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Sessions cover scientific, clinical and commercial development of RNA therapeutics and vaccines. networking. 



Day one | Day two

Day 1 - Wednesday September 6th

9:00am LSX Welcome & Chair Opening Remarks

Jade Osei Tutu,
Senior Conference Director - Therapeutics Lead, LSX

9:15am Keynote Panel – Which RNA modalities can truly make a transformative personalized medicine?

  • How is the field advancing in RNA biology and technology platform to truly deliver a personalized approach?
  • What top approaches and indications are being fast tracked to the clinic for both small and long RNAs?
  • How are industry working together to address delivery bottlenecks?

Michael Wenger, VP Clinical Development, BioNTech
Eric Swayze, EVP of Research, Ionis
Michelle Werner,
CEO, Alltrna; CEO-Partner Flagship Pioneering
Matthew Hawryluk, EVP, CBO, Gritstone

Michael Wenger, VP Clinical Development, BioNTech  Eric Swayze, EVP of Research, Ionis  Michelle Werner, CEO, Alltrna  Matthew Hawryluk, EVP, Chief Business Officer, Gritstone

10:00am Keynote Address – Investing in RNA: modality, technology or indication?

  • Where are RNA medicines succeeding to sustain future investment?
  • Where are the technology gaps which require extra backing?
  • With the field rapidly growing how do investors decide their routes of investment (RNA modalities, supporting AI applications, RNA diagnostics or manufacturing tools)?

Nina Kjellson, General Partner, Canaan Partners
Karin Kleinhans, Partner, MRL Ventures Fund
Alicia Irurzun-Lafitte, Partner, UCB Ventures
John Boyce, CEO, Tiger Gene Venture Capital
Tim Luker, VP of Venture Science, Eli Lilly 

Nina Kjellson, General Partner, Canaan  Karin Kleinhans, Partner, MRL Ventures Fund  Alicia Irurzun - Lafitte, Partner, UCB Ventures  John Boyce, CEO, Tiger Gene Venture Capital  Tim Luker, VP Venture Science, Corporate BD, Eli Lilly

10:45am Morning Break


Oligo-Based Therapeutics Track


11:30am Chair Remarks

11:35am Presentation: Targeting DM1, DMD & FSHD Rare Genetic Indications with Novel Oligos

  • How novel oligos hold promise for targeting rare genetic indications such as DM1, DMD, and FSHD

  • How targeting the underlying genetic causes of these diseases mean oligos have the potential to provide more effective and specific treatments for patients with rare conditions

  • Delving into platform technologies being developed to safely target DM1, DMD & FSHD


12:00pm Presentation: Technologies To Accelerate RNA Medicines 


12:25pm Presentation: Advancing ASO Development Via TANGO Platform For CNS Disorders

  • How preclinical studies have shown TANGO ASOs can be used to reduce the synthesis of non-productive mRNA and increase the synthesis of productive mRNA

  • Exploring the utility of the approach in developing mutation-independent therapies for autosomal dominant haploinsufficiency diseases

  • Presenting of both pre-clinical and clinical data

Huw Nash, CBO, Stoke Therapeutics

 Huw Nash, COO and CBO, Stoke Therapeutics-1

12:50pm Presentation: ASOs In The Neurodegenerative Disease Space

  • How ASOs delivered intrathecally have now been developed successfully for genetic forms of rare neurological disorders in both children and adults

  • Next steps and the development of ASOs targeting non genetic forms of neurodegenerative diseases through the restoration of normal protein levels

  • Exploring the current clinical status of ASO intrathecal delivery, known ASO specific AE/side effects and ASO targeting sporadic forms of ALS

 Angela Genge, CMO, QurAlis

 Angela Genge, CMO, QurAlis 

mRNA Therapeutics & Vaccines Track


11:30am Chair Remarks

11:35am Presentation: Pre-Transcriptional Control of MYC Expression Using Programmable Epigenomic mRNA Medicine

  • Overexpressed in more than 50% of all cancers, MYC is a master oncogene that has been historically undruggable

  • How precision epigenomic control offers a novel strategy to treating MYC-driven diseases while overcoming issues that make it undruggable

  • Omega epigenomic controllers (OECs) are programmable mRNA therapeutic candidates designed to tunably and durably modulate gene expression pre-transcriptionally

