Day 1 - UTC+08:00
- Jade Osei-Tutu - Head of RNA Leaders, Informa Connect
Highlighting the strategic role of government programs in Singapore, Korea, Australia, and Japan in advancing RNA vaccine and therapeutic development, with a focus on funding, infrastructure, and fostering innovation ecosystems.
Exploring collaborative frameworks between governments, academia, and industry to enhance RNA research, streamline clinical tri
- Soon Tuck Sit - Director, BMRC,, A*STAR
- Wensheng Wei - Professor, Peking University
- Paul Mcdonald - Chief Executive Officer, RNA Australia
- Manki Song - Deputy Director General of Science, International Vaccine Institute
- Rebecca Mckenzie - Project Leader RNA Platform, Malaghan Institute
Examining the evolving regulatory frameworks for nucleic acid therapeutics across Asia-Pacific and Western regions, with a focus on harmonizing standards to support global innovation and accessibility.
Discussing challenges and opportunities in navigating diverse regulatory landscapes, including strategies for accelerating approvals and ensuring safety and efficacy in emerging RNA-based treatments.
- Jack Wong - CEO and Founder, Asia Regulatory Professionals Association (ARPA)
- Mimi Lee - Former Program Manager (ARPA-H), CEO & Founder, OPUS 132
- Yacoub Habib - CEO, Ophidion, Inc.
- Bo Ying - CEO, Abogen Biosciences
- Xiangrong Song - Co-Founder & CEO, WestGene Biopharma
- Luca Bolondi - CEO, Crane Biosciences
- RIBOFLUX™ delivers fast, scalable mRNA manufacturing to support programs from early development through clinical supply.
- With production capacities ranging from 10 mg to 250 g/day, the platform provides a flexible solution for growing mRNA demand.
- RiboPro's technology enables efficient manufacturing, reducing timelines while improving access to clinical-grade material.
- Heyi Zhang - Product Specialist, RiboPro
- Evolution of RNA Therapeutics, Multiple Mechanisms and Approved Drugs
- Unique Features of RNAi Therapeutics
- Hepatic and extra-hepatic Delivery
- Challenges and Opportunities
- Muthiah (Mano) Manoharan, PhD - Senior Vice President of Drug Innovation and Distinguished Research Scientist, Alnylam Pharmaceuticals
- This presentation will discuss a novel “Trojan horse” technology that enables the direct delivery of gene-silencing cargos, such as siRNAs and ASOs, to the brain via intravenous or subcutaneous administration, eliminating the need for intrathecal or intracranial administration.
- Therapeutic efficacy in Huntington’s disease demonstrated at a dose significantly lower than the standard intrathecal ASO dose (less than 1,000 the dose used intrathecally) or ASOs delivered via other shuttles like transferrin or CD98 shuttles. Over 95% halting of disease progression in chronic preclinical studies achieved.
- In another neurodegenerative disease target, equivalent protein reduction achieved following a single IV dose of an ASO complexed to our Trojan horse shuttle at a dose that is only 1/3,000 relative to that given via the ICV route, a true testimony to the power of targeted brain-centric shuttles.
- Confirmed safety and consistent brain delivery in repeat-dose non-human primate studies.
- This non-invasive delivery approach offers a significant clinical advantage over invasive modalities or other BBB shuttles and could be a powerful enabler for any siRNA/ASO or peptide/protein programs targeting CNS indications.
- Yacoub Habib - CEO, Ophidion, Inc.
We have established LuxiAP™, a platform built on three novel bridged nucleic acids—AmNA, scpBNA and GuNA—and one DNA modification, 5′-CP DNA, to advance antisense therapeutics toward clinical use. In animal studies, LuxiAP™ has shown the potential to reduce neurotoxicity and immune activation while improving potency and duration of action. The platform has also generated extensive background data that support collaborations with global pharmaceutical partners, including Servier, Takeda and other undisclosed companies, demonstrating external validation of the technology. We are now applying LuxiAP™ not only in partner programs but also in our own discovery pipeline. In ophthalmology, where ASO development has historically been limited by inflammatory responses, we have established technologies to improve tolerability and open new therapeutic opportunities. This presentation will highlight key animal validation data for LuxiAP™ and provide an update on our in-house ASO program for glaucoma neuroprotection.
- Hideaki Sato - President and CEO, Luxna Biotech
- Oxana Beskrovnaya, Ph.D. - Chief Innovation Officer, Dyne Therapeutics
- Developing a scalable cell-derived nanoparticle platform for RNA delivery to the brain
- In preclinical animal studies, the platform showed functional RNA delivery to over 70% of neurons following a single IV injection
- Luca Bolondi - CEO, Crane Biosciences
Resistant cancers survive by deploying RNA strategies that regulate translation and stability, thereby modulating tumor persistence and immune signaling. By decoding these adaptive RNA programs, we can harness or modulate these regulatory circuits to control tumor adaptation and reduce the emergence of new resistance mechanisms. Leveraging multi-omic profiling and AI-guided modeling, this approach transforms stress-responsive RNA programs into programmable RNA therapeutics, including inducible translation circuits and targeted RNA modulators. Through this framework, RNA therapeutics can dynamically respond to tumor stress—enhancing resilience, precision, and adaptability—while exploiting the same code-control mechanisms tumors use to survive.
- Adaptive RNA Programs: Resistant tumors rewire coding and noncoding RNA programs to regulate translation and stability, enabling therapeutic persistence and immune evasion. These adaptive circuits can be therapeutically harnessed or modulated to control tumor persistence.
- Programmable RNA Therapeutics: By integrating multi-omic data and AI-guided modeling, stress-responsive translation circuits can be designed as programmable RNA therapeutics that dynamically respond to tumor adaptation, control protein output, and reduce the emergence of new resistance mechanisms.
- Shobha Vasudevan - Senior Group Leader, Senior Principal Scientist II, A*STAR
- Zee-Fen Chang - Chair Professor, Institute of Molecular Medicine, Director, Center of Nucleic Acid Medicines, College of Medicine, National Taiwan University
