Day 2- Thursday, September 11th - EST (Eastern Time, UTC-05:00
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
- Dmitry Samarsky - Chief Technology Officer, GALCONDA THERAPEUTICS
- Fred Fleitz - Group Vice President, CMC, Arrowhead Pharmaceuticals
- Christopher O'Donnell - Global Head of Cardiovascular & Metaboli, Novartis
- Steve Hughes - CMO, Avidity Biosciences
Explore steps must the industry take to drive progress of personalised cancer mRNA vaccines and therapeutics?
Allogenic or autologous? Where is the industry placing its bets
Discuss to what extent delivery is hindering the progress of mRNA therapeutics and vaccines for cancer
Review the progress that has been made in mRNA-encoded immunotherapies
Insight into the future of mRNA therapeutics and vaccines for cancer patients, including 2026 milestones
- Anna-Rose Welch - Editorial and Community Director Advancing RNA, Life Science Connect
- Thaminda Ramanayake - CBO, CureVac
- Daniel Getts - CEO, Myeloid Therapeutics
Overview of the Cancer Support Community Massachusetts
Insight into the patient advocacy process to support clinical trials for cancer therapeutics
- Sally Werner - CEO, Cancer Support Community
- Stuart Hwang - Partner, Remiges Ventures
- Travis Hughes - Digitalis Ventures, Digitalis Ventures
- Uncover how Del-brax merges antibody precision with oligonucleotide therapeutic prowess to tackle the root genetic cause of FSHD.
- Examine the encouraging clinical outcomes observed in trials, highlighting Del-brax's capacity to transform treatment paradigms for this uncommon muscular condition.
- Contemplate the future ramifications of AOC technology for other genetic disorders and its potential to reshape personalized medicine strategies.
- Steve Hughes - CMO, Avidity Biosciences
- 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
- Stefan Lutz, Ph.D. - Senior Vice President, Research, Codexis
Positive topline 48-week data from the ALPACAR-360 Phase 2 study of zerlasiran in patients with elevated lipoprotein(a) (Lp(a)) with highly significant and sustained reductions in Lp(a) to week 48 (end of treatment period).
Zerlasiran was well tolerated.
Data support advancing zerlasiran into Phase 3.
Exploring the first-ever clinical stage circRNA therapeutic, RXRG001, for the treatment of HNC patients with radiation-induced xerostomia and hyposalivation.
- Weiyi Zhang - CEO & CSO, RiboX Therapeutics
Discussing how ML-enhanced RNA structure prediction can accelerate drug discovery and the development of RNA-based therapeutics by enabling better design and optimization of RNA molecules
Exploring a cutting-edge RNA design platform, which enables the creation of optimized RNA sequences for immunomodulation, enhancing the efficacy of next-generation mRNA vaccines and RNA-based protein therapeutics
- Gilles Besin, Ph.D. - Chief Scientific Officer, Orbital Therapeutics
GenAI-based gRNA design drives exquisite target editing and specificity to enable therapeutic application for mutation correction or disruption of pathogenic proteins
Systemic administration of RNAfix with a novel AAV5-based capsid crosses the blood-brain-barrier and demonstrates broad RNA editing in neurons across the CNS, reaching 90% in deep brain regions
Taken together, the RNAfix platform unlocks potential treatments for rare and common CNS diseases
- David Huss - CSO, Shape Therapeutics
Utilising dual use of se of CRISPR-Cas9 and CRISPR-Cas12a genome editing technologies to correct genetic mutations associated with hemoglobinopathies, including sickle cell disease and thalassemia.
A Preclinical update
- John Zuris, PhD - Associate Director, Editing Technologies, Editas Medicine
Driving RNA editing oligonucleotide product KRRO-110, from a proprietary platform OPERA to restore normal ATT protein production in patients with Alpha-1 antitrypsin deficiency.
- Todd Chappell - COO, Korro Bio
- The FORCETM 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.
- Oxana Beskrovnaya, Ph.D. - Senior Vice President, Head of Research, Dyne Therapeutics
Discuss the results of the Phase 3 study demonstrating the efficacy of vutrisiran in treating cardiomyopathy, including improvements in cardiac biomarkers and patient outcomes
Discuss the future development plans for vutrisiran, such as exploring additional indications or combination therapies, and the potential impact on the treatment of cardiomyopathy
- Satish Eraly - Senior Medical Director, Alnylam Pharmaceuticals
Reviewing Phase 1 saRNA vaccine to prevent rabies
Insight into further pre-clinical candidates utilizing saRNA therapeutic technology
- Nathaniel Wang - CEO, Replicate Bioscience
Updates on its Phase 2 clinical trials for mRNA-based therapeutic candidate for Ornithine Transcarbamylase Deficiency and Cystic Fibrosis
- Weijun Chen, PhD - Director, RNA Technologies Lead, Intellia Therapeutics
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.
- Karina Thorn - Corporate Vice President, Head of Research, Global Nucleic Acid Therapies Research, Novo Nordisk
- Michelle Hall - Associate VP - Genetic Medicine Research, Eli Lilly & Company
- Malgorzata Gonciarz - Global Head xRNA Therapeutics, Novartis
Explore the current state of RNA therapeutics in 2025, including USA legislation and opportunities in growing markets like China, while examining emerging technologies that are shaping the field.
Discuss critical steps for accelerating clinical progression of new RNA approaches, considering the shift from large-scale vaccine production to diverse, small-scale applications for cancer and rare diseases.
Evaluate strategies for US companies to progress RNA programs in growing markets such as China, highlighting successful partnership models and the potential impact of next-generation technologies on the future of the RNA space.
- Samir Ounzain - CEO, HAYA Therapeutics
- Tamar Grossman - Co-Founder, La Jolla Labs
- Renee Williams - Managing Partner, Williams Biotech Consulting, LLC