  • Detailing how MYC-targeting OECs have demonstrated promising antitumor activity across preclinical models

  • Learn how a landmark clinical trial is underway to evaluate a first-in-class MYC-targeting OEC in hepatocellular carcinoma and other tumor types associated with the MYC oncogene

Mahesh Karande, CEO, Omega Therapeutics

Mahesh Karande, CEO, Omega Therapeutics

12:00pm Presentation: Advancing mRNA Vaccine Technologies To Improve Stability, Efficacy & Delivery

  • Overview of the Jenner Institute's work on mRNA vaccine development for various infectious diseases

  • Discussing the challenges in mRNA vaccine development and how to address them

  • Explaining and exploring how to improve vaccine stability, efficacy, and delivery

  • Insights into the future of mRNA vaccine development at the Jenner Institute - potential applications for global health and opportunities for collaborations

Cesar Lopez Camacho, Principal Investigator, University of Oxford

Cesar Lopez Camacho, Principal Investigator, University of Oxford

12:25pm Presentation: Increasing RNA Based Medicine Safety Profiles via saRNA Preclinical Development

  • Highlighting the benefits of saRNA preclinical development in enhancing the safety of RNA-based medicines

  • Explore Ziphius’ self-amplifying RNAs development as a second-generation technology to deliver more efficacious RNA vaccines

  • Insights into preclinical data for infectious diseases

Chris Cardon, CEO, Ziphius Vaccines

Chris Cardon, CEO, Ziphius Vaccines

12:50pm Presentation: Reviewing Discovery Approaches Needed To Promote Developing of mRNA Therapeutics

Next-Generation RNA Track

Rapid Fire Innovation

11:30am Chair Remarks

11:45am Presentation: Utilizing Transcriptome-Wide Screening To Identify RNA Targets

  • Transcriptome-wide screening is a powerful tool for identifying RNA targets and understanding gene expression patterns in complex biological systems

  • How by analyzing the entire set of RNA transcripts in a given sample, transcriptome-wide screening can reveal novel targets for therapeutic intervention

  • Providing insights into the molecular mechanisms underlying diseases

Peng Yue, CEO, ReVir Therapeutics

Peng Yue, CEO, ReviR Therapeutics

12:00pm Presentation: Developing Fully Programmable RNA-Based Therapies

  • How Senda has established the first-ever Atlas to explore programmable systems at the molecular level and across all kingdoms of life

  • Organizing, analyzing, and accessing the entire code of nearly limitless combinations of natural nanoparticles for programming to cells

  • Programming nanoparticles with key bioproperties to unlock the therapeutic promise of all types of information molecules and other biomolecules through the Senda platform 

Guillaume Pfefer, CEO, Senda Bioscience

 Guillaume Pfefer, CEO, Senda Bioscience_

12:15pm Presentation: Targeting Age-Related Diseases with Controllable Self-Replicating RNAs

  • Development of therapeutics for aging-related diseases using a proprietary controllable self-replicating RNA platform (c-srRNA)

  • Discussing the first clinical trial in phase I/II for telomere biology disorders with bone marrow failure, an ultra rare indication

  • Outlining the risks to patients with telomere biology disorders including a high risk of developing bone marrow failure

  • Explaining the treatment - ex vivo cell therapy potentially extending the telomeres of the patients’ hematopoietic stem cells using the ZSCAN4 gene, with multiple unique, built-in safety features

Akihiro Ko, CEO, Elixirgen Therapeutics

Akihiro Ko, CEO, Elixirgen

12:30pm Presentation: Next-Generation Lipid Nanoparticle Technology Enabling Extrahepatic Nucleic Acid Delivery

  • Enabling safe and efficient delivery of nucleic acids to a range of tissues through an unencumbered LNP toolbox

  • Discussing the range of technologies within NanoVation's IP portfolio

  • Providing examples of potential applications

Jayesh Kulkarni, CSO, NanoVation Therapeutics

 Jayesh Kulkarni, Chief Scientific Officer & Founder, NanoVation Therapeutics

12:45 pm Chair Review: The chair will share their thoughts on the rapid-fire innovation session and open the floor for Q&A

