Day 2 – Thursday 5th September 2024 - PT (Pacific Time, GMT-08:00)
Day 2 – Thursday 5th September 2024 - PT (Pacific Time, GMT-08:00)
The RNA Leaders ‘Women in RNA (WiRNA) Forum’ is a dedicated space to empower and connect women and allies who are shaping the future of RNA research and therapeutics. Networking Breakfast to hear and learn about career journeys.
- Tamar Grossman - VP, Global Head of RNA & Targeted Therapeutics, Johnson & Johnson
- Christine Esau - VP, Biology, Arrowhead Pharmaceuticals
- Jacob Sten Petersen - SVP Nucleic Acid Therapeutics, Novo Nordisk
- Divalent siRNA consists of two identical siRNA duplexes linked at the sense strand. This configuration confers broad distribution throughout the brain and spinal cord following a single dose into the cerebrospinal fluid
- The siRNA backbone chemical motif and ionic formulation have been engineered for potency and tolerability. Broad, durable, and safe target knockdown is demonstrated in non-human primate
- Effective knockdown of target transcript by di-siRNA is applied to make candidate di-siRNA therapeutics for syndromes of unmet medical need including Huntington’s Disease, genetic epilepsy, and pain
- Stefan McDonough - SVP Neuroscience, Atalanta Therapeutics
- Understanding the drivers of ALS to create precision therapies
- FlexASO platform to produce ASOs targeting STATHMIN-2 loss to decrease axonal degeneration for TDP-43 associated ALS
- Kasper Roet - CEO and Founder, QurAlis
- Exploring the key features of siRNA design for CNS delivery and silencing
- Chemical architecture as a tool to modulate PK/PD properties of siRNA
- Vignesh Hariharan - Post Doctoral Associate, University of Massachusetts
- Pioneering a peptide-based “Trojan horse” carrier technology to deliver therapeutics such as ASO’s and siRNA’s across the BBB and into brain cells following intravenous administration using receptor-mediated transcytosis (RMT).
- Utilisation of intravenous delivery ensures uniform distribution to all regions of the brain at a fraction of the dose that would be required if delivered intrathecally.
- Exploring a lead program that targets Huntington's protein and completely halted disease progression in humanized mice studies.
- Discussing further indications in neurodegenerative diseases with gain in function, such as ALS, Alzheimer’s disease, Parkinson’s disease and frontotemporal dementia.
- Yacoub Habib - CEO, Ophidion
- Akihiro Ko - CEO, Elixirgen Therapeutics
- Discuss strategies for directing mRNA vaccines to specific tissues or cell types for a more targeted response
- Explore formulations beyond standard LNPs to address stability, immunogenicity, or ease of administration.
- Ashish Sarode - Associate Director, Sanofi
- Dry powder material thanks to disruptive, unique technology formulating and drying the most fragile RNA/LNP composition
mRNA activity preserved or even increased in dry powder form, proven in-vitro and in-vivo
Substantially lower costs, new treatments enabled, and particle engineering possible for inhalable RNAs
- Ann Gidner - CEO, Ziccum
- The landscape of mRNA medicines is evolving, marked by an expanding pipeline of drugs, a diverse range of targeted prophylactic and therapeutic indications, and advancements in drug design and delivery
- Innovative technologies and solutions are imperative to address key challenges in mRNA manufacturing, safety, and efficacy
- Primrose Bio develops and supplies advanced RNA polymerases, the key enzyme used in mRNA manufacturing by in vitro transcription (IVT). The company’s panel of RNA Polymerases (Prima RNApols) enhances mRNA quality and lowers costs by improving target capped mRNA yield and reducing the amount of expensive IVT reagent input, while greatly reducing double-stranded RNA (dsRNA) impurities, to address manufacturing needs across a wide range of therapeutic and vaccine applications, capping chemistries, and other mRNA manufacturing and product requirements
- Diane Retallack - COO, Primrose Bio
The rapid development of mRNA-based vaccines was a remarkable achievement. However, the industry consensus is that the achievement and assessment of quality must accelerate to drive new RNA product development. But what does quality mean for an RNA therapeutic?
- Discuss key considerations in the development of RNA therapeutics
- Evaluate case studies of scaling producing RNA for hundreds from research grade through GMP
- Phil Challis - SVP Manufacturing, etherna
- Rabia Khan - CEO, Serna Bio
- Exploring OPERA (Oligonucleotide Promoted Editing of RNA), a platform for ADAR (Adenosine Deaminase Acting on RNA) based editing to address diseases of the liver and CNS
- Explaining the benefits of OPERA compared to existing gene therapy and editing approaches, such as reduced risk of off-target effects, simplified delivery and increased stability
- Todd Chappell - COO, Korro Bio
- Sophia Lugo - CEO, Radar Therapeutics
- The RNAfix platform has broad therapeutic application to correct missense mutations, modulate protein expression, or modify protein-protein interactions
- Using diffusion models trained on billions of proprietary experimental data points, the RNAfix platform can generate specific and efficient ADAR-recruiting gRNAs for any adenosine transcriptome-wide
- In pharmacology models, RNAfix achieved >90% target editing in the mouse and non-human primate brain, demonstrating unparalleled efficiency and robust translatability across species
- David Huss - CSO, Shape Therapeutics
- Splice Editors increase trans-splicing efficiency by leveraging CRISPR-Cas systems
- Splice Editor-enabled multi-kilobase edits unlock several key indications and pathogenic variants
- Splice Editing is a promising new modality for RNA editing
- Jacob Borrajo - CEO, Amber Bio
- Creating a comprehensive platform to support oligonucleotide discovery and development
- Enabling simple, cost-effective and fast synthesis across a broad range of oligonucleotides, including ASOs, siRNA, aptamers, oligo conjugates, PMOs and PPMOs
- Developing WuXi TIDES, to enable greener and more efficient development solutions
- David Madge - VP Discovery Services, WuXi AppTec
- Introduction to GalAhead™, a GalNAc-RNAi therapeutic platform
- Exploring the validation of the GalAhead™ muRNA technology in vitro and in vivo
- A progress report on muRNA GalAhead-based program
- Jim Weterings - VP Research, RNA Therapeutics & Delivery, Sirnaomics
- Expanding delivery opportunities with an Antibody Oligonucleotide Conjugates (AOCTM) platform utilising mAbs to achieve extrahepatic delivery of oligonucleotides
- Data-informed engineering of AOC components and their characteristics
- Advancing siRNA based AOCs to treat neuromuscular diseases
- Ramana Doppalapudi - VP, Chemistry, Avidity Biosciences
- Delve into developing the LUNAR mRNA platform
- Examine early clinical data on applying LUNAR mRNA for the treatment of liver disease
- Discuss expanding the scoping to identify respiratory targets with pre-clinical models
- Pad Chivukula - CSO & COO, Arcturus Therapeutics
- Discuss targeting HPV-driven tumors with mRNA therapies
- Learn how to streamline the manufacturing process by developing microfluidic technology for efficient mRNA production
- John Stubenrauch - COO, Nutcracker Therapeutics
• Accelerating discovery of saRNA for vaccine and therapeutic applications
• Discussing pre-clinical results for an saRNA vaccine program
• Differentiating saRNA discovery and design for a therapeutic application from a vaccine approach
- Rachel Groppo - Director, mRNA Research & Discovery, Johnson & Johnson
- Building an integrative RNA-targeting small molecule discovery platform that integrates large-scale genomics, advanced screening, and state-of-the-art med chemistry to identify and develop drug-like small molecule regulators of RNA target expression.
- Discussing lead program as a case study to demonstrate our discovery process. By targeting the RNA of an oncogenic transcription factor, we are able to show stellar anti-tumor in vitro and in vivo efficacy and robust PK-PD relationship, unlocking the therapeutic potential of this previously undruggable target.
- Simon Xi - CEO, Rgenta Therapeutics
- Exploring RBP (RNA binding protein) biology and its regulatory function in post-transcriptional processing of RNA
- Modulating gene expression by drugging RBPs to restore pathogenic mutations associated with neurodegeneration and cancer
- Bhawanjit Brar - CEO, Jaan Biotherapeutics
- Exploring LEADTM (Ligand and Enhancer Assisted Delivery) to achieve extrahepatic delivery of siRNA
- Focusing on targets in the muscle, adipose and CNS
- Weimin Wang - CEO, Sanegene Bio
- The iNeuroRx® platform consists of ASO design technology and a best-in-class ALS / FTD disease modeling platform, which assesses efficacy. The platform is crucial for target discovery and validation, and ASO candidate optimization
- The iNeuroRx® platform provides for the identification of the most potent, durable and safe antisense oligonucleotide (ASO) drug candidates
- AcuraStem has three ASO programs in development targeting UNC13A, SYF2 and PIKFYVE (partnered with Takeda)
- Sam Alworth - CEO, AcuraStem
- Look how precision epigenomic control offers a novel strategy for treating MYC-driven diseases while overcoming issues that make it undruggable
- 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
- 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
- Cesar Lopez-Camacho - Group Leader, Vaccitech
- Gary Xiao - Professor, Dalian University of Technology
The goal for the next generation of CRIPSR-based medicines is the development of potent and safe therapeutics that can be delivered in vivo specifically to the target cells of interest. The mRNA/LNP format is currently showing the most promise to achieve this challenging goal, and a number of levers can be optimized to drive progress. These include:
Vehicle optimization (LNP compositions and addition of targeting moieties to the surface (tLNPs)Cargo Optimization (mRNA optimization (including UTR optimization and use of miRs) and gRNA optimization (including modifications and shorter guide lengths)Analytical method development (for characterization of these complex entities)
- Steven Wolk - VP, Chemistry, Editas Medicine
- Developing RNA Gene WritersTM that utilize TPRT to write or rewrite the genomeEnabling stable long sequence writing of whole genes as well as short sequence re-writing with no double-stranded breaks
- Exploring the benefits of DNA Gene Writers, including the potential for permanent correction
- Insight into novel LNP technology for Gene WritingTM and how this enables improved safety profile, simplified and cost-effective manufacturing and reduced genotoxicity
- Hari Pujar - COO, Tessera Therapeutics
The RNA Leaders CXO 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 right approach to engage and partner with pharma assessing the key factors why pulling together a biotech valuation, from an investor, pharma and biotech perspective. It will also include a general discussion focused on brainstorming solutions to the collective challenges of CEO/CSO/CBOs in the sector.
- Tim Luker - VP Ventures, Eli Lilly & Company
- What emerging trends and breakthroughs in RNA design, delivery systems, and large-scale manufacturing are investors excited about in RNA?
- Where are RNA technologies and platforms succeeding to sustain future investment?
- Where are the technology gaps that require extra backing?
- With many RNA companies still pre-seed, how can biotechs work with potential investors to bridge the increasing demand on data to secure investment?
- Qing Zhang - Partners, LDV Partners
- Michelle Werner - CEO, Alltrna
- Kent Rogers - Partner, ARCH Venture Partners
- Michael Hodges - Partner, Claris Ventures
- What technologies and scientific platforms are considered as next-gen RNA?
- How is the field advancing in RNA biology and technology platforms to be ready for next-gen applications?
- What top approaches and indications are being fast tracked to the clinic for both small and long RNAs?
- Trevor Hallam - CSO, Sail Biomedicines
- Leslie Williams - CEO, hC Bioscience
- Pad Chivukula - CSO & COO, Arcturus Therapeutics
- Rachel Groppo - Director, mRNA Research & Discovery, Johnson & Johnson