Day 2- Thursday, September 11thThursday, 11 September 2025 - EST (Eastern Time, UTC-05:00

The RNA Leaders ‘Women in RNA (WiRNA) Forum’ is a dedicated space to empower and connect women and allies who are shaping the future of RNA research and therapeutics.

A round table breakfast to hear and share about career, research journeys and RNA inspirations in the space.

• Discuss current regulatory hurdles and evolving guidelines impacting oligonucleotide drug development

• Explore strategies for navigating regulatory pathways and fostering collaboration between industry, patient advocacy groups and regulatory agencies

• Examine best practices for demonstrating safety, efficacy, and quality of oligonucleotide therapeutics throughout the development lifecycle

Designing del-brax, an antibody-oligonucleotide conjugate, to efficiently deliver siRNA to muscle and target DUX4, the root cause of FSHD.·

Clinical trial results demonstrating improved functional mobility and muscle strength in this rare, progressive neuromuscular disorder.

Evaluating the potential of Avidity’s platform for other genetic disorders and its potential to reshape RNA delivery.

• Discuss siRNA synthesis by assembly of multiple short, single-stranded RNA fragments into the desired double-stranded RNA duplex using ligation

• Compare various ligation approaches with a particular focus on substrate design. In combination with double-stranded RNA ligase engineering and protocol optimization, such integrated process development can increase target drug substance yield and reduce manufacturing costs

• Process performance will be demonstrated on the synthesis of a commercially approved siRNA asset

• Explore innovative RNAi-lipid conjugates targeting immune cell PD-L1 for refractory cancer treatment, focusing on pre-clinical discovery and efficacy of a novel RNAi compound.
• Delve into the compound's mechanism of action and its potential to overcome immunotherapy resistance, highlighting promising pre-clinical results.
• Unveil the strategic clinical trial design, outlining the approach for translating this cutting-edge therapy from laboratory to patient care in challenging cancer cases.

• The FORCE^TM platform enables TfR1-mediated delivery of oligonucleotides and other payloads to muscle and CNS.
• We leveraged the FORCE platform to develop DYNE-101 and DYNE-251 as potential treatments for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), respectively.
• Clinical data in both programs support the potential of the FORCE platform to deliver functional improvement in serious neuromuscular disorders.

• Vutrisiran, a subcutaneously administered RNA interference therapeutic agent that inhibits the production of hepatic transthyretin, is in development for the treatment of transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a progressive, fatal disease. HELIOS-B, the Phase 3 trial of vutrisiran in ATTR-CM, randomized 655 patients 1:1 to vutrisiran (25 mg) or placebo every 12 weeks for up to 36 months. The primary endpoint was a composite of all-cause mortality (ACM) and recurrent cardiovascular events; secondary endpoints included ACM and the change from baseline to month 30 in the distance walked in 6 minutes (6MWD, a measure of exercise capacity) and in the score on the Kansas City Cardiomyopathy Questionnaire (KCCQ-OS, a heart failure-specific patient-reported outcome). These endpoints were assessed in both the overall population and the monotherapy population (the patients who at study baseline were not receiving the TTR stabilizer, tafamidis, at the time the only approved treatment for ATTR-CM). As announced in June 2024, HELIOS-B met all primary and secondary endpoints: vutrisiran reduced the risk of the composite primary endpoint in the overall and monotherapy populations by 28% (p=0.012)and 33% (p=0.016), respectively; reduced the risk of ACM by 35% (p=0.010) and 36% (p=0.045); improved the 6MWD relative to placebo by 26 m (p<0.001)and 32 m (p<0.001); and improved KCCQ-OS relative to placebo by 5.8 points (p<0.001) and 8.7 points (p<0.001). The overall incidence of AEs was similar in vutrisiran- and placebo-treated patients, while the incidence of SAEs was lower in vutrisiran-treated patients. Collectively, these results indicate the safety and efficacy of vutrisiran in the treatment of ATTR-CM, and formed the basis for the global approval of vutrisiran in this indication, including, to date, in the US, EU, UK, Japan, and Brazil.

• Argonaute RNA's revolutionary bispecific technology simultaneously silences two target genes with a single therapeutic, achieving >90% mRNA knockdown of both targets and addressing the critical limitation of conventional therapies that improve either steatosis or dyslipidemia at the expense of the other
• The company's proprietary linker technology demonstrates exceptional safety profile with no inflammatory markers while delivering dramatic LDL-C reduction that predicts >50% decrease in major adverse cardiovascular events, positioning this therapeutic as a breakthrough solution for ASCVD and MASH/MASLD patients

• Uncover innovative partnership paradigms and strategic approaches that catalyze breakthroughs and expedite the evolution of RNA-based therapeutics, with a spotlight on emerging opportunities in the Asia-Pacific and European regions and established collaborations in North America.

• Dissect the critical components of high-impact collaborations among biotech firms, pharmaceutical giants, and key industry players within the global RNA ecosystem.

• Pinpoint promising avenues and navigate potential hurdles in forging transcontinental strategic alliances, particularly between North American companies to propel RNA research, optimize manufacturing processes, and enhance clinical translation efforts.

• Examine case studies of successful local and international partnerships that have leveraged diverse expertise, to accelerate RNA therapeutic development and market access. Highlight strategies for fostering robust, long-term collaborations in the RNA pharma landscape.

• Exploring the state of mRNA innovation: Discuss advancements in therapeutics, vaccines, and editing technologies, while addressing the impact of geopolitical factors, market trends, and emerging opportunities in the US.
• Strategies to overcome challenges: Gain insights into navigating regulatory hurdles, mitigating supply chain disruptions, and securing funding to drive successful mRNA product development.
• Case studies in mRNA technologies: Reflect on successful applications, such as personalized cancer vaccines and RNA editing, to understand their impact and potential for future innovation.