1:15 pm Lunch Break



2:30pm Presentation: Advancing Clinical Development Of Rare Muscular Diseases Through Effective Delivery Of Antibody Oligonucleotide Conjugates (AOC)

  • Harnessing the targeting power of antibodies and the therapeutic potential of oligonucleotides - AOCs offer a promising approach to treating serious diseases

  • Significantly improving patient outcomes

  • Providing an update on the use of AOCs in early clinical programs of muscular-related indications 

Mike Flanagan, CSTO, Avidity Biosciences

Mike Flanagan, CSTO, Avidity Biosciences

2:55pm Presentation: Drug And Target Discovery With Explainable AI

  • Answering the question for scientists and researchers - what is “explainable” artificial intelligence?

  • Why “explainability” is necessary for pharma and life science

  • An RNA therapeutics use case: Applying explainable AI to augment understanding

  • How to harness the power of explainable AI to reshape the future drug development 

Lykke Pedersen, Head of RNA Therapeutics, Abzu

Lykke Pedersen, Head of RNA Therapeutics, Abzu

3:20pm Presentation: Discovering Gene Silencing Targets And Accelerating The Development Of RNAi Therapies
  • Silence’s mRNAi GOLD™ platform: update on the R&D pipeline

  • Target selection and validation using Translational Genomics

  • Approaches to overcoming pre-clinical challenges in the candidate selection process
Marie Wikström-Lindholm, SVP Head of Molecular Design, Silence Therapeutics

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

2:30pm Presentation: Novel, Synthetic DNA And Delivery Modalities For mRNA Therapies And Vaccines

  • Why mRNA production is limited by availability of high quality, GMP grade DNA and how synthetic DNA produced enzymatically can address the draw backs associated with plasmid derived DNA templates

  • Introducing opDNA, a synthetic DNA template with a 3’ open end and 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

  • Achieving significantly higher mRNA yields, comparable proinflammatory cytokine/chemokine responses, and equivalent gene expression as compared to plasmid derived template

  • How next-generation delivery systems such as Hermes™ nanoparticles can enable wider use of nanoparticle based delivery across a range of therapeutic areas

Amy Walker, Director of Discovery, 4basebio

Amy Walker, Director of Discovery, 4basebio

2:55pm Presentation: Developing LNPs To Enable In-vivo Base Editing

  • Why LNPs are very efficient at hepatic delivery of nucleic acids

  • New technologies are necessary to identify novel tissue tropism

  • How Novel formulations can access new tissues

Luis Brito, VP Delivery Platform, Beam Therapeutics

Luis Brito, VP Delivery Platform, Beam Therapeutics

3:20pm Presentation: Developing Delivery Platforms That Address RNA Therapeutics Bottlenecks

Non-Linear and Regulation RNAs

2:30pm Presentation: Increasing Healthy Protein Levels To Address Genetic Diseases By Drugging regRNAs

  • Regulatory RNAs (“regRNAs”) are the cells natural rheostat system to control gene expression

  • Antisense Oligonucleotides (“ASOs”) can be used to specifically target regRNAs and increase gene expression

  • CAMP4 is progressing multiple RNA Amplifiers into the clinic to address the genetic basis of diseases

Josh Mandel-Brehm, CEO, CAMP4 Therapeutics

Josh Mandel-Brehm, CEO, CAMP4 Therapeutics

2:55pm Presentation: In-Depth Analysis Of  The Potential Of lncRNA Therapeutics

3:20pm Presentation: An Integrated Platform For In Vivo CAR Using Circular RNA

  • oRNATM technology is a new advancement in RNA therapeutics that uses engineered linear RNAs to produce highly-efficient autocatalytic circularization

  • Explaining the advantages over traditional linear mRNA therapies, including simplified production, increased protein expression, and reduced immune system response

  • How this novel approach is an opportunity to improve the safety and efficacy of CAR T-cell therapies by enabling more precise targeting of cancer cells and reducing off-target effects

Tom Barnes, CEO, ORNA Therapeutics

Tom Barnes, CEO, Orna Therapeutics

3:55pm Afternoon Break


Clinical Development

4:25pm Presentation: Antisense Oligonucleotide Therapy – Translating Advanced Oligos To The Clinic

  • Advances in ASO design and delivery have facilitated their translation from preclinical research to clinical applications, enabling more effective and targeted therapies for patients

