Friday, September 25Friday, 25 September 2026 - EST (Eastern Time, UTC-05:00

• TTX-MC138 is an antisense oligonucleotide therapeutic targeting miR-10b, a driver of metastatic disease, with preclinical studies showing successful delivery to metastatic lesions and complete regression without recurrence

• Phase 0 microdose study (NCT05908773) demonstrated drug accumulation in metastatic lesions, stability in circulation with an 18.7-hour half-life, and robust pharmacodynamic activity

• Phase 1a dose-escalation study (NCT06260774) enrolled 16 patients across four dose levels (0.8-4.8 mg/kg), met primary safety endpoints, and established a recommended Phase 2 dose with median treatment duration of four months

• 44% of patients achieved stable disease lasting ≥4 months, with one thyroid cancer patient showing reversal of rising thyroglobulin levels to undetectable during treatment

This presentation introduces Chemoenzymatic Synthesis as a next-generation solution for oligonucleotide synthesis. Unlike traditional solid-phase synthesis, Chemoenzymatic Synthesis assembles short, chemically prepared oligonucleotide fragments using enzymatic ligation under mild aqueous conditions, reducing impurities and improving yield, scalability, and cost efficiency. By optimizing fragment design, ligation parameters, and purification strategies, Chemoenzymatic Synthesis enables high-purity oligonucleotide manufacturing with enhanced reproducibility. 

• PGN-EDODM1 represents an advanced peptide therapeutic engineered with enhanced properties for treating Duchenne muscular dystrophy type 1 (DMD1), incorporating optimized cell-penetrating sequences and improved stability that enable efficient delivery to skeletal and cardiac muscle tissues, with preclinical studies demonstrating superior dystrophin restoration, functional muscle improvement in animal models, favorable pharmacokinetic profiles, and a well-characterized safety profile that supports progression into clinical development where the therapeutic aims to address the underlying pathophysiology of DMD1 and provide meaningful clinical benefit to patients suffering from this progressive and life-threatening neuromuscular disorder.

• An overview of advancing SGB-7342 into Phase 1 clinical trials for the treatment of overweight and obesity, focusing on the RNAi mechanism, study design, patient selection, dose escalation strategy, and key endpoints for assessing safety and pharmacological activity.

This presentation explores next-generation siRNA manufacturing strategies that address growing industry demand for scalable, cost-effective production. By integrating enzymatic ligation and thermostable enzyme technologies, we demonstrate pathways to improve efficiency, reduce cost of goods, and enable flexible manufacturing at scale.

• Transition from conventional SPOS to hybrid enzymatic ligation for improved yield, purity, and sustainability

• Design and screening of thermostable ligases to enable high-temperature, high-efficiency ligation

• Impact of elevated temperature on impurity control, fragment selectivity, and ligation performance

• Scalable manufacturing strategies (C-to-C, C-to-P, P-to-P) to optimize cost, robustness, and process efficiency

• From laboratory breakthrough to patient impact: evaluating clinical progress in genome and RNA editing across therapeutic areas, including recent trial data, efficacy outcomes, and lessons learned from first-generation editing therapies
• Overcoming translational barriers in precision editing: innovations in delivery systems, tissue-specific targeting, editing efficiency optimization, and navigating the evolving regulatory landscape for transformative genetic medicines

• Explore the cutting-edge frontiers of RNA drug discovery as industry leaders discuss breakthrough technologies and strategies shaping the next generation of genetic medicines. This session will examine emerging areas of innovation, including in vivo mRNA CAR-T approaches, circRNAs, RNA editing and antisense oligonucleotide (ASO) development for CNS disorders.
• Gain insights into overcoming key challenges in stability, immunogenicity, and delivery while discovering how advances in chemical modifications, formulation science, and AI-driven design are unlocking new therapeutic possibilities across oncology, rare diseases, metabolic disorders, and beyond.

• Explore funding strategies and partnership models accelerating RNA therapeutics development, including mRNA vaccines, siRNA therapies, and antisense oligonucleotides, with insights on navigating investment landscapes and collaborative frameworks

• Examine successful pharma partnerships driving genetic medicine innovation, from early-stage biotech collaborations to large-scale alliances between companies like Moderna-Merck, BioNTech-Pfizer, and Alnylam-Regeneron that are transforming therapeutic development and commercialization