Showcasing the powerful capabilities of our Sunny Suite platforms = ensuring consistent high-throughput formulation screening through process optimization and pre-clinical scale-up

• Illustrating significant acceleration in LNP formulation screening—performing up to 96 experiments in under 6 hours
• Highlighting the seamless transition to process optimization—adjusting flow rates and ratios—and the effortless scale-up to continuous flow on a single instrument

• Ionizable lipids are known to present varying degrees of immunotoxicity. Understanding the role of key cellular mediators such as the NLRP3 inflammasome in this context will be key to developing the next generation of safe, well tolerated non-viral delivery systems
• We developed a series of bio-degradable lipids that are rationally engineered to elicit specific immune responses
• Applications of these lipids to create precision mRNA therapeutics and vaccines will be presented

• Advancing a lncRNA tailored drug development platform to discover potential therapeutic targets in the dark genome
• Update on early clinical candidate, HTX-001, for treatment of nonobstructive hypertrophic cardiomyopathy

• Highlighting new prospects of regRNA as an untapped therapeutic target
• Using a programmable next-gen sequencing platform - RAP Platform^TM - to map regRNA and identify ASO drug candidates.
• An update on clinical progression through to dosing studies

• An overview of NCTM’s mRNA production process, from pDNA production to bulk freezing
• A list of QC assays and CQA ranges for the NCTM mRNA platform process
• A summary of the manufacturing-scale, hands-on training course for mRNA production, created in collaboration with industry partner
  
  

• Leveraging engineered tRNAs to overcome genetic disease
• Using ‘PTCX’ to design tRNAs that recognize and suppress non-sense mutations via insertion of required amino acid
• Insight into new secondary tRNA platform, ‘SWTX’, enabling labelling of disease causing proteins for degradation

• Investigate the potential of self-amplifying mRNA to create effective immunotherapies for solid tumors
• Evaluate the of safety, tolerability, and efficacy of saRNAs in the clinic

The manufacture of high-quality, GMP grade DNA is a major bottleneck in the production of mRNA and viral vectors for use in gene therapy and vaccines.

• Evaluate a scalable, fully enzymatic synthesis process for the production of linear DNA constructs via our Trueprime™ amplification technology
• Compare the synthesis of in vitro transcribed (IVT) mRNA using opDNA™ versus linearised plasmid DNA across a panel of constructs ranging from 1.5kb to 9kb.
• See how the technology can overcome the difficulties associated with complex polyA tails for mRNA constructs, which are inherently difficult to synthesise via bacterial propagation systems

• Expanded use of conventional mRNA vaccines has been 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

• panCAR combines a novel, synthetic, circular coding RNA platform (oRNA) and immunotropic lipid nanoparticles (LNP) to drive CAR expression on surface of multiple immune effector cells after i.v. administration, eliminating the need for external manufacturing.
• LNPs deliver oRNA payloads to immune effector subsets including T cells, NK cells and macrophages in vivo, potentially increasing efficacy and broadening application beyond hematologic malignancies.
• oRNA-enabled panCAR therapies promise a transient, re-dosable and scalable immune cell therapy without requiring immunodepletion

• Exploring a tRNA biology platform that utilizes ML and high-throughput automated in vitro assays to design and test tRNA molecules with optimized sequences and chemical modifications

• Developing tRNA therapeutics that identify and correct nonsense mutations (also called premature termination codons) to restore protein production

• How to engineer vectors for circular-mRNA expression
• Enabling reduced dosing by enhancing payload expression level and potency
• Dual-function gene therapy vectors for a “remove-&-replace” strategy

This workshop will equip RNA developers with the knowledge and strategies to navigate the critical steps in RNA production and delivery processes:

• Scaling IVT mRNA production for clinical applications

• Utilizing novel LNP formulations for efficient mRNA delivery

• Selecting high-quality siRNA for reliable research data

• Implementing robust quality control measures for RNA therapeutics

• Comprehensive characterization of mRNA therapeutics including detection of dsRNA and off-target cellular changes
• Precision optimization of mRNA therapeutics including codon selection and reduction of miRNA regulation
• Multiomic profiling of siRNA and small molecule targets and direct measurement of off-target binding

• Discussing the opportunities in RNA drug and clinical design
• Optimizing RNA therapeutics through large-scale analysis – what key tools are required?
• What lessons can be learnt from current short and long RNA clinical trials?

• Using ImmunoPrism, an ML-based predictive immune modelling platform, to refine predictive biomarkers for immuno-oncology patient response to treatment
• An update on clinical validation studies focussing on HNSCC (Head and Neck Squamous Cell Carcinoma), NSCLC (Non-Small-Cell Lung Cancer), CRC (colorectal cancer) and UCC (urothelial cancer).

• Advancing a new standardised approach to RNA therapeutic analytics using mass spectrometry
• Driving precise measurement of cellular RNA drug uptake, to improve delivery processes