabolism diseases

Drugging RNA using small molecules opens for new treatment avenues for patients. This session presents examples of using small molecules for modulation of miRs relevant to cardiovascular, renal and metabolism diseases.

  • Synergies and complementarity to the rapidly evolving RNA therapeutic approaches
  • Assays and screening assays
  • A look at recent and upcoming work including collaboration between Big Pharma and academia

Malin Lemurell, Executive Director and Head of Medicinal Chemistry Early CVRM, Biopharmaceuticals R&D, Astra Zeneca

Malin Lemurell


17:25 Early drug discovery of novel RNA drug targets

By applying machine learning in combination with fragment-based screening using NMR, it is possible to revolutionize the lead-finding process. It enables the rapid validation of new RNA targets for the presence of valid binding sites for small molecules. Furthermore, starting points for chemical optimization can be identified within a very short time. This session examines how to apply AI in combination with experimental screening to identify novel RNA drug targets and inhibitors of these targets.

  • Addressing the complexities of dynamic RNA molecules when identifying novel targets
  • Application of RNA target selection and hit-to-lead generation in the SARS CoV-2 genome
  • Application of hit-to-lead optimization in the bacterial ribosome

Marcel Blommers, CSO, Saverna Therapeutics

Marcel Blommers, CSO, Saverna Therapeutics


17:50 Close of congress

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