2 FULL DAYS FOCUSED ON THE LATEST RNA DEVELOPMENTS

Oligo-Based Therapeutics Track

Translation

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

Dyne

12:00pm Presentation: Technologies To Accelerate RNA Medicines 

Entegris

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

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

  

mRNA Therapeutics & Vaccines Track

Discovery

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

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

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

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

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

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

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

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

• 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
  

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

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

• oRNA^TM 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

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

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.
   

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

Oligo-Based Therapeutics Track

Delivery

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

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

mRNA Therapeutics & Vaccines Track

Manufacturing

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

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

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

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

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

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

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

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

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

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

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

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