  • Why developing robust analytical methods is critical for ensuring the quality, safety, and efficacy of ASO drugs

  • How optimizing the manufacturing process can improve efficiency, scalability, and cost-effectiveness

  • Providing an update on Ionis' new ASO technology platforms

Eric Swayze, EVP of Research, Ionis

Eric Swayze, EVP of Research, Ionis

4:50pm Presentation

5:15pm Presentation: GalAhead™: A Proprietary GalNAc-RNAi Therapeutic Platform To Downregulate Single And Multiple Genes

  • Development of 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 to simultaneously silence two or more targets

  • Presenting data validating these two technologies both in vivo and in vitro, as well as discussing applying to non-melanoma skin cancer program

Dmitry Samarsky, CTO, Sirnaomics

Dmitry Samarsky, Chief Technology Officer, Sirnaomics


4:25pm Panel: Delivery Techniques To Achieve Tissue And Cell Specificity

  • 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?

Annette Bak, Head of Advanced Drug Delivery, AstraZeneca
Luis Brito, VP Delivery Platform, Beam Therapeutics

Annette Bak , Head of Advanced Drug Delivery, AstraZeneca  Luis Brito, VP Delivery Platform, Beam Therapeutics


Small Molecules Targeting RNA

4:25pm Presentation: A New Platform To Discover RNA-Targeted Small Molecules

  • Drugging RNA holds the potential to vastly expand druggable space beyond currently addressable protein targets

  • To reduce this new modality to practice - explaining how Arrakis assembled a comprehensive technology toolbox including RNA fold prediction, high-throughput screening and optimization of small-molecule RNA interactions

Nick Marsh, Senior Director & Head of Molecular Pharmacology, Arrakis

Nick Marsh, Senior Director & Head of Molecular Pharmacology, Arrakis

4:50pm Presentation: Advancing Understanding Of Mrna Biology Regulation By Small Molecules With Mrna Lightning Platform 


  • Discovery of small molecule mRNA drugs and their mechanisms of action with automated high scale phenotypic screening in live mRNA biology and MOAi technology that uses AI to elucidate the mechanism of action of active molecules.

  • The MOAi technology acts as a "Compass" by integrating AI-driven MOA recognition and Machine Learning-guided MOA assays to identify patterns and suggest experiments to understand how compounds work and identify their molecular targets.

  • Hear about the MOA of six distinct series of molecules in oncology and immunology programs using AI approached by Anima in the last year.
Kevin Pong, CBO, Anima Biotech

Kevin Pong, CBO, Anima Biotech

5:15pm Presentation: Applications of Mass Spectrometry in RNA-Based Drug Discovery

  • How mass spectrometry can be an enabling tool in many aspects of RNA-based drug discovery

  • Why affinity-selection mass spectrometry (ASMS) is commonly used to identify small molecule ligands to oligonucleotides

  • Explaining how mass spec can also be used in both biomarker and potency assays to determine if an RNA therapeutic has functional activity in a cell-based or animal experimental model

Can Ozbal, CEO, Momentum Biotechnologies

Can Ozbal, CEO, Momentum Biotechnologies

5:40pm Networking Drinks Reception

8:00pm Close of Drinks Reception



Day 2 - September 7th

9:00am Chair Opening Remarks

9:10am Keynote Panel – Navigating and de-risking RNA discoveries with Big Pharma

  • Which RNA technologies are of the most interest and why?
  • Assorted drug development strategies including internal R&D, partnership programs and external innovation?
  • How do Pharma evaluate early-stage collaborations with biotech? 

Tamar Grossman, VP, Global Head of RNA, Gene Therapy & Delivery, Janssen
Pedro Serrano, Senior Director, Head of RNA & Protein Modulation, Takeda
Malgorzata Gonciarz, AVP, Genetic Medicine, Eli Lilly
Pierre Cote, Head of Strategic Partnership & Innovation, mRNA, Sanofi

Tamar Grossman, VP, Global Head of RNA and Targeted Therapeutics, Johnson & Johnson  Pedro Serrano, Senior Director, Head of RNA and Protein Modulation, Takeda  Malgorzata Gonciarz, Associate Vice President, Genetic Medicine, Eli Lilly-1  Pierre Cote, Head of Strategic Partnership & Innovation, mRNA, Sanofi

