Marc has over 20 years' experience in discovery and development of genetic medicines, including RNAi and gene therapy. He started his career at Merck and Co. Inc., taking on roles of increasing responsibility in the early days of the field. More recently, Marc served as SVP, Discovery Research at Dicerna Pharmaceuticals (a Novo Nordisk Company) and as CSO of Carbon Biosciences. Marc is currently CTO & Head of US Operations at SanegeneBio, working on novel RNAi delivery strategies and target biology. His therapeutic area experience spans diverse indication spaces including oncology, antiviral and metabolic diseases. Marc has a Ph.D. in Biochemistry from Thomas Jefferson University.

Vikram Agarwal completed his Ph.D. in Dr. David Bartel's lab at the Massachusetts Institute of Technology, where he utilized computational approaches to study the principles of microRNA targeting. He completed his post-doctoral research in Dr. Jay Shendure's laboratory at the University of Washington, where he applied deep learning methods and massively parallel reporter assays to investigate the mechanisms of transcriptional gene regulation. Transitioning into industry, Vikram worked at Calico Life Sciences LLC, an Alphabet company whose mission is to better understand the mechanisms underlying human aging. There he applied deep learning methods with Dr. David Kelley to better understand transcriptional and post-transcriptional gene regulation as well as its link to human genetics. He is currently the Head of mRNA Platform Design Data Science at the mRNA Center of Excellence at Sanofi, where his team is applying machine learning and deep learning methods towards the design of enhanced mRNA therapeutics.

Sudhir Agrawal, D. Phil., FRSC, is the founder and president of ARNAY Sciences. He also serves as an Affiliate Professor in the Department of Medicine at UMass Chan Medical School. For more than three decades, his research has focused on RNA therapeutics, including antisense and immunotherapy. He has specialized in the chemical engineering of oligonucleotides to improve the drug-like properties of RNA therapeutics. He is the inventor of the gapmer antisense, which is widely used in the approval and development of antisense drugs. In recent years, he has introduced novel cyclic-structured oligonucleotides for use in RNA therapeutics. He has authored over 300 research papers, chapters, and reviews, along with more than 450 patents and applications worldwide, and has edited four books. He currently serves on the scientific advisory boards of several biotechnology companies. He earned his D.Phil. in Chemistry from Allahabad University in India and conducted postdoctoral research with Mike Gait at the MRC Laboratory of Molecular Biology in Cambridge, UK. His research in the antisense field began in Paul Zamecnik’s laboratory at the Worcester Foundation for Experimental Biology in Shrewsbury, MA, now part of UMass Chan Medical School. He co-founded Idera Pharmaceuticals and held various roles there until 2017. In 2022, the Oligonucleotide Therapeutic Society presented him with the Lifetime Achievement Award.

Nadim Akhtar, PhD, is a Senior Principal Scientist in the New Modalities and Parenteral Development Department in Pharmaceutical Technology and Development function at AstraZeneca. Nadim has been working for AstraZeneca since 2007. During this time Nadim has worked on both early- and late-stage portfolio supporting development of small molecules and new modalities i.e. oligonucleotides, peptides, polymeric nanoparticles, dendrimers, and mRNA products. Nadim is currently responsible for developing characterisation and control strategies for New Modalities.

Daniel G. Anderson is a Professor in the Department of Chemical Engineering, the Institute for Medical Engineering and Science, the Koch Institute for Integrative Cancer Research, and the Harvard-MIT Division of Health Science and Technology at MIT. The research done in Prof. Anderson’s laboratory is focused on developing new materials for medicine. His work has led to advances in a range of areas, including medical devices, cell therapy, drug delivery, gene therapy and material science. Prof. Anderson received a B.A. in mathematics and biology from the University of California at Santa Cruz and a Ph.D. in molecular genetics from the University of California at Davis. His work has resulted in the publication of over 500 papers, patents and patent applications. These advances have led products that have been commercialized or are in clinical development, as well as to the foundation of companies in the pharmaceutical, biotechnology, and consumer products space. Dr. Anderson is a founder of Living Proof, Olivo Labs, Crispr Therapeutics (CRSP), Sigilon Therapeutics, Verseau Therapeutics, oRNA Therapeutics, VasoRx, and Soufflé Therapeutics.

Ben is a synthetic organic chemist with a PhD and postdoctoral training in asymmetric synthesis. He spent 22 years in chemical development at GSK, including seven years on small-molecule programs and fifteen years leading mid- to late-phase development of therapeutic oligonucleotides. His work contributed to two marketed oligo medicines, and he has a strong interest in emerging technologies and sustainability in oligo manufacturing. Since 2026, Ben has worked as an independent oligonucleotide chemistry consultant.

Rob Blakemore, PhD, is the Director of Discovery Research at CREATE Medicines, where he contributes to the development of CREATE’s mRNA-based in vivo immune cell programming therapeutics. Dr. Blakemore has 18 years of R&D experience spanning product, platform, and assay development. Prior to CREATE, Rob served as the Associate Director of RNA Sciences at Orbital Therapeutics. Earlier in his career he established the circRNA team at ElevateBio. Dr. Blakemore received his Ph.D. in Molecular Microbiology from Tufts University.

I am Emeritus Professor of Nucleic Acid Chemistry at Oxford University. I work on modifications of nucleic acids for use in biology and medicine and I am co-founder of several nucleic acid-based companies, most recently Assemblon Ltd. My current interests are in chemical modification of mRNA and developing new DNA backbones for therapeutic oligonucleotides. I have published around 500 research papers and patents. Awards include the Royal Society of Chemistry (RSC) prizes for Nucleic Acid Chemistry and Interdisciplinary Research, the RSC Khorana Prize for work at the chemistry and life science interface, Chemistry World entrepreneur of the year and BBSRC Innovator of the Year.

Tracey Burr is Executive Director of CMC Regulatory Affairs at Ionis Pharmaceuticals and is responsible for Ionis’ early- and late-phase, as well as commercial compounds. She has 20 years of drug development and global CMC regulatory experience and has spent her career working at small to mid-size pharmaceutical and biotechnology companies. Tracey received a PhD in Materials Science and Engineering from MIT, M.Sc. in Chemistry from Caltech, and BA in Chemistry from Wellesley College.

James J. Carroll is Founder, President and CEO of RNA NanoBiotics, Inc., where he leads the development of programmable RNA nanostructures for targeted delivery of therapeutic payloads. His career has spanned many years in nucleic acid therapeutics, targeted drug delivery and translational oncology, with work involving targeted radiation therapy, RNA-based therapeutics and multifunctional delivery systems. At RNA NanoBiotics, he is advancing phi29 pRNA three-way and four-way junction nanoparticles designed to combine targeting ligands, small-molecule drugs and nucleic-acid therapeutics within precisely assembled structures. Carroll is a former President of Wharton Alumni Angels and has completed executive programs at Wharton, MIT Sloan and Harvard. His current interests include extrahepatic RNA delivery, combination therapeutics and improving therapeutic index through selective, programmable drug delivery.

Dr. Corey graduated with a BA from Harvard in 1985 and a PhD from UC Berkeley under the direction of Dr. Peter Schultz in 1990. He has had his own laboratory at UT Southwestern since 1992 and is currently the Rusty Kelley Professor of Biomedical Science in the Department of Pharmacology.

Cecilia Cotta-Ramusino has spent 25 years in academia and industry. She has made pioneering discoveries in the field of DNA damage, functional genomics, and gene editing. She also has extensive experience in company building and entrepreneurship. Cecilia was one of the first scientists at Editas, the first CRISPR-based therapeutic company, where she helped to define and shape the vision of the Editas platform. After Editas, Cecilia moved into the interface between biology and AI, joining Insitro as its first employee, where she served as Head of Functional Genomics. Since 2019, Cecilia has been in the leadership team as SVP -Head of Platform and Digital Systems at Tessera Therapeutics.

Dr. Hagen Cramer is a renowned expert in all chemistry-related aspects of oligonucleotide therapeutic development. He has been working in the biotech industry for over 20 years spanning discovery, development, and manufacturing of oligonucleotide-based therapeutics. Early in his career, he worked with Gemini Technologies and Ridgway Biosystems on the discovery of 2-5A antisense for the treatment of a variety of cancers and viral diseases. In 2005, he joined Girindus, which was later acquired by Nitto Denko Avecia, where he headed the process development department, eventually becoming Director of Operations. More recently, he joined Wave Life Sciences where he was responsible for securing drug substance and drug product for its clinical pipeline of stereodefined oligonucleotide therapeutics. In 2020, he joined QurAlis as Chief Technology Officer supporting all programs targeting diseases affecting the CNS.

Rakesh Dixit is an accomplished executive, inventor, and scientist with over 35 years of success with top biotechnology and pharmaceutical companies, including Merck, Johnson & Johnson, and Medimmune - AstraZeneca. Currently, he is President and CSO of Regio Biosciences and Bionavigen, LLC. He is a Board Member of Regio Biosciences and a key member of multiple scientific advisory boards. Rakesh is also a chief adviser and consultant for more than 20 companies worldwide. His biopharmaceutical peers selected Rakesh as one of the 100 Most Inspiring People in the Pharmaceutical Industry by PharmaVOICE in 2015. Rakesh received the Most Prestigious Award of Long-Standing Contribution to ADCs by World ADC (Hanson-Wade), 2020. From 2006 to 2019, Rakesh was a Global Vice President of the Biologics R&D at Medimmune - AstraZeneca. Rakesh has unique expertise in developing biologics (e.g., monoclonal antibodies, bispecific biologics, antibody-drug conjugates, fusion proteins, peptides, gene and cell therapies, etc.) and small-molecule biopharmaceuticals. His areas of expertise include discovery, early and late preclinical development, safety assessment, DMPK, and translational sciences. Dr. Dixit conducted extensive graduate and post-graduate training in Pharmacology/Toxicology–Biochemistry with both Indian and USA institutions (e.g., Case Western Reserve University, Medical College of Ohio, University of Nebraska) and is a Diplomate and Board Certified in Toxicology from the American Board of Toxicology, Inc. since 1992.

As the Founding Chief Technology Officer, at Soufflé Therapeutics, Vadim leads research, discovery and development to translate breakthroughs into medicines for patients. Bringing more than 20 years of experience, Vadim has led multiple platform and biology teams focusing on nucleic acids, small molecules, and radiopharmaceuticals which have contributed to numerous development and clinical candidates. He previously served as Global Head of RNA and Targeted Therapeutics at Johnson and Johnson (J&J), with worldwide platform responsibility for RNA medicines, protein conjugates, ADCs and the targeted nanotherapies discovery portfolio. Prior to this, Vadim led an internal venture research team at J&J that established a universal protein targeting platform, contributing to the creation of Poseida, Fusion, and Aro Biotherapeutics. Vadim began his career at Merck, where he held roles of increasing responsibility, eventually leading a team focused on siRNA conjugate research.

Elisabeth is Chief Scientific Officer at Tevard Biosciences, joining the leadership team in 2025 to spearhead the company’s pioneering tRNA-based gene therapies for rare diseases with high unmet need. With over 25 years in the biopharmaceutical industry, she has directed and managed drug discovery and development efforts yielding 11 IND filings, four Phase I/II trial candidates, and one Phase III candidate. Prior to Tevard, Elisabeth held senior R&D leadership roles at Tactile Therapeutics, Alterome Therapeutics, Aravive, and Kinnate Biopharma, where she directed multidisciplinary teams and advanced programs from discovery to clinical-stage development across neurology, oncology, and rare disease. Elisabeth’s commitment to the ethical development of effective and accessible medicines is her key focus in life. In addition to her professional work, Dr. Gardiner acts as a patient advocate in the rare disease and oncology space. Elisabeth earned her PhD from the University of Wisconsin-Madison and holds a BS and an MS from Texas A&M University.

Dr. Giangrande was until recently at Eleven Therapeutics in Cambridge, MA and an Adjunct Professor of Internal Medicine at the University of Iowa. She obtained her Bachelor of Science degree in Biochemistry at Wheaton College, Norton, MA in 1994 and her PhD in Pharmacology and Cancer Biology at Duke University, Durham, NC in 1999. Her career has been dedicated to the investigation and development of RNA-based therapeutic approaches (including RNA aptamers, siRNAs, mRNA, RNA editing) for cancer, cardiovascular disease, rare diseases, and critical illness. As a consequence, Dr. Giangrande developed a deep understanding of these technologies, diseases, and associated disease mechanisms. She is an internationally recognized expert in oligonucleotide therapeutics and delivery. Towards this end, she was the first to demonstrate that RNA ligands (aptamers) can be used to deliver therapeutic siRNAs to target cells. Her 70+ publications and 9+ patents are a testament to this body of work and her commitment to the development of novel RNA-based therapies for many diseases.

Trained synthetic organic chemist with >20 years of experience in synthesis of drug substances, intermediates in CROs and in innovative Biotechs. OVer 12 years of experience in clinical and late phase product development, and CMC. Currently overseeing development and manufacturing of PMOs and related drug substances at Entrada therapeutics.

Sushma Gurumurthy, PhD is a distinguished scientist with over 15 years of experience in oncology research and mRNA therapeutics. Since joining Moderna in 2015, Sushma has led the preclinical discovery and development of mRNA therapeutics for application in oncology. Prior to Moderna, she led different oncology early-stage discovery programs at the Belfer Institute in Dana Farber Cancer Institute. She received her PhD from the University of Kentucky and completed her postdoctoral training at the Massachusetts General Hospital Cancer Center and Harvard Medical School.

Dr. Michelle Lynn Hall is a Co-Founder and General Partner at Entrée Bio, an early-stage biotechnology venture capital fund where she invests in companies translating deep biological insights into scalable therapeutic impact. She brings extensive operator experience to venture capital. She has led computational and experimental teams across genetic medicines and drug discovery, spanning diverse modalities such as small molecules, biologics, oligonucleotides, mRNA, gene editing, and non-viral delivery during her tenure at Schrödinger, Moderna, and Eli Lilly. Her operational background directly shapes her investment approach. She targets platforms and programs grounded in biological mechanism, supported by rigorous data, and engineered to address real-world translational and development constraints. She prioritizes probability shifts, portfolio optionality, and building scalable biotechs that can expand beyond a single candidate asset. As a hands-on partner to founders, especially in early stage formation, Dr. Hall collaborates closely on scientific strategy, clinical development planning, and articulating the narrative connecting early discovery to patient impact.

Alan Horsager, PhD, is Co-Founder and Managing Partner of Concept Bio, a biotech venture studio dedicated to transforming early- and mid-stage therapeutic discoveries into development-ready companies. He has more than 15 years of experience founding and leading innovative life science ventures across gene therapy, molecular diagnostics, and RNA therapeutics. Alan is also Co-Founder and CEO of Twin Peaks Bio, an immuno-oncology company advancing bifunctional oligonucleotides that simultaneously activate innate immunity and inhibit oncogenic signaling in solid tumors. Previously, he served as President and CEO of Duet BioTherapeutics, which pioneered the CpG-STAT3 inhibitor platform, and as CEO of Episona, an epigenetics company developing diagnostics for male infertility. Earlier in his career, Alan’s translational research helped lay the groundwork for optogenetic gene therapy approaches for restoring vision in retinitis pigmentosa. He earned his PhD in Neuroscience from the University of Southern California and has since dedicated his career to bridging cutting-edge academic innovation with practical therapeutic development.

Dr. Xiao Shelley Hu is a seasoned pharmaceutical scientist with over 20 years of experience driving innovation across multiple therapeutic areas. She earned her Ph.D. and M.S. in Pharmaceutics from The Ohio State University, an M.S. in Environmental Chemistry from the Chinese Academy of Sciences, and a B.S. in Pharmacy from Peking University. As the President and Founder of Translational Consulting LLC, Dr. Hu provides specialized expertise in DMPK, clinical pharmacology, pharmacometrics, and translational strategies to pharmaceutical and biotech companies. Her leadership experience includes serving as Vice President and Head of DMPK and Clinical Pharmacology at Wave Life Sciences, where she spearheaded regulatory filings and advanced global programs involving antisense oligonucleotides, siRNA, and A-to-I editors. Previously, at Akebia Therapeutics, she led Clinical Pharmacology and Bioanalytical Science Department, overseeing registrational studies leading to the approval for VAFSEO. During her tenure at Biogen, she played a pivotal role in the approval of PLEGRIDY and contributed to TECFIDERA world-wide approval. A published author and sought-after speaker, Dr. Hu is committed to leveraging her deep expertise to champion data-driven approaches and shape the future of personalized medicine.

Frank has served as Chief Scientific Officer (CSO) of Secarna since the company's founding in 2015. He brings more than 20 years of experience in the discovery and development of oligonucleotide-based therapeutics across a range of disease areas, with particular expertise in oncology and metabolic disorders. In his current role, he leads the development of Secarna's oligonucleotide platform, focusing on diverse therapeutic modalities and delivery technologies. Prior to joining Secarna, Frank held scientific leadership positions at Antisense Pharma/Isarna Therapeutics and The Genetics Company. He earned a PhD in Biochemistry from the University of Zurich, Switzerland.

Dr. Jaspreet Khurana is an RNA molecular biologist by training who is passionate about transforming innovative platforms into meaningful therapies for patients. Following a PhD in Biomedical Sciences from UMass medical school, Worcester, he joined Princeton and Columbia University for a postdoc where he utilized functional genomics to study genomic rearrangements. With 13+ years of post-PhD expertise in RNA molecular biology, he has proven experience leading cross-functional teams across early stages of drug development. Currently he is focused on mRNA platform development for immunotherapy programs at Strand Therapeutics.

Anastasia Khvorova, PhD, is The Remondi Family Chair in Biomedical Research and Professor in the RNA Therapeutics Institute (RTI) and Program in Molecular Medicine at the University of Massachusetts Medical School (UMMS). She has more than twenty years of experience developing oligonucleotide technology and therapeutics. Her lab brings together hardcore organic and oligonucleotide chemists, RNA biologists, and pharmacologists to develop novel approaches and solutions to understanding natural and therapeutic RNA trafficking and delivery. She established the RTI’s Nucleic Acid Chemistry Center, which provides expertise in RNA chemistry to labs within and outside UMMS, and is the only non-profit center in North America capable of synthesizing complex RNAs at scales necessary to support both in vitro and in vivo studies. Dr Khvorova joined UMMS after several years in industry, during which she served as Chief Scientific Officer at lead biotech companies (Dharmacon, ThermoFisher; RXi Pharmaceuticals) and co-founded several startups. She is At-Large Director and Scientific & Research Council Chair of the American Society of Gene & Cell Therapy and served for several years as Director of the Oligonucleotide Therapeutics Society. Dr Khvorova is named as inventor on more than 150 patents and 200 patent applications, and she has authored more than 80 peer-reviewed publications, including seminal articles in Cell, Nature, and Nature Biotechnology (citation index exceeding 2000 per article) defining the field of RNAi drug design and development. Dr Khvorova is principal investigator on four major National Institutes of Health grants.

Brooke Koshel currently serves as Director of Process Development at Wave Life Sciences where she leads a team of scientists focused on characterizing stereopure oligonucleotides. Prior to joining Wave, Brooke was a Principal Scientist at Waters Corporation, where responsibilities included fostering collaborations with academic and industry partners, hosting training sessions and workshops on analytical method development, and evaluating new technology, software, and consumables. During this time, she authored and contributed to numerous technical communications. Brooke holds a PhD in Analytical Chemistry from Purdue University where she studied novel separation techniques for early disease detection.

Art Krieg, MD, is currently an adjunct Professor in the UMass Chan Medical School RNA Therapeutics Institute and serves on the scientific advisory boards of several companies developing oligonucleotide therapeutics. Art founded Zola Therapeutics in 2023, and serves as CEO with the goal of developing a new generation of TLR7/8/9 oligonucleotide agonists for cancer immunotherapy, and small molecule antagonists for the treatment of SLE and other autoimmune diseases. Previously, Art founded Checkmate Pharmaceuticals, until its acquisition by Regeneron in 2022. Previously, Art was CSO at Sarepta until July 2014; co-founder and CEO at RaNA Therapeutics from 2011 to 2013; CSO of Pfizer’s Oligonucleotide Therapeutics Unit from 2008 to 2011; co-founder, CSO of Coley Pharmaceutical Group from 1997 to 2008, and Professor, University of Iowa College of Medicine Division of Rheumatology from 1991 to 2001. Art co-founded the first antisense journal, Nucleic Acid Therapeutics, which he edited for 16 years, and the Oligonucleotide Therapeutics Society, for which he recently served as President. He has published more than 250 scientific papers, and is an inventor on >50 issued US patents covering oligonucleotide technologies.

Neil is a biopharma executive, co-founder, and board member with deep experience in organizational leadership and company building. He started his career at the Novartis Institutes of BioMedical Research (Cambridge, MA), where he established and co-led the effort to target RNA biology with small molecules. Neil then served as the Director of RNA Biology at Arrakis Therapeutics, where he led the platform-building efforts that were the cornerstone of the company's Series B financing and pharma partnerships with Roche and Amgen. Neil was VP, Head of Platform Development at Alltrna, where he co-invented a novel oligonucleotide modality based on engineered tRNAs as a universal medicine for premature termination codon mutations across thousands of human diseases. His company-building efforts resulted in a Series A financing from Flagship Pioneering and set the stage for a Series B raise to advance the organization's first drug candidates to the clinic. Neil served as CSO at Ananke Therapeutics and subsequently co-founded Verto Therapeutics to discover and develop selective mRNA translation modulators for genetically validated targets that are traditionally difficult to drug using conventional approaches. He is currently the Therapeutics Division Lead at General Inception, a venture studio that partners with technical founders to build world-class companies and deliver innovative medicines to patients in need. Neil earned a BS in Biology from James Madison University and a PhD in Molecular & Cellular Physiology from Penn State University. He was an American Cancer Society Postdoctoral Fellow in the Department of Cell Biology at Harvard Medical School.

Sarah is Senior Director of Toxicology at Wave Life Sciences. Prior to joining Wave, Sarah was Senior Director of Toxicology at PepGen Inc. focusing on peptide-conjugated oligonucleotides and Toxicologist at Biogen where she worked on several modalities including small molecules and antisense oligonucleotides. She did her postdoctoral training at AstraZeneca and then joined the company as a Discovery Safety Scientist. She holds a PhD in Pharmacology and Toxicology from University of Arizona and is a Diplomate of the American Board of Toxicology.

Aaron Larsen is Chief Executive Officer at Quill Therapeutics, where he is developing novel mRNA/LNP technologies for new medicines. Previously he was CSO at Terrain Bioscience where he led RNA delivery programs from discovery through IND-enabling studies and clinical translation across globally distributed teams. Over 14+ years in RNA medicines, he has built integrated computational and experimental platforms for mRNA and LNP design at Moderna, Beam Therapeutics, Resilience, Terrain — work that contributed to an approved COVID-19 mRNA vaccine and personalized cancer vaccines, and to a $200M technology transfer establishing a national cGMP vaccine capability in APAC. He holds a PhD in chemistry from McGill and completed postdoctoral work with Jack Szostak at Harvard.

Dr. Leberer received his PhD in Biology at the University of Konstanz, Germany (1986), and conducted post-doctoral training in molecular biology at the Banting and Best Institute of the University of Toronto, Canada. He then obtained the Habilitation for Professor of Biochemistry at the University of Konstanz, Germany (1992). From 1989-1998, he served as Senior Research Officer in genetics and genomics at the Biotechnology Research Institute, National Research Council of Canada, Montreal. He was also Adjunct Professor at McGill University, Montreal. Since joining the pharmaceutical industry in 1998, Dr. Leberer carried out various managing roles in Hoechst Marion Roussel, Aventis and Sanofi, including responsibilities in functional genomics, biological sciences, alliance management and external innovation for oligonucleotide-based therapeutics. In addition, from 2012-2018, he has been the Scientific Managing Director of the Innovative Medicines Initiative COMPACT Consortium on the delivery of biopharmaceuticals across biological barriers and cellular membranes, Brussels. Since March 2021, Dr. Leberer is Senior Life Sciences Consultant at ELBIOCON (www.elbiocon.com). He serves as a member in Scientific Advisory Boards of several biotechnology companies, and he has been the Head of the Supervisory Board of BioM, Munich. His research has focused on the molecular mechanisms of signal transduction and the role of signalling molecules in human diseases. He is the co-discoverer of the p21 activated protein kinase (PAK) family of cell signalling proteins and of novel virulence-inducing genes in pathogenic fungi. He is co-author of more than 60 publications in prestigious peer-reviewed journals including Nature and Science.

Comprehensive experience in biotech/biopharma, covering the entire product development process, from Discovery to product commercialization. Supported 3 commercial products and 3 launches of brand new anticancer monoclonal antibody products. Strong background in protein characterization by mass spectrometry. Developed serum stability by mass spec for ADC and Ab-siRNA characterization. Co-invented a revolutionary chain paring platform for bi-specific antibodies with outstanding paring efficiency. Designed de novo 4-helix bundle proteins and produced a super stable protein that was quoted in the 2024 Nobel Prize for Chemistry.

Dr. Liu has over 8 years of experience in oligonucleotide therapeutics and more than 10 years of industrial experience in discovery and pre-clinical research on cardiometabolic diseases. Dr. Liu joined Wave Life Sciences in 2018 and now services as a senior principal scientist. His current focus includes siRNA platform development, and research and pre-clinical development of siRNA therapeutics for metabolic diseases, i.e. obesity, type 2 diabetes, MAFLD/MASH, and dyslipidemia. Dr. Liu received his Ph.D. from the University of Pennsylvania, and prior to joining Wave, he had post-doctoral experience at the University of Pennsylvania and Pfizer.

Dr. Ajit Magadum, PhD, is a Assistant Professor, Center for Regenerative Medicine, Department of Internal Medicine, Heart Institute, University of South Florida.. He received his PhD from the Max Planck Institute for Heart and Lung Research, Germany. During his postdoctoral tenure at Mount Sinai in New York, Ajit delved into the innovative realm of modified mRNA (modRNA) as a potent gene therapy tool for combating cardiovascular and metabolic diseases (CVMD). He made significant strides in designing modRNA delivery systems tailored for the cardiovascular system, employing various carriers, to ensure robust and enduring modRNA expression within cardiac tissues. Ajit's groundbreaking work unveiled a treasure trove of novel genes (6 targets), delivered as modRNA to the heart, inducing cardiomyocyte proliferation and cardiac regeneration, inhibiting cardiac hypertrophy and fibrosis offering promising avenues for treating CVMD and fibrotic conditions. Ajit is celebrated for pioneering the development of cell-specific mRNA delivery platforms known as SMARTs (Specific Modified mRNA Translation System) in the context of CVD in 2016. This innovation allowed for the precise targeting of modRNA expression exclusively in cardiomyocytes or non-cardiomyocytes within the heart, opening up new horizons for cell-specific mRNA therapeutics in the realm of CVMD. He has published over 20 papers, along with the successful filing of 3 patents, which have been licensed and sublicensed to leading biotechnology companies. He won the Outstanding Research Innovation Award from Mount Sinai Hospital, New York, in 2017 for his contributions to mRNA therapeutics development for CVMD. Ajit received the esteemed ISHR-NAS Young Investigator Award (YICA, runner-up) in 2022 and the prestigious Melvin L. Marcus Early Career Investigator Award from the American Heart Association (AHA) in 2022. At present, Ajit's research endeavors continue to revolve around leveraging modRNA and cell-specific modRNA as innovative therapeutic modalities to target CVMD.

Muthiah Manoharan, PhD. Dr. Muthiah (Mano) Manoharan serves as a Senior Vice President of Drug Innovation, a Scientific Advisory Board Member, and a Distinguished Research Scientist at Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA. In 2003, he was the first chemist hired at Alnylam. He and his team pioneered the discovery and development of the chemical modifications, GalNAc conjugation chemistry, and LNP delivery platform that make RNA interference-based human therapeutics possible. This work led to the approval of four RNAi therapeutics: ONPATTRO® (patisiran, 2018), GIVLAARI® (givosiran, 2019), OXLUMO® (lumasiran, 2020) and Lequio® (inclisiran, 2020). Dr. Manoharan has had a distinguished career as a world-leading chemist in the field of oligonucleotides. He is an author of more than 225 publications (nearly 50,000 Google Scholar citations with an h-index of 101 and an i10-index of 399) and over 400 abstracts, as well as an inventor of over 250 issued U.S. patents. Prior to Alnylam, Dr. Manoharan worked in the field of antisense oligonucleotide therapeutics at Ionis (formerly Isis) Pharmaceuticals and LifeCodes Corporation (1988-1990) . Dr. Manoharan received his B.Sc. and M.Sc. degrees in chemistry at the American College, Madurai, India. He earned his Ph.D. in chemistry (with Professor Ernest L. Eliel) at the University of North Carolina, Chapel Hill and carried out post-doctoral research (with Professor John A.Gerlt) in the field of oligonucleotide chemistry at Yale University and at the University of Maryland. Dr. Manoharan is the winner of the Lifetime Achievement Award of the Oligonucleotide Therapeutics Society (2019), the M. L. Wolfrom Award from the American Chemical Society (2007) and D. Horton Industrial Carbohydrate Chemistry Award from the American Chemical Society (2021).

Mark is Senior Vice President, Clinical Development at Aera Therapeutics. His prior experience in oncology, rare disease, genetic medicine, immune therapy and cell therapy research and development spans 30 years across academia, pharma, and biotech. Prior to joining Aera, Mark served as Vice President, Clinical Development and Head of R&D Medicine at Intellia Therapeutics, where he led multiple gene editing programs at the development candidate and early development stages. Before Intellia, Mark held multiple positions with increasing responsibility and was Executive Medical Director at AbbVie Oncology, where he directed early-stage and late-stage development over global regions. Mark is a physician and surgeon, with prior appointments in academic medicine at Harvard University, the National Institutes of Health, and the University of Chicago.

Federico Mingozzi, PhD, MBA, is Chief Executive Officer of Nava Therapeutics, where he leads efforts to advance mRNA-based therapeutics and overcome the limitations of non-viral delivery. A leading expert in gene therapy and immune responses to viral vectors, Dr. Mingozzi has played a central role in advancing clinical programs for genetic and acquired diseases, including metabolic disorders, bleeding disorders, inherited blindness, and neuromuscular conditions. Prior to Nava, he served as Chief Science & Technology Officer at Spark Therapeutics, where he led research spanning gene therapy innovation, delivery technologies, therapeutic modalities, and manufacturing. Dr. Mingozzi holds undergraduate and doctoral degrees from the University of Ferrara in Italy and an MBA from Drexel University. He has served on the board of the American Society of Cell and Gene Therapy and currently serves as a board member of Sensorion Pharma.

Chris is an independent consultant specializing in CMC related activities for drug substance and drug product. His experience spans from developing oligo related analytical methods in the laboratory, to managing a commercial QC laboratory, and then into being the manufacturing plant manager for Agilent Technologies in Boulder CO. These roles have provided him exposure to many CMC strategies that have been successfully utilized for clinical, pre-commercial, and commercial oligonucleotides in both the drug substance and drug product arenas. Chris also has a wide range of experience in facility buildouts, both new and appended, allowing exposure and understanding of the many unseen, as well as the very visible, parameters related to facility, equipment, process, and personnel that are necessary to align and control in order to ensure a successful and robust scaleup / technical transfer.

Mayurbhai (Mayur) Patel leads Chemistry Development and CMC at Judo Bio, where he oversees oligonucleotide process development, analytical strategy, formulation, large-scale manufacturing, and supply chain. He brings more than a decade of experience in nucleic acid chemistry and CMC and has developed scalable manufacturing processes for 15+ oligonucleotide programs spanning preclinical through late-stage clinical development. Prior to Judo Bio, Dr. Patel was Director of Oligonucleotide Chemistry and CMC at Korro Bio, where he built and led cross-functional teams across synthesis, analytics, formulation, and CMC, and led CMC strategy for the company’s first clinical program in alpha-1 antitrypsin deficiency. Earlier, he contributed to a broad RNA therapeutics portfolio at Nitto Denko Avecia. Dr. Patel earned his PhD in nucleic acid chemistry from Seton Hall University, has authored numerous publications in RNA therapeutics, and currently serves on the Editorial Advisory Board of Nucleic Acid Insights.

Dan Peer is a Professor and the Director of the Laboratory of Precision NanoMedicine at Tel Aviv University (TAU). He was also the Vice President for Research and Development at Tel Aviv University from 2020-2026. From 2017 - Present, he is the Founding and Managing Director of the SPARK program of Translational Medicine at TAU. Professor Peer’s work was among the first to demonstrate systemic delivery of RNA molecules using targeted nanocarriers to the immune system and he pioneered the use of RNA interference (RNAi) in immune cells. His lab was the first to show systemic, cell specific delivery of modified mRNA in an animal to induce therapeutic gene expression of desired proteins. This has enormous implications in cancer, inflammation and infection diseases (e.g. COVID 19 mRNA vaccines). In addition, his lab was the first to show high efficiency, systemic, cell specific therapeutic genome editing in cancer. Prof. Peer has more than 150 pending and granted patents. Some of them have been licensed to several pharmaceutical companies and one is currently under registration (as a new biological drug in Inflammatory Bowel Disease). In addition, based on his work, five spin-off companies were generated aiming to bring innovative personalized medicine into clinical practice. Prof. Peer received more than 30 awards and honors and he serves on the scientific advisory board and as Board Member of more than 15 companies, and on the editorial board of more than 20 journals. In 2023 he was elected to the US National Academy of Engineers (NAE). In 2024, he was elected, Fellow, of the US National Academy of Inventors. In 2025, he was elected Fellow of the Controlled Release Society He received numerous awards and honors amoung them the 2026 Rappaport Award; The 2026 The Landau award in Nanotechnology and the 2026 CRS Founders' Award to name a few.

Paul is Vice President and Head of CMC at City Therapeutics, where he leads CMC strategy, development, and manufacturing operations for the company's oligonucleotide pipeline. With nearly two decades of experience spanning City Therapeutics, Korro Bio, Biogen, Stoke Therapeutics, and Alnylam Pharmaceuticals, he has extensive expertise in oligonucleotide CMC, process development, formulation, technology transfer, and regulatory submissions, including supporting the FDA approval of patisiran and tofersen.

Dmitry Samarsky, PhD, has been at the inception of RNAi technology and drug development—starting in 2001 as Director of Technology Development at Sequitur (acquired by Invitrogen) and (in 2005) as Director of Technology Development at Dharmacon (now part of GE). He then served as VP of Technology Development at RXi Pharmaceuticals, USA (2007-2011), SVP of Technology and International Business Development at RiboBio, China (2011-2016) and, most recently, as Chief Scientific Officer at Silence Therapeutics, Germany/UK (2016-2018) and Ceif Technology Officer at Sirnaomics, US. He is currently a Founding Scientist, CSO and Board Member at ARNAgen Therapeutics, US. Dr. Samarsky has authored more than 40 scientific papers, articles, book chapters, patents and patent applications. He has been an invited speaker at more than 100 international conferences, and currently serves on the Scientific Advisory Boards for the OTS (Oligonucleotide Therapeutics Society). Dr. Samarsky received his doctorate in biochemistry and molecular biology from University of Massachusetts, Amherst (1998), followed by a postdoctoral position as an H. Arthur Smith Fellow for Cancer Research in Michael Green's lab at University of Massachusetts Medical School (1998-2001).

Laura Sepp-Lorenzino oversaw all platform and pipeline research activities across in vivo and ex vivo (engineered cell therapy) areas as Intellia’s Chief Scientific Officer. Before joining Intellia, she was vice president, Head of Nucleic Acid Therapies, Research, and member of the External Innovation team at Vertex Pharmaceuticals, Inc. She also served as vice president, entrepreneur-in-residence