Day 2 – Thursday, 14 March 2024 - CET (Central European Time, GMT+01:00)
Day 2 – Thursday, 14 March 2024 - CET (Central European Time, GMT+01:00)
- Delivery and conjugates for oligos - Led by Roche
- ASOs opportunities by exploring RNA biology and mechanisms - Led by Senisca and Williams Biotech Consulting
- Raw materials and processes – moving to green chemistry options - Led by Novartis
- Clinical design for Oligos - Led by Servier
- Target selection – lessons learned from small molecules RNA Biology - Led by Anima Biotech
- Gal Gur - VP Business Development, Anima Biotech Inc.
- Felix Schumacher - Program Leader Targeted Therapeutics, Roche
- Lorna Harries - CSO, Senisca
- Renee Williams - Managing Partner, Williams Biotech Consulting, LLC
- Tomaz Einfalt - Principle Investigator II / Project Team Lead xRNA, Novartis
- Keith Mayl - ALS Strategy Lead | Senior Clinical Development Lead, Servier
- Delivery and conjugates for oligos - Led by Roche
- ASOs opportunities by exploring RNA biology and mechanisms - Led by Senisca and Williams Biotech Consulting
- Raw materials and processes – moving to green chemistry options - Led by Novartis
- Clinical design for Oligos - Led by Servier
- Target selection – lessons learned from small molecules RNA Biology - Led by Anima Biotech
- Felix Schumacher - Program Leader Targeted Therapeutics, Roche
- Lorna Harries - CSO, Senisca
- Tomaz Einfalt - Principle Investigator II / Project Team Lead xRNA, Novartis
- Keith Mayl - ALS Strategy Lead | Senior Clinical Development Lead, Servier
- Gal Gur - VP Business Development, Anima Biotech Inc.
- Characterisation, Quality and CMC considerations for mRNA Therapeutics - Led by Intertek
- Improving mRNA production and costs for 2nd-generation mRNA vaccines and therapeutics - Led by Primrose Bio
- mRNA 2.0 - Led by Sensible Biotechnologies
- mRNA delivery and expression beyond the liver - Led by AGS Therapeutics and Janssen
- Clinical routes for mRNA therapeutics - lessons learnt from mRNA vaccines - Led by Sanofi
- Marie-Hélène Leopold - CCO, AGS Therapeutics
- Miroslav Gaparek - CEO, Sensible Biotechnologies
- Tamar Grossman - VP, Global Head of RNA & Targeted Therapeutics, Johnson & Johnson
- Claire Tardiveau - Strategic Partnerships & Collaborations Lead, Sanofi
- Michele Rubino - CBO, Primrose Bio
- Helge Zieler - CEO, Primrose Bio
- John Todd - Business Development Director, Intertek
- Characterisation, Quality and CMC considerations for mRNA Therapeutics - Led by Intertek
- Improving mRNA production and costs for 2nd-generation mRNA vaccines and therapeutics - Led by Primrose Bio
- mRNA 2.0 - Led by Sensible Biotechnologies
- mRNA delivery and expression beyond the liver - Led by AGS Therapeutics and Janssen
- Clinical routes for mRNA therapeutics - lessons learnt from mRNA vaccines - Led by Sanofi
- John Todd - Business Development Director, Intertek
- Helge Zieler - CEO, Primrose Bio
- Michele Rubino - CBO, Primrose Bio
- Miroslav Gaparek - CEO, Sensible Biotechnologies
- Marie-Hélène Leopold - CCO, AGS Therapeutics
- Tamar Grossman - VP, Global Head of RNA & Targeted Therapeutics, Johnson & Johnson
- Claire Tardiveau - Strategic Partnerships & Collaborations Lead, Sanofi
The rapid development of mRNA-based COVID 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? How is Quality achieved? And how is Quality assessed?
In this interactive workshop, etherna manufacturing experts will discuss key considerations in the development of RNA therapeutics. Based upon our experience in developing our products and with expertise from producing RNA for hundreds of customer projects from research grade through GMP. Our team will present their views and seek your opinions on the next steps for the industry. Discuss topics include:
• The impact of construct design on RNA quality
• Upstream and downstream process optimization
- Phil Challis - VP Manufacturing Services, etherna
- Exploring the Potential of the Dark Genome as a Novel Therapeutic Target Landscape
- Leveraging Long Non-Coding RNAs as Safe and Effective Targets
- Early Pre-Clinical Data
- Samir Ounzain - CEO & Scientific Co-Founder, HAYA Therapeutics SA
- MicroRNAs (miRNAs) are short endogenous non-coding RNAs that function as important post-transcriptional regulators of gene expression of many biological processes and have been implicated in the pathogenesis of a wide range of human diseases, including cardiometabolic diseases.
- We identified microRNA-22 (miR-22) as a key regulator of lipid and metabolic homeostasis using miR-22 knockout and transgenic mice, respectively.
- Furthermore, miR-22 is upregulated in adipose tissue of obese human subjects and its levels correlate with the severity of fibrosis in MASH patients. The therapeutic potential of miR-22 inhibition in MAFLD and obesity was assessed in mouse models of NASH and obesity, non-human primates, and human liver organoids using a systemically delivered antimiR-22 oligonucleotide compound.
- Here, we report that pharmacological inhibition of miR-22 has a great protective effect towards hepatic lipid accumulation, inflammation, fibrosis and weight loss.
- Riccardo Panella - CSO, Resalis Therapeutics
- DM1 is a rare neuromuscular disease with no disease modifying treatments. Arthex’s therapeutic approach in DM1 is miR-23b inhibition, which demonstrated a dual beneficial effect on DM1 models.
- In order to alleviate functional and molecular symptoms of DM1 disease, preferentially in the tissues affected by the disease, Arthex is developing a safe lipid-conjugated antimiR-23b oligonucleotide (ATX-01).
- Explore how the conjugated antimiR reaches muscle, heart, brain and diaphragm more efficiently than the naked molecule in the HSALR mice and shows improved PK/PD properties. Importantly, the level of compound delivered into brain by IV injection in the DMSXL animals was enough to produce important target engagement and concomitant rescue of exploratory behaviour of the mice.
- Beatriz Llamusi - CEO & Co-Founder, Arthex Biotech
- NtensifyTM is an automated technology that synthesizes and purifies mRNA that offers a tailored solution for mRNA vaccine developers, supporting the entire drug development journey from candidate discovery to commercial production.
- NtensifyTM 's distinctive advantages include a de-risked construct-agnostic mRNA process that ensures high yields and quality, reduced minimizing reagent consumption, and a unique design that eliminates the need for resource-intensive scale-up.
- NtensifyTM 's streamlined vaccine production capability significantly reduces reagent costs by an order of magnitude of 2 to 3 times and can achieve a remarkable time advantage for entering the market, offering a transformative solution to safeguard global communities.
- Thomas Guyon - Product Marketing Manager, Quantoom Biosciences
- Expanded use of conventional mRNA vaccines has limited by unacceptable side effects at doses required for protection
- First generation srRNA showed some improvements in bioactivity, but were correspondingly more toxic narrowing their utility
- Here, we show next generation srRNA have major bioactivity improvements clinically, showing capacity to confer protection at very low doses without corresponding increase in toxicity
- Nathaniel Wang - CEO, Replicate Bioscience
ST Pharm has developed its own mRNA platform technology and lipid-nanoparticle (LNP) drug delivery system, known as STLNP®. The novel ionizable lipids used for STLNP formulation show similar physicochemical property and performance to other well-known LNPs.
STLNPs could be delivered to a specific organ depending on the optimized conditions, as determined by in vivo biodistribution. hEPO mRNA encapsulated STLNPs were compared with various other LNPs to evaluate the protein expression level and the adverse effects of immune stimulation. STLNPs demonstrated higher hEPO expression and lower immune activation effect with no MCP-1 and IL-6 secretion, which were comparable to other known LNP system.
In vivo animal (mice) delivery efficiency and antigen-specific immune response were investigated utilizing SARS-CoV-2 spike mRNA formulation with several different STLNPs. Some STLNPs proved to be good drug delivery system for prophylactic and therapeutic mRNA vaccines. The repeated GLP toxicity study in SD rats also proceeded to evaluate any potential toxicity, and turned out to be less toxic.
The STLNP® is an efficient and effective RNA delivery system including mRNA, circRNA, and saRNA in the field of cell and gene therapy. In this presentation, we will introduce STLNPs’ diverse applications to demonstrate the targeted drug delivery
- Joo-Sung Yang - SVP, ST Pharm
- Visualizing the complete mRNA life cycle from multiple disease models using High content - High throughput mRNA imaging technologies
- Decoding mRNA biology underlying disease phenotypes with AI using mRNA image neural networks, knowledge graph, large language model, and its copilot
- Applications in drug discovery, target discovery, mRNA vaccine optimization, and RNA-based drug optimization with examples and case studies in Oncology and Immunology
- Gal Gur - VP Business Development, Anima Biotech Inc.
Biophysical binding assays provide a compelling approach for targeting RNA as a therapeutic modality in drug discovery applications. This talk will cover simple, automated and high throughput workflows utilizing mass spectrometry (MS) to measure drug candidate binding. Two workflows will be described in detail: 1) non-covalent/reversible binding by Affinity Selection Mass Spectrometry (ASMS) and 2) covalent/irreversible binding by intact mass shift assays. Both methodologies are fully solution phase and facilitate the efficient interrogation of large data sets in a high-throughput manner followed by further hit candidate characterization. Some of the advantages of these methods over other drug target binding assay methods are minimal target requirement, straightforward assay development, protocol flexibility and target versatility. Primary hits are immediately identified and available for further confirmation and follow-up experiments (eg. Binding affinity/Kd, Kinact/Ki measurements, peptide mapping, quantitative proteomics, etc.). Confirmed hits can be further followed up in cell-based assays to interrogate attenuation of protein expression or splicing.
- Small molecule discovery using NMR and AI
- Inhibitors targeting ncRNA
- siRNA conjugates that interact with proteins in the RISC complex
- Marcel Blommers - CSO, Saverna Therapeutics
- In our earlier discovery efforts, miRNA-10b was identified as a master regulator of the viability of metastatic tumor cells
- This knowledge allowed us to develop a therapeutic (TTX-MC138) based on miR-10b inhibition that could cause complete and persistent regression of metastases in cancer models with no evidence of systemic toxicity
- For clinical development of TTX-MC138, we conducted critical, exploratory IND enabling studies in rats, dogs, and non-human primates resulting in FDA authorization (IND163800) for initiation of an ongoing microdosing Phase 0 clinical trial in patients with advanced metastatic cancer of multiple tissue origins to assist in the identification of susceptible tumor types (or patients), but also to support future clinical efforts by providing proof of concept and quantification of delivery to clinical metastases
- Zdravka Medarova - Founder & CTO, TransCode Therapeutics
- Explore ALN-APP Phase 1 clinical design and data
- Insight's into Alnylam's modular and reproducible platform for silencing CNS disease genes
- Paul Nioi - VP, Discovery & Translational Research, Alnylam Pharmaceuticals
- Respiratory syncytial virus (RSV) is the leading cause of lower respiratory tract infections worldwide, causing significant morbidity and mortality on both infants and the elderly. There are two major antigenic subtypes of RSV, RSV-A and RSV-B, with divergent RSV fusion (F) protein sequences. Their epidemic predominance can alternate during different seasons and also overlap within a season, demanding vaccines that can protect from both RSV subtypes. Currently, widely available and cost-effective preventative options are limited. However, maternal RSV vaccine and vaccine for older adults, which are the subtypes A and B pre-F protein-based vaccines, recently became available clinically with approximately 70% protection efficacy.
- We have developed a novel mRNA-based RSV vaccine (VER-027) expressing the prefusion forms of both subtypes A and B F proteins and present the preclinical results from mouse immunogenicity and protection efficacy studies of VER-027.
- Sujin Lee - Professor, Emory University
- Compacting and modularising mRNA product manufacture to accelerate discovery, and scaling to align with demand
- Implementing novel intensification technologies at GMP level for pandemic readiness
- Evaluating the opportunity of smart manufacturing to transform manufacturing methods towards cost-effective commercialisation
- François Hallac - Post-Doc Manufacturing Systems, King's College London
- 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
- César López-Camacho - Principal Investigator, University of Oxford
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.
- Christalyn Rhodes - AVP - Genetic Medicine -External Partnerships and Academic Collaborations, Eli Lilly
- Michael Kyriakides - Investment Partner, Syncona
- Ajan Reginald - CEO, Roquefort Therapeutics
Sail Biomedicines' platform harnesses the power of multiple first-in-category technologies to unlock the comprehensive programming of medicines for the first time:
- First-in-category programmable payloads of Endless RNA™ (eRNA), that can be programmed to express diverse proteins inside the body;
- Programmable nanoparticles that leverage a universal chemical code that enables directed and repeatable deployment of payloads directly to cells and tissues of choice;
- And emerging, proprietary AI technologies, which together offer vast therapeutic potential
- Trevor Hallam - CSO, Sail Biomedicines
The mRNA Technology Transfer Programme represents a critical effort to promote global health equity by enhancing access to vaccine technology in low- and middle-income countries (LMICs). This initiative is primarily focused on mRNA vaccine technology, which gained significant attention due to its pivotal role in developing COVID-19 vaccines.
Here's how the mRNA Technology Transfer Programme aims to accelerate equity in vaccine access:
- Technology Sharing: Central to this programme is the transfer of mRNA vaccine technology to LMICs. This involves sharing knowledge, skills, and technical know-how so that these countries can produce vaccines domestically
- Capacity Building: The programme supports efforts to build the necessary infrastructure in LMICs to manufacture mRNA vaccines. This includes setting up production facilities fit for purpose, ensuring quality control, and developing regulatory frameworks that meet international standards
- Training and Development: Another critical aspect is training the local workforce in LMICs to operate mRNA vaccine production facilities. This ensures that the knowledge transfer is sustainable and that local teams can independently manage production processes
- Research and Development Support: Beyond production, the programme supports research and development efforts in LMICs to advance their own vaccine candidates using mRNA technology. This could help diversify the types of vaccines available and tailor them to the specific needs of different regions
By enabling LMICs to produce their own vaccines, the programme aims to make these vaccines more accessible and affordable for their populations. This is crucial for ensuring equitable access to vaccines, especially during pandemics or outbreaks where supply constraints can limit vaccine availability in poorer countries.
- Ike James - Head of Technology Transfer, Medicines Patent Pool
- What technologies and scientific platforms are considered as RNA 2.0
- 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?
- How are industry working together to address bottlenecks around first generation products to make room for new technologies?
- Dan Staner - VP, General Manager Germany & Switzerland & Head of Middle East Region, Moderna
- Morten Lindow - Co-Founder & Senior Lead RNAHub, Roche
- Marie Wikstrom-Lindholm - CSO, Silence Therapeutics
- Tamar Grossman - VP, Global Head of RNA & Targeted Therapeutics, Johnson & Johnson
- Tilo Netzer - Head of Commercial Development mRNA Business Uni, Lonza
- Sonja Merkas - Freelance Manager, Freelance