9:50am Presentation: Reviewing The Regulatory Considerations And Research For RNA Based Products  

  • Navigating the regulatory landscape for mRNA and oligonucleotides
  • Providing an update on the new legal categories being set for all RNA modalities 

Fouad Atouf, SVP, Global Biologics, US Pharmacopeia

Fouad Atouf, SVP, Global Biologi cs , U.S. Pharmacopeia

10:15am Keynote Panel – Innovating Manufacturing Of RNA-Based Therapeutics and Vaccines

  • How to ensure efficient scale-up of up and downstream processes
  • How is industry moving to set the next generation of RNA products to be GMP standard?
  • What CMC strategies are already in place from approved from ASOs, oligos, mRNA drugs that can be used as a gold standard template?

Andreas Kuhn, SVP RNA Biochemistry & Manufacturing, BioNTech SE
June Kim, CMC Lead, CEPI
Patrick Thiaville, CTO nucleic acids, Exothera

Andreas Kuhn, Senior Vice President RNA Biochemistry & Manufacturing, BioNTech   June Kim, CMC Lead, CEPI-1   Patrick Thiaville CTO, Nucleic Acids Exothera  

10:55am Morning Break


Oligo-Based Therapeutics Track


11:40am Chair Remarks

11:45am Presentation: Empowering Enhanced Oligo Delivery To Improve RNA Modulating Therapies In Rare Muscle Diseases

  • Oligonucleotides have long promised to transform healthcare but the delivery of them has lagged and remains a major challenge

  • How PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform leverages cell-penetrating peptide conjugates to improve the biodistribution, activity and tolerability of oligonucleotide cargo therapeutics

  • Explaining the lead product candidates in Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) leverage novel EDO peptides to reach key muscle targets, including cardiac tissue

  • How Phase 1 data in healthy volunteers suggests biodistribution to muscle tissues, potential for high levels of exon skipping and dystrophin production in DMD, while preclinical data in DM1 has shown sustained correction of mRNA mis-splicing for up to six months following a single dose

Pallavi Lonkar, VP, Head of Bioanalytical, DMPK, PepGen

Pallavi Lonkar, VP, Head of Bioanalytical, DMPK, PenGen

12:10pm Presentation: Innovating Delivery And Formulation Technologies For Oligo Based Therapeutics

12:35pm Presentation: Utilizing Endolysosomal Trafficking To Enhance Effective Oligo Delivery Safely

  • To improve the delivery of oligonucleotides to the liver, lung, and central nervous system, researchers have identified small molecules that disrupt both endocytic recycling and lysosomal fusion

  • By blocking these two processes, the molecules can trap oligonucleotides within endocytic compartments, where they can be released more easily and effectively

  • This approach has been shown to significantly enhance oligonucleotide delivery and activity, without causing the toxic effects associated with endosome or lysosome permeabilization

Aimee Edinger, Professor, Developmental & Cell Biology & Chancellor's Fellow, UC Irvine

Aimee Edinger, Professor, Developmental & Cell Biology and Chancellors Fellow, UC Irvine

mRNA Therapeutics & Vaccines Track


11:40am Chair Remarks

11:45am Presentation: Innovating And Producing R&D And GMP mRNA

  • Exploring AGC Bio’s unique mRNA Program Elements (including custom pDNA in GMP and non-GMP quantities)
  • Technical process development services based on your preferred host system
  • Procedures for manufacturing high-quality pDNA / mRNA for some of the most demanding applications
  • Single-use technology to mitigate cross-contamination risks & In-house analytics via AGC’s Global Quality Network

Gerhard Schneider, Executive Advisor, mRNA Services, AGC Biologics

Gerhard Schneider Head of Production, AGC Biologics

12:10pm Presentation: Advancing mRNA Therapeutics With Liver Disease To Market – CMC Development

  • Considerations and challenges of CMC development of mRNA therapeutics

  • Addressing the critical data for targeting hepatic conditions

  • Assess how to reduce drug substance impurities and maintain potency

Dushyant Varshney, CTO, Arcturus Therapeutics

Dushyant Varshney, Chief Technology Officer, Arcturus Therapeutics

12:35pm Presentation: Route To Clinic – Critical Steps For mRNA Process Optimization

  • Exploring the key steps when moving from the bench side through to the clinic

  • Delving into the critical process controls and optimization routes to streamline mRNA development.

