Wednesday, September 23 - EST (Eastern Time, UTC-05:00
This interactive workshop will guide biotech management teams through a practical framework for approaching valuation in strategic partnering situations, from assessing core asset attributes to understanding how negotiation dynamics and deal terms shape perspectives value. Participants will explore how scientific differentiation, development stage, risk profile, and commercial potential translates into intrinsic value assumptions, and how partnering structures, market dynamics and other external factors may influence headline value and partner alignment.
- Renee Williams - Founder and Managing Partner, Williams Biotech Consulting, LLC
- Jeff Raphael - Founder & Managing Partner, OmniScope Partners
The RNA Leaders CEO Forum is an exclusive group workshop that brings together CEO’s from the RNA therapeutics space to collaboratively discuss challenges, share insights, and brainstorm strategic solutions in a peer-to-peer setting. This year's forum will focus specifically on the strategic opportunity and challenges of moving to China for RNA drug development, covering critical topics including regulatory navigation, market access, manufacturing considerations, intellectual property protection, and partnership strategies. Participants will gain actionable insights from their peers' experiences and expand their network of trusted industry colleagues.
Explore the latest advances in targeted delivery systems for mRNA and oligonucleotide therapeutics through presentations from multiple industry leaders who will share their insights on overcoming delivery barriers and enhancing therapeutic precision. This interactive workshop brings together diverse perspectives on delivery platforms, targeting strategies, and formulation innovations.
- Haig Aghajanian - Founder, Capstan Therapeutics
This workshop-style session brings together multiple thought leaders to examine current regulatory landscapes, manufacturing complexities, and innovative approaches to overcoming development hurdles. Participate in an open Q&A dialogue that encourages knowledge sharing and collaborative problem-solving.
Title: What Breaks Between Discovery and Clinical CMC and How to Fix It Early
Abstract:
Early chemistry decisions disproportionately determine downstream CMC success - most IND delays stem from avoidable gaps in scalability, impurity understanding, and analytical readiness rather than fundamental chemistry failure. Phase-appropriate development is critical - the goal is not to perfect the process early, but to align chemistry, analytics, and control strategy with evolving regulatory expectations from discovery through clinical stages. Bridging discovery to clinical requires integration, not handoff - successful programs proactively align chemistry, process, analytical, and regulatory strategies from the outset, rather than addressing them sequentially.
Speaker: Mayurbhai Patel
- Vinita Doshi - Senior Scientist, Intellia
- Vega Shetty - Scientist, Intellia
- Mayurbhai Patel - VP of CMC and Chemistry Development, Judo Bio
- Jade Osei-Tutu - Head of RNA Leaders, Informa Connect
- Building a pharma innovation ecosystem for RNA therapeutics: implementing structured scouting frameworks, venture partnerships, and co-development agreements to complement internal R&D and rapidly advance promising RNA candidates to the clinic
- De-risking RNA programs through external expertise: accessing specialized manufacturing, analytical, and regulatory knowledge to navigate complex development challenges and achieve faster IND submissions and clinical proof-of-concept
- Malgorzata Gonciarz - Global Head xRNA Therapeutics, Novartis
- Decoding the RNA M&A landscape: key drivers behind recent mega-deals, valuation metrics for emerging modalities, and strategic rationale for Big Pharma partnerships versus full acquisitions
- Renee Williams - Founder and Managing Partner, Williams Biotech Consulting, LLC
- Leveraging best-in-class oligonucleotide chemistry and genetic insights to address obesity and cardiometabolic disorders
- The role of PN and stereochemistry in building differentiated clinical assets
- Beyond RNAi: RNA editing, bifunctional constructs, and pushing the boundaries on what is possible in RNA medicine
- Chandra Vargeese, PhD - Chief Technology Officer, Wave Life Science
RNAi innovators and manufacturers are increasingly adopting ligation-based approaches for siRNA production, driven by clear advantages in efficiency, scalability, and process simplicity relative to traditional solid-phase oligonucleotide synthesis. At the same time, the incorporation of phosphorothioate (PS) linkages—essential for therapeutic stability and efficacy—introduces diastereomeric complexity that challenges both manufacturing control and analytical characterization.
In this presentation, we highlight how advances in sequential enzymatic fragment synthesis and ligation—core capabilities of Codexis’ ECO Synthesis® Manufacturing Platform—address these limitations. By combining biocatalytic innovation, process optimization, and machine learning–guided design, this approach enables control over PS diastereomeric composition while enhancing ligation efficiency and reproducibility.
The result is a streamlined, scalable manufacturing strategy that improves downstream processing and delivers high-quality siRNA with superior consistency, purity, and performance.
Leveraging computational intelligence to transform RNA therapeutics: from target identification and molecular design to personalized treatment strategies for rare disease populations
Bridging the gap between AI innovation and clinical reality: case studies in RNA drug development, regulatory considerations, and translational pathways to patient care
- Anders Näär - Professor of Metabolic Biology, UC Berkeley
- Tamar Grossman - CEO, La Jolla Labs
- Edward Miracco, Ph.D. - Director, RNA Chemical Biology/External Technology Investments, Moderna, Inc.
- Crystal Jin - CEO, Bound Therapeutics