Day 1- Wednesday September 10thWednesday, 10 September 2025 - EST (Eastern Time, UTC-05:00

• Developing a novel reactor system that co-immobilizes T7 polymerase and DNA templates, enabling efficient, high-purity mRNA synthesis with reduced dsRNA impurities

• Enabling scalable and cost-effective, streamlined mRNA therapeutics manufacturing

• Adoption of RNA as a vaccine and therapeutic modality highlights the need for robust, cost-effective production and improved targeted delivery.
• Look into how a rapid and efficient manufacturing process that combined with in-process analytics enable achieving IVT yields up to 25 g of RNA per L and attaining 80-100 % of RNA recovery after purification.
• Discover how innovating mRNA-LNP formulation supported by in-process analytics can lead to the generation of mRNA LNPs that have expanded extrahepatic biodistribution, improved drug delivery and cellular targeting.

• Key process adaptations and challenges in scaling individualized cancer vaccines

• Strategies to streamline and automate bespoke mRNA vaccine workflows for clinical use.

• Discuss recent discoveries highlighting the diverse roles of RNAs, with a focus on microRNAs in gene regulation and cellular processes beyond canonical translational repression.

• Discuss the involvement of microRNAs in complex biological pathways and their implications for human health and disease.

• Discuss the potential of microRNA-based diagnostics across various

• Jaan Biotherapeutics Inc. is a private company that is developing novel therapies to activate the cardiac regeneration process in diseased hearts using proprietary technology.

• The technology manipulates microRNAs to activate an endogenous cardiac muscle regeneration process which has been shut down in the adult human heart during evolution.Our current focus is Ischemic Heart Disease (IHD), but the therapy can be applied to many cardiac diseases where cardiac muscle regeneration is required.

• The chemistry of oligonucleotides has played a crucial role in giving drug-like properties to oligonucleotides.
• To optimize oligonucleotide-based therapeutics, we explored oligonucleotide shapes that bring the 3'- and 5'-ends into transient cyclic structures.
• Oligonucleotide spatial structures exhibit reduced protein binding because of their shape and diminished interactions with pattern recognition receptors resulting from the inaccessibility of the 5’-end.
• These designs find extensive applications across diverse mechanisms of action for RNA therapeutics, including RNaseH-mediated knockdown, modulation of splicing, siRNA, and gRNA.

Developing a proprietary bio-chip base manufacturing system which integrates RNA biochemistry and microfluidic engineering, for fully automated and scalable production of mRNA therapeutics and vaccines

• Implementing green chemistry and energy-efficient processes to reduce the environmental footprint of mRNA vaccine production.

• Exploring sustainable sourcing of raw materials and waste reduction strategies

Discussing how AI-driven automation can streamline and optimize the mRNA vaccine manufacturing process, improving scalability, reducing production time, and ensuring consistent quality for improved global access.

• EVE16 (Engineered Valency-Enhanced CD16A) is an immunoactivating receptor whose stability, surface expression, and function are dependent on the signaling adaptor proteins CD3ζ and FCER1G. These adaptors are selectively expressed in T cells, NK cells, and monocytes, restricting EVE16 expression to these immune cell types.

• Targeted LNP delivery of EVE16-encoding mRNA enables in vivo expression specifically in immune cells, allowing for high-precision immune cell engineering.

• EVE16 simultaneously interacts with a target receptor and the Fc domain of monoclonal antibodies, enabling synergistic activity with therapeutic mAbs.

• Exploring the use of ligand-siRNA conjugates to selectively silence SLC proteins in kidney cells.

• Application of this novel approach to improve patient outcomes of systemic disease

Pioneering the use of CRISPR technologies to modulate long non-coding RNAs driving cancer progression

Exploring a first-in-class USP6-based mRNA immunotherapy to upregulate immunostimulatory pathways associated with an anti-tumour immune response

RNAi is well-positioned to positively impact the lives of patients living with obesity and related metabolic disorders, due to its strong clinical track record of safety, efficacy and durability. As a modality, mRNA silencing enables pharmacological intervention in diverse disease-associated mechanisms including thermogenesis, lipogenesis, energy expenditure and inflammation. SanegeneBio is developing differentiated tissue-selective delivery technologies using our LEAD (Ligand and Enhancer Assisted Delivery) platform. In this presentation, we will share Phase 1 clinical data demonstrating potential for best-in-class siRNA payloads, our novel ligand-based approaches targeting adipocytes and skeletal muscle, and preclinical proof-of-concept for several obesity targets. Together, these data support the promise of RNAi-based medicines as a transformative modality in the treatment of obesity—capable of addressing limitations of existing therapies and establishing a new standard of care.