Venkata Indurthi, CSO, Aldevron

Venkata Indurthi, CSO, Aldevron

Next-Generation RNA Track

Rapid Fire Innovation

11:40am Chair Remarks

11:45am Presentation: Precision tRNA Reprogramming — New Approaches To Correcting Translational Dysfunction In Cancer

  • Discuss tRNAs as a potential therapeutic strategy to enhance cancer treatment by precisely delivering anticancer agents to the tumor cells

  • Explore HOVANA platform for utilizing tRNAs as modalities

Nick Davis, Founder & CTO, HOVANA

Nick Davis, CEO, HOVANA

12:00pm Presentation: Advancing Agnostic tRNAs To Directly Modify the Proteome

  • Why agnostic tRNAs are a promising tool for directly modifying the proteome by incorporating non-natural amino acids into proteins with high efficiency and specificity

  • How the advancement of agnostic tRNAs could lead to the development of novel therapeutics for a wide range of diseases, including cancer and genetic disorders

Leslie Williams, CEO, Hc Bioscience

Leslie Williams, CEO, Hc Bioscience

12:15pm Presentation: Gene Writing- Harnessing Mobile Genetic Elements To Write DNA Sequences With RNA

  • RNA Gene Writers enable genome engineering in diverse ways, without making double-strand DNA breaks, using all-RNA compositions of matter

  • Tessera’s pioneering Gene Writer systems have the potential to make nearly any type of genetic change needed to treat or cure disease

  • Insights into also developing non-viral delivery platforms designed to deliver RNA Gene Writers to targeted tissues, potentially enabling the in vivo application

  • Recent preclinical data demonstrates the ability to write therapeutic messages in the genome of non-human primates, generation of tumor-clearing CAR T cells by all-RNA delivery, and the ability to target non-viral delivery systems to hematopoietic stem cells and T cells

Michael Severino, CEO Tessera Therapeutics; CEO-Partner Flagship Pioneering

Mike Severino, CEO, Tessera Therapeutics -1

12:30pm Chair Review: The chair will share their thoughts on the rapid-fire innovation session and open the floor for Q&A

1:05pm Lunch Break


RNA Leaders CEO Forum

2:20pm RNA Leaders CEO Forum: Evaluations  

The RNA Leaders CEO Forum provides a unique, powerful and valuable space for the candid sharing of ideas and experience between executives of ~20 RNA Biotech private companies. 

This forum will include 2-3 case studies discussing the how to assess and establish the right biotech valuation, from an investor, pharma and biotech perspective. 

It follows a strict Chatham House Rule policy and includes case study peer review and a general discussion focused on brainstorming solutions to the collective challenges of CEOs in the sector. And due to numbers requires pre-registration. 

Clinical Development

2:20pm Presentation: Targeting Solid Tumors With Self-Amplifying RNAs 

  • Delve into utilizing self-amplifying RNAs to specifically target and effectively treat solid tumors with minimal off-target effects

  • Learn about the clinical development of self-amplifying RNAs including working to produce the initial safety and efficacy clinical data.

Matthew Hawryluk, EVP, CBO, Gritstone

Matthew Hawryluk, EVP, Chief Business Officer, Gritstone

2:45pm Presentation: Approaches To Designing Clinical Trials With Defined Endpoints For RNA Modalities?

3:10pm Presentation: mRNA Vaccines And Beyond Individualized Vaccine Concepts For Oncology 

  • Vaccines using non-mutated tumor-associated antigens

  • CAR-T cell therapy plus amplifying mRNA vaccine

  • mRNA concepts beyond vaccines: mAbs and cytokines

Michael Wenger, VP Clinical Development, BioNTech

Michael Wenger, VP Clinical Development, BioNTech

RNA Editing & Cell Therapy

2:20pm Presentation: Personalized Medicine Via Therapeutic Genome Editing

  • Deep dive into pioneering editing and delivery solutions to harness the immense power of CRISPR-based technologies

  • For in vivo programs, using their proprietary (LNP) platform to deliver to the liver a two-part genome editing system: a guide RNA specific to the disease-causing gene and messenger RNA that encodes the SpCas9 enzyme, which carries out the precision editing

  • Human proof-of-concept data of NTLA-2001 and NTLA-2002 are being developed for transthyretin amyloidosis and hereditary angioedema

  • For ex vivo, how CRISPR is used to engineer T and NK cells for the treatment for immuno-oncology and autoimmune diseases

  • Exploring preclinical data of an allogeneic approach involves knocking out the endogenous T cell receptor, HLA Class II and HLA-A while retaining HLA-B and -C

Derek Hicks, CBO, Intellia Therapeutics
Derek Hicks, CBO, Intellia Therapeutics

2:45pm Presentation: Stealth Editors™ - Nuclease-Free In Vivo Gene Editing Optimized For Pronounced Pharmacology, Safety, Non-Viral Delivery And Breadth Of Addressable Pathogenic Variants

  • Stealth Editors utilize synthetic oligos to "tag" a locus and recruit cellular machinery to perform genome editing. They have the ability to correct transitions, tranversions, insertions, deletions and perform gene writing in a PAM sequence-unrestricted manner opening up a large addressable markets  

  • Multiple in vivo editing data sets across various gene targets have demonstrated pronounced pharmacology 

  • This approach is non-immunogenic and leverages high fidelity human DNA repair machinery likely resulting in a favorable in vivo safety profile 
  • These editors are low molecular weight and are delivered using non-viral technologies such as LNPs

Dietrich Stephan, CEO, Neubase Therapeutics

Dietrich Stephan

3:10pm Presentation: Treatment Of Alpha-1 Antitrypsin Deficiency Via RNA Editing

  • Understanding the technology that enables precise RNA editing

  • How RNA editing differs from other genetic medicine approaches

  • Illustrating the utility of the technology via presentation of pre-clinical data on Alpha-1 Antitrypsin

Ram Aiyar, CEO, Korro Bio

Ram Aiyar, CEO, Korro Bi

3:35pm Afternoon Break



4:05pm Presentation: Nanoparticle-Encapsulated siRNAs For Gene Silencing In The Haematopoietic Stem-Cell

  • Nanoparticle-encapsulated siRNAs have been developed for targeted gene silencing in hematopoietic stem cells, which could potentially lead to new treatment options for blood disorders and other related conditions

  • How nanoformulations have been formulated and screened for their ability to deliver RNA payloads to various tissues, enabling targeted gene suppression with siRNA, gene expression with mRNA, and even permanent genetic editing using the CRISPR/Cas9 system

  • Outlining the technology’s potential for the development of new therapies for a wide range of diseases, offering targeted and efficient delivery of RNA payloads with minimal side effects

Daniel Anderson, Professor, Chemical Engineering & Institute for Medical Engineering & Science, MIT

Daniel Anderson, Professor, Chemical Engineering and Institute for Medical Engineering and Science, MIT

4:30pm Presentation: Deep-Dive Into saRNA Target Selection And Formulation For Mature Oligonucleotide Modalities

  • Tackling 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 the durability 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 & Head of R&D, MiNA Therapeutics

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


4:05pm Presentation: Discovery & Validation Of MicroRNA Biomarkers Of Clinical Drug-Induced Organ Injury

  • How innovative scientific approaches have facilitated significant progress in (RNA) drug development

  • Explaining how safety and tolerability of drug candidates and newly approved drugs often remain a key concern, leading to labelling restrictions, black box warnings, and withdrawal of otherwise promising innovative medicines

  • Within the TransBioLine project TAmiRNA leads the discovery and validation of novel microRNA-based safety biomarkers using a unique RNA-sequencing workflow enabling absolute quantitation of RNAs in liquid biopsies

  • Development of reliable safety biomarkers that indicate injury of the liver, kidneys, pancreas, blood vessels, and central nervous system for drug development purposes

 Matthias Hackl, CEO, TAmiRNA
Matthias Hackl , CEO , TAmiRNA

4:30pm Presentation

Shraddha Sharma, Associate Director, mRNA Center of Excellence, Sanofi

Shraddha Sharma, Associate Director, mRNA Center of Excellence, Sanofi


5:00pm End of RNA Leaders USA 2023