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Congress agenda 



Day 1 - Tuesday 18th October

Jade Osei-Tutu, Senior Conference Director, LSX 

Jade Osei-Tutu

 

Due to the pandemic RNA approaches have seen an increase in investment and resources in recent years, spanning multiple applications including personalized medicine, drug targets, biomarkers, and vaccines, and making it a top three area for investment within genetic medicine. This session will give a quick overview of how RNA technologies have developed in 2022.

Malgorzata Gonciarz, AVP Genetic Medicine, Eli Lilly 

Malgorzata Gonciarz, Associate Vice President, Genetic Medicine, Eli Lilly

Supported by:

Catalent logo

The mRNA vaccine for COVID-19 reinvigorated the RNA field in the last few years but there is still a way to go. This discussion presents thought-leaders from across different RNA approaches on what the next generation of RNA medicines could look like and the challenges around delivery and stability the industry are working on to continue the growth with RNA.

  • Which indications beyond COVID-19 are on the horizon RNA modalities?
  • Which new technologies offer exciting contributions to the field, and where are the major technology gaps still to be filled?
  • What are key hurdles the industry still needs to address to fully to unlock the full potential of RNA therapeutics?
  • Is industry working together to review success and unsuccessful clinical progress to build effect next generation products?

Moderator:

Rowan Walrath, life sciences reporter, Boston Business Journal

Rowan Walrath, Life sciences reporter, Boston Business Journal

Speakers:
Brett Monia
, CEO, Ionis Pharmaceuticals 
Frank de Rosa, CTO & Global Head of Research, mRNA Center of Excellence, Sanofi 

Brett Monia, CEO, Ionis PharmaceuticalsFrank de Rosa, Chief Technology Officer & Global Head of Research, mRNA Center of Excellence, Sanofi

Cell and Gene Therapy investment takes up a large share of biotech investment and with spotlight RNA this only increases the opportunity in the genetic medicine space. This session presents leading Life Science investors who have their foot in the game for different RNA therapeutics and Vaccine applications and keen to back the emerging field further.

  • Which technologies areas within RNA are most exciting and disruptive right now and
  • Which RNA approaches have the easiest route to market in the next 5 years and so ROI?

Moderator:
Angelica Peebles,
Health Reporter, Bloomberg News

Angelica Peebles, Health Reporter, Bloomberg News

Speakers:
Lucio Iannone
, VP of Venture Investments Health, Leaps at Bayer 
Nina Kjellson, General Partner, Canaan 
Nathaniel Wang, CEO, Replicate Bioscience
Geoffrey von Maltzahn, General Partner, Flagship Pioneering

Lucio Iannone, VP of Venture Investments Health, Leaps at BayerNina Kjellson, General Partner, CanaanNathaniel Wang, CEO, Replicate BioscienceGeoffrey von Maltzahn, General Partner, Flagship Pioneering

 

  • Overview of key challenges in genetic medicine

  • Nucleic acid innovation case study: Ribonucleic acid (RNA) editing via upregulation of endogenous Adenosine Deaminase RNA Specific (ADAR)

  • Delivery innovation case study: Nose to brain delivery

Michelle Hall, AVP, Genetic Medicine, Eli Lilly 

Michelle Hall, Eli lilly

 

  •  Alltrna is pioneering tRNA biology to control the translational process of proteins. 
  • Theirplatform enables the design of engineered tRNAs with therapeutic properties
  • Theirmedicines have the potential to treat thousands of diseases that share the same genetic alteration
Lovisa Afzelius, Founding CEO and Director, Alltrna

Lovisa Afzelius, CEO, Alltrna, & Origination Partner, Flagship Pioneering


11:25am The Therapeutic Potential of oRNA

Robert Mabry, CSO, Orna Therapeutics 

Robert Mabry, CSO, Orna Therapeutics

11:35am A Paradigm Shift – Therapeutics in Cardiovascular Diseases Based on Non-coding RNAs

Non-coding RNAs (ncRNAs) are critical for the regulation of important cellular processes and their dysregulation is a hallmark of many diseases, Cardior has developed a therapeutic approach to restore normal levels and functions of these players in the pathological processes of cardiac diseases.

  • Regulatory non-coding RNAs as new emerging targets
  • Targets are complete pathways, not only single mRNAs
  • Translation of this approach into clinical reality
  • Currently huge European phase 2 study ongoing: HF-REVER

Thomas Thum, CSO & Founder, Cardior Pharmaceuticals

Thomas Thum CSO & Founder, Cardior Pharma

 

11:45am Bringing the AI revolution to RNA therapeutics 

Eleven is in the process of building the first-of-its-kind AI system to elucidate the structure-activity relationship (SAR) of RNA molecules designed to identify the best functional molecules. To train our AI system, we developed the TERÅ platform, which employs a massively parallel DNA-encoded library approach to test combinatorically modified RNA molecules and obtain a functional readout from living cells.

Yaniv Erlich, CEO, Eleven Therapeutics 

Yaniv Erlich, CEO, Eleven Therapeutics

11:55am Next-generation lipid nanoparticle technologies to enable nucleic acid therapeutics

NanoVation Therapeutics offers an unencumbered LNP toolbox to enable safe and efficient delivery of nucleic acids to a range of tissues. During this presentation, our CSO, Dr. Jayesh Kulkarni, will discuss the range of technologies within NanoVation's IP portfolio and provide examples of potential applications.

Jayesh Kulkarni, CSO & Co-founder, NanoVation Therapeutics 

Jayesh Kulkarni, Chief Scientific Officer & Founder, NanoVation Therapeutics

12:05pm Chair Questions
Malgorzata Gonciarz
, AVP Genetic Medicine, Eli Lilly

Malgorzata Gonciarz, Associate Vice President, Genetic Medicine, Eli Lilly-1

Afternoon Chair:
David Oxley, President, Exopharm

David Oxley, President, Exopharm

mRNA technology has changed the way therapies are developed. The overall potential of mRNA is clear, with mRNA therapeutics being developed in many research areas. The speed and potential cost savings of mRNA technology make it an interesting technology for personalized medicine. However, much of the current equipment used in manufacturing is repurposed from the biotech industry and is designed for much larger scales than needed for mRNA. What are the challenges associated with smaller scales?

Katarina Stenklo, Enterprise Solutions Commercial Activation Leader, Cytiva

Katarina Stenklo, Enterprise Solutions Commercial Activation Leader, Cytiva

Moderna’s product pipeline reflects the progress of clinical programs currently in development to create mRNA vaccines for a wide range of infectious diseases.

This session will provide an overview of vaccine candidates in clinical development and an update on Moderna’s cytomegalovirus (CMV) vaccine development program.

Lori Panther, VP, Clinical Development, Infectious Disease, Moderna Therapeutics

Lori Panther, VP, Clinical Development, Infectious Disease, Moderna Therapeutics

mRNA production is limited by availability of high quality, GMP grade DNA. Synthetic DNA produced enzymatically can address the draw backs associated with plasmid derived DNA templates. opDNA is a synthetic DNA template with a 3’ open end, which can feed directly into IVT processes without the need for linearisation. opDNA is devoid of a bacterial backbone and can be designed with long continuous poly-(A) tails encoded within the sequence. opDNA achieves significantly higher mRNA yields, comparable proinflammatory cytokine/chemokine responses, and equivalent gene expression as compared to plasmid derived templates. Furthermore, opDNA templates can be generated in significantly shorter time frames independent of sequence complexity or length, accelerating the therapeutic development of gene therapy and vaccine programmes.

Heikki Lanckriet, CEO & CSO, 4basebio 

Heikki Lanckriet, CEOCSO, 4basebio

  • mRNA delivery to the diseased liver
  • New concept of Transcription factor based mRNA Therapy
  • Efficacy of an mRNA therapy in different animal models for liver fibrosis
  • Overview of CureVac’s pipeline of novel RNA applications for infectious diseases, oncology and rare diseases

Patrick Baumhof, SVP of Technology, CureVac 

Patrick baumhof

  • Engineering successful scaleup (not scale out)
  • Strategies to address variability   
  • Rapid formulation development 

Camden Cutright, Business Development Director, Micropore Technologies
Camden Cutright, Business Development Director, Micropore Technologies

Small activating RNAs (saRNA) are double stranded 21 nucleotide RNA that either target promoters or enhance genes leading to mRNA upregulation. saRNAs can be delivered with liposomes into the systemic circulation or subcutaneously by conjugation with aptamers or GalNAC. MTL-CEBPA is an investigative drug that resulted from the conjugation of saRNA CEBPA with NOV 340 lipsomes that targets tumour associated macrophages in order to alter favourably the tumour microenvironment. The relevance of the technology is that it can deliver transcription factors to the bone marrow CD34+ cells and that the effect lasts at least 6 weeks in vivo. MTL-CEBPA has been administered safely in over 130 patients with advanced cancer and improved clinical outcome in a sub-set of patients when co-administered with TKI or checkpoint inhibitor. Clinical outcomes will also be presented. 

  • Development of RNA activation to target tumour associated macrophages
  • Upregulation of transcription factors genes in the bone marrow
  • Administration of RNA activation drug in over 130 patients with advanced cancer
  • Safety profile and clinical outcomes

Konstantina Skourti Stathaki, Principal Scientist, MiNA Therapeutics

Konstantina Skourti Stathaki, Principal Scientist, MiNA Therapeutics

 

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

  • 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 BDDSTM.

Branden Ryu, CEO, Biorchestra

Branden Ryu,CEO,Biorchestra

While existing delivery technologies allow for effective systemic RNA delivery to hepatic tissues, extrahepatic targeting and endosomal release have remained challenging for all RNA species.

We have designed a unique peptide-based non-lipidic delivery platform that rapidly self-assembles siRNA and mRNA into a stable ~60 nm polyplex. The 21 a.a. cationic amphipathic peptide (OligoPhoreTM/SemaPhoreTM) is programmed with all the functionality to enable transfection of myriad cell types and pH-responsive intra-endosomal disassembly, which is followed by endosomal permeabilization and full escape of RNA.

We have demonstrated safe systemic delivery and efficacious knockdown with siRNA, or translation/expression with mRNA, in >20 publications using ~15 animal models for cancer, atherosclerosis, metabolic syndrome, arthritis, and aortic aneurysm, among others.

Our lead candidates for initial clinical applications in pancreatic/colorectal cancer and arthritis comprise peptide-RNA polyplexes of siRNA’s against KRAS and NF-kB, respectively.

Samuel Wickline, CSO, Altamira Therapeutics

Samuel Wickline, Chief Scientific Officer, Altamira Therapeutics, Inc.

  • What are the true bottlenecks in targeted delivery of RNA treatments?
  • A lot of work has been done to improve RNA product safety and efficacy but what is hindering scalability and large licensing deals of non-vaccine approaches?
  • What delivery tools are the industry keen to advance to realize seamless cell and tissue delivery, tolerability, and expression control of RNA, and why?

Moderator:
Suzanne Saffie-Siebert, CEO, SiSaf 

Suzanne Saffie-Siebert CEO at SiSaf

Speakers:
Patrick Baumhof, SVP of Technology, CureVac 
David Slack, President and CBO, Lisata Therapeutics
Dietrich Stephan, Chairman & CEO, NeuBase Therapeutics
Matthew Hawryluk, EVP, Chief Business Officer, Gritstone Bio

Patrick baumhofDavid Slack CEO, Cend TherapeuticsDietrich Stephan Matthew Hawryluk, EVP, Chief Business Officer, Gritstone

 

Day 2 - Wednesday 19th October

Jade Osei-Tutu, Senior Conference Director, LSX

Jade Osei-Tutu

Morning chair:

David Sokolowski, Global Workflow Manager, Nucleic Acid Therapeutics, Cytiva

David Sokolowski, Global Workflow Manager, Nucleic Acid Therapeutics, Cytiva

 

Before the COVID-19 pandemic Big Pharma’s relationship with RNA has been fractious, but with RNA based modalities dominating next generation genetic medicines, a handful of thought-leaders are now driving internal and external programs to build RNA capabilities. This session explores  Big Pharma's 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?

Moderator:

Angelica Peebles, Health Reporter, Bloomberg News

Angelica Peebles, Health Reporter, Bloomberg News 

Speakers:
John Androsavich
, Global Head, RNA Medicine Lead - Emerging Science & Innovation, Pfizer 
Renee Williams, AVP of External Strategy and Genetic Medicine, Eli Lilly 
Colin Moran, Global Early Innovation Partnering Lead, Discovery Sciences, Johnson & Johnson
Robert Goodnow, Head Drug Discovery Sciences, Takeda  

John Androsavich, Global Head, RNA Medicine Lead - Emerging Science & Innovation, PfizerRenee Williams Eli Lilly Colin Moran J&JRobert Goodnow

The Australian and Victorian Governments have deemed mRNA & RNA as a strategic investment priority. They’ve committed to local researchers receiving the support and industry collaboration opportunities needed to develop new vaccines and therapeutics.

 The panel will discuss opportunities within the Australian sector, including:

  • Research funding and collaboration with Victoria’s 22 world-leading medical research institutions 
  • Clinical trial capability for RNA therapeutics and vaccines
  • Emerging supply chain development
  • How government support for commercialization of research has resulted in global RNA industry leaders choosing Victoria as their base for APAC regional operations

Moderator: John Carroll, Director of the Monash Biomedicine Discovery Institute (BDI) & Dean of Biomedical Sciences, Monash University
Samantha O'Connor
, VP, Head, Business & Operations Emerging Sciences & Innovation, Pfizer
Kate Jeffrey, Executive Director, Moderna
David Hoey, CEO, Vaxxas

John Carroll, Director of the Monash Biomedicine Discovery Institute (BDI) & Dean of Biomedical Sciences, Monash UniversitySamantha OConnor, Head, Business and Operations, Emerging Sciences & Innovation (ES&I), Pfizer-1Kate Jeffrey, Executive Director, ModernaDavid Hoey, CEO, Vaxxas

RNA viruses are diverse and abundant pathogens responsible for numerous human ailments, from common colds to AIDS, SARS, Ebola, and other dangerous diseases.

RNA viruses possess relatively compact genomes and have therefore evolved multiple mechanisms to maximize their coding capacities, often using overlapping reading frames. In this way, one RNA sequence can encode multiple proteins via mechanisms including alternative splicing and ribosomal frameshifting.

Many such processes in gene expression involve the RNA folding into three-dimensional structures that can recruit ribosomes without initiation factors, hijack host proteins, cause ribosomes to frameshift, and expose or occlude regulatory protein binding motifs to ultimately control each key process in the viral life cycle.

This session will discuss the structure of HIV-1 and SARS-CoV-2 RNA in cells and the importance of alternative conformations assumed by the same RNA sequence in controlling viral gene expression.

Silvi Rouskin, Professor, Harvard University 

Silvi Rouskin, Professor, Harvard University

mRNA processing is a highly regulated mechanism that controls gene expression and is known to be dysregulated in a wide variety of disease states. This session explores mechanisms of mRNA processing regulation and advances in the drug discovery toolkit that are leading to novel insights for small molecule discovery. 

  • Understanding the complexity of mRNA processing mechanisms 
  • Developments in screening methodologies 
  • Small molecule control of transcription factor expression in vitro and in vivo 

Zaven Kaprielian, CSO, Remix Therapeutics

Zaven Kaprielian, CSO Remix Therapeutics

RNA structures play key roles in disease biology.  This session will present rational methods to identify and optimize small molecules targeting RNA structure.  This includes approaches to eliminate RNAs by targeted degradation.
  • Using genetics and informatics to define RNA structures that cause disease
  • Using Informatics and other rational approaches to design compounds targeting these RNAs
  • Developing molecules to target RNA for elimination from cells to mouse models of important diseases

Matt Disney, Professor, Department of Chemistry, Scripps Research

Matthew Disney, Chair, Scripps Research

The success of mRNA-based COVID-19 vaccines has resulted in increased opportunity for advancing mRNA as a platform for vaccine and therapeutic discovery.

This momentum brings to the forefront a need for optimizing mRNA-based discovery and development workflows. A key bottleneck in discovery workflows is acquiring synthetic mRNA for screening, validation and optimization of candidates.

Relying on current commercial mRNA solutions can mean long lead times of weeks to months, lack of control over the workflow or sometimes unexpected results after weeks of waiting to test identified mRNA candidates. Recent automated solutions for synthesis of mRNA from a digital sequence can overcome this bottleneck in mRNA synthesis.

Find out how a push-button automation platform can build hundreds of mRNA candidates in a week to enable researchers to build, screen and optimize candidate sequences quickly reducing workflow time to a few days and not months.

 

Krishna Kannan, Senior Director, Product Development, CODEX DNA 

Krishna Kannan, Senior Director, Product Development, CODEX DNA

 

  • Brief review of platform innovations for delivery to the liver
    • Overview of select products
  • A look at the pipeline: the next wave of RNAi therapeutics
  • Expansion into extrahepatic applications

Kirk Brown, CNS Program Lead, Alnylam Pharmaceuticals

Kirk Brown, CNS Program Lead, Alnylam Pharmaceuticals

As we drive towards mining increasingly diverse target classes and biological processes the range of synthetic and recombinant modalities we employ has expanded significantly. One area of recent success has been the use of interference RNA modalities such as antisense oligonucleotides and siRNA. The tools needed to design, make and test these therapeutics differs substantially from those needed for traditional small molecules, requiring the assembly of new platforms and the building of new skill sets. This presentation will discuss recent steps to establish and validate a comprehensive toolbox enabling our clients to quickly and economically access capabilities in this exciting modality area.

Dave Madge, VP, WuXi AppTec

Dave Madge, Vice President, WuXi AppTec

Afternoon Chair:

Dave Madge, Vice President, WuXi AppTecDave Madge, Vice President, WuXi AppTec

 

Sirnaomics has developed proprietary GalNAc-RNAi therapeutic platform, GalAhead™, comprising two key technological components – mxRNA™ (miniaturized RNAi triggers) and muRNA™ (multi-unit RNAi triggers).

mxRNAs are composed of single ~30 nt long oligonucleotides to downregulate individual genes, while muRNA molecules are comprised of multiple oligonucleotides (2 or more) to simultaneously silence two or more targets.

Dmitry will present data validating these two technologies both in vivo and in vitro, as well as progress report on quickly expending GalAhead therapeutic pipeline.

Dmitry Samarsky, CTO, Sirnaomics

Dmitry Samarsky, Chief Technology Officer, Sirnaomics

Nucleic acid-based therapies offer the promise of specific knockdown of individual transcripts to modify disease, and approximately twenty therapies including siRNA modalities have been approved for use in humans, however for CNS diseases, remains to be developed fully.  

This talk will discuss work from a foundational publication (Alterman et al., 2019) showed that branched divalent siRNA with two individual siRNA oligonucleotides joined by a covalent linker achieved knockdown of HTT transcript and protein widely in brain following ICV administration. Exploring the combined results indicate that the combination of branched siRNA with our proprietary formulation presents the tolerability, brain distribution, and durability necessary to tackle diverse neurological diseases.

  • Presenting modifications to the original chemical motif that gave improvements in potency and in acute tolerability upon ICV dosing as measured by increased knockdown of the target gene and by reduction in behavioral effects including seizure.
  • Demonstrating a well-tolerated dose of a lead compound >50% knockdown of target protein in multiple regions of mouse brain, including hippocampus, motor cortex, and striatum, that lasted at least six months. Furthermore, we demonstrate that the di-siRNA platform provides phenomenal potency and durability of silencing for multiple different targets.
  • Transcriptomic measurements in multiple brain areas showed no consistent changes in transcripts other than the target transcript in response to di-siRNA demonstrating a high degree of specificity for the di-siRNA platform.
  • The formulation has enabled approximately ten-fold higher tolerability of compound dosed ICV in mice and rats. 

Aimee Jackson, CSO, Atalanta Therapeutics

Aimee Jackson, CSO, Atalanta Therapeutics

  • Update on R&D and clinical pipeline 
  • Investigating new disease areas including delivery methods
  • Regulatory challenges and key lessons for clinical development of oligonucleotides therapies 

Brett Monia, CEO, Ionis Pharmaceuticals

Brett Monia, CEO, Ionis Pharmaceuticals

Over the last decade, we have seen a breathtaking number of novel therapeutic modalities and strategies in the area of oligonucleotide/nucleic acid/RNA-based therapeutics. This includes new modalities (e.g. single guide RNAs), new versions of “old” platforms (e.g. siRNA- and ASO-conjugates) and “light speed” maturation of new technologies (e.g. mRNA-vaccines). These developments resulted in numerous approved therapeutics and a growing number of compounds in preclinical and clinical development. At the same time, new players with no “institutional” experience in the nucleic acid area entered the therapeutic area.

This development raises significant challenges for custom-research and custom-manufacturing organizations. Novel approaches to identify lead candidates, to manufacture the compounds at different scales and to analyze/bio-analyze those new modalities must be in place in time.

Through case studies, we will present how Axolabs has been addressing those challenges and providing solutions to support the development programs of our clients.

Martin Kögler, Scientist, Chemical Research & Manufacturing Sciences, Axolabs GmbH

Martin Koegler, Scientist, Chemical Research and Manufacturing Sciences, Axolabs GmbHJPG

 

ASOs delivered intrathecally have now been developed successfully for genetic forms of rare neurological disorders. The delivery route has been successful in both children and adults. The next step is the development of ASOs targeting non genetic forms of neurodegenerative diseases through the restoration of normal protein levels known to be depleted as a result of the underlying pathophysiology of the disease. This talk will cover one such target and the development of relevant ASO’s

  • Current clinical status of ASO intrathecal delivery
  • Known ASO specific AE/side effects
  • ASO targeting sporadic forms of ALS

Angela Genge, CMO, QurAlis 

angela Genge, Quarlis

RNA genomics technologies go beyond traditional RNA-Seq and illuminate structural and regulatory features of RNA as well as the RNA interactome. This presentation will highlight novel, accessible technologies encompassing:  

  • Simplified assays for transcriptome-wide ribosomal profiling
  • RNA structure probing of in vitro transcribed mRNA
  • SHAPE databases profiling RNA structure in human cell lines and normal tissues
  • Scalable, multiplexible technology for mapping RNA binding protein binding sites
  • Direct, quantitative profiling of miRNA targets

Karen Chapman, CSO, Eclipse Bio

Karen Chapman, CSO, Eclipsebio

NeuBase is accelerating the genetic revolution by developing a new class of precision genetic medicines that Drug the Genome™. The Company’s therapies are built on a proprietary platform called PATrOL™ that encompasses a novel peptide-nucleic acid antisense oligonucleotide technology combined with novel delivery shuttles that overcome many of the hurdles to selective mutation engagement, repeat dosing, and systemic delivery of genetic medicines.

With an initial focus on silencing disease-causing mutations in debilitating neurological, neuromuscular, and oncologic disorders, NeuBase is now focused on in vivo gene editing applications. NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions, who currently have limited to no treatment options.

  • A description of how the NeuBase's editing technology functions
  • Potential benefits of a non CRISPR/Cas-based editing system
  • Opportunity for repeat dosing to achieve efficiencies for clinical benefit

Dietrich Stephan, Chairman & CEO, NeuBase Therapeutics 

Dietrich Stephan

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