SUMMARY BULLETS:

• Novel ligand-mediated approaches for selectively targeting adipocytes, muscle and other metabolic compartments are needed to realize the potential of RNAi for obesity therapy
• LEAD technology represents a new ligand-and-enhancer conjugation approach, enabling improved specificity and potency for obesity targets spanning multiple biological mechanisms
• In this presentation, we will share clinical and preclinical data demonstrating potential for best-in-class RNAi payloads, differentiated pharmaceutical properties and therapeutic proof-of-concept

• Unveil groundbreaking techniques for targeted pulmonary delivery of mRNA therapeutics, showcasing innovative formulation methods and administration strategies designed to overcome the unique challenges of the respiratory system in Cystic Fibrosis (CF) patients, potentially transforming treatment efficacy and patient outcomes

• Highlight cutting-edge progress in mRNA design, stability enhancement, and lung-specific targeting approaches, illustrating how these advancements address key hurdles in CF treatment, including mucus penetration, cellular uptake, and sustained therapeutic effect, while considering the potential for tailored medicine solutions in this genetic disorder

• Explore the innovative Transport Vehicle Technology that leverages the transferrin receptor pathway for enhanced delivery of therapeutic enzymes and oligonucleotides across the blood-brain barrier, potentially revolutionizing treatments for neurological disorders.

• Examine the mechanistic insights, preclinical evidence, and clinical potential of this targeted delivery approach, highlighting its ability to overcome traditional obstacles in CNS drug delivery and improve the efficacy of enzyme replacement therapies and oligonucleotide-based treatments for brain diseases.

• Insight into Phase 1 self-replicating mRNA therapeutic, STX-001, for treatment of solid tumours

• Exploring discovery of new circRNA-based approach to target haematological malignancies through engineered immune cell modulation

• Discover comprehensive solutions for the design, characterization, and optimization of RNA therapeutics, enhancing the development process from concept to clinical application.

• Learn about a state-of-the-art platform that offers in-depth characterization of therapeutic potency, safety, and purity, crucial factors for successful drug development in the RNA space.

• Understand the challenges posed by double-stranded RNA (dsRNA) by-products, which can decrease therapeutic stability and translation efficiency. Explore an advanced technology that identifies specific sequences and structures promoting dsRNA formation, enabling improved therapeutic design and efficacy.

• Utilizing a proprietary mRNA platform to develop an LNP-mRNA vaccine combating solid tumours

• Discussing journey into the clinic, and opportunities for future oncology indications including of the lung, liver, cervical and hematologic cancers

• Discover a revolutionary Trojan horse technology that enables intravenous delivery of gene-silencing treatments to the brain. Gain insights into how this approach achieves uniform brain distribution at significantly lower doses compared to intrathecal methods, potentially transforming the treatment of neurological disorders.

• Explore an innovative nanoparticle formation process that combines negatively-charged therapeutic cargoes with a proprietary peptide. Learn how these neutral nanoparticles interact with the blood-brain barrier to effectively deliver treatments to brain cells, opening new possibilities for targeted drug delivery.

• Uncover cutting-edge optimization techniques for nanoparticle production, including the use of commercial equipment and the impact of particle size on brain uptake. Gain valuable knowledge about the relationship between dosage, brain uptake, and protein knock-down efficacy, potentially revolutionizing treatments for conditions like Huntington's disease

• Discover innovative delivery platforms tailored for RNAi therapeutics, including novel nanoparticle formulations, conjugate technologies, and engineered carriers designed for specific tissue targeting.
• Uncover the latest strategies in overcoming biological barriers for RNAi delivery, focusing on improved cellular uptake and endosomal escape mechanisms.
• Examine the potential impact of advanced RNAi delivery systems on treating various diseases, highlighting recent successes and future prospects in precision medicine.

• Explore the shift from passive to active RNA delivery systems in precision medicine, examining nuances between delivery methods for siRNA, mRNA, and ASOs.

• Discover cutting-edge tissue targeting strategies, including lipid nanoparticles and conjugate technologies, enhancing specificity for different RNA modalities.

• Compare innovative delivery platforms like exosomes and engineered viral vectors, optimized for various RNA cargoes and therapeutic goals.

• Uncover the potential of active RNA delivery in transforming treatments for genetic disorders and cancer, discussing emerging hybrid systems for optimal targeting.