Day 2 – Thursday 6th March 2025Thursday, 6 March 2025 - CET (Central European Time, GMT+01:00)

1. Developing a personalised mRNA vaccine – CMC challenges and considerations
2. Innovating mRNA therapeutic dosing and administration
3. Clinical progression of mRNA therapeutics & vaccines
4. Advancing Next-Generation Delivery Solutions for mRNA
5. Optimizing mRNA targeting and characterisation

In an ever-growing space, key leaders discuss how to protect RNA innovation.

• Untangling a highly fragmented RNA IP environment across mRNA, oligonucleotides and next generation RNA approaches
• Reviewing IP strategy for RNA platform vs RNA products
• Discussing how to navigate a saturated LNP delivery space, and the route to filing a patent on new delivery technologies

• Creating an AI-designed endless RNA (eRNA) and programable natural nanoparticles, to achieve precise delivery and extended therapeutic durability in patients with cancer, metabolic disease, infectious disease, rare disease and autoimmune disorders

• Insight into preclinical data and movement into the clinic

• Exploring wider srRNA pre-clinical candidates and their journey into the clinic
• Reviewing recent clinical data on saRNA prophylactic rabies vaccine, RBI-4000

• Progressing a novel circRNA approach to improve patient outcomes with Hantaan virus and Yellow Fever virus
• Crafting an innovative nanoparticle delivery technology that ensures room temperature stability

• Elevating endogenous utrophin protein expression via the RNAa mechanism
• Delivering duplex RNA to muscle and heart by proprietary LiCO platform
• in vivo evaluation of PD and efficacy in utrophin humanized mdx mice

• Pioneering the next generation of genetic medicines with our Gene Writing™ platform, designed to introduce a broad range of edits to the genome from inserting whole genes or exons to introducing single nucleotide changes
• Developing RNA Gene Writers that use target-primed reverse transcription (TPRT) as a mechanism to edit the genome with all-RNA compositions
• Advancing a proprietary non-viral delivery platform leveraging lipid nanoparticles (LNP) to deliver Gene Writers to the liver, hematopoietic stem cells, T cells and beyond

• KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro’s proprietary RNA editing platform, Oligonucleotide Promoted Editing of RNA (OPERA™). KRRO-110 is designed to co-opt an endogenous enzyme, Adenosine Deaminase Acting on RNA’s (ADAR), to edit the “A” variant on SERPINA1 RNA, repair an amino acid codon, and restore secretion of normal AAT protein.
• This repair of the endogenous protein has the potential to clear protein aggregates from within liver cells to create a potentially clinically differentiated benefit for liver function and to preserve lung function by providing an adequate amount of normal AAT protein.

• Delving into the LUNAR Delivery Platform

• Discussing clinical progression of ARCT-810 and ARCT-032 programs to improve outcomes for patients with Cystic Fibrosis and Ornithine Transcarbamylase (OTC) Deficiency

• Advancing mRNA-based ATAK CAR therapy to detect and eliminate GPC3-advanced hepatocellular carcinoma, with an update on phase 1 patient dosing

• Discussing clinical progression of secondary program to treat GPC3-breast cancer

• Updates on Phase 1 & 2 clinical candidates against influenza, RSV and acne vulgaris, and sharing outlook on future indications
• Discussing how integrated translational science enables us to improve immunogenicity and reactogenicity of mRNA vaccines

• MGMT-unmethylated glioblastoma has a poor prognosis, with a median overall survival of approximately 12 months after surgery and chemoradiation with temozolomide
• mRNA vaccines have been shown to induce CD4+ and CD8+ T cell responses against a variety of cancer antigens and offer the possibility to encode multiple antigens on a single construct
• Results from an ongoing phase 1 clinical trial evaluating the safety and immunogenicity of CVGBM, an investigational multiantigen mRNA vaccine, in patients with newly-diagnosed and surgically resected MGMT-unmethylated glioblastoma show promising immunogenicity with good tolerability

The RNA Leaders CXO Forum provides a unique, powerful and valuable space for candidly sharing ideas and experiences 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 and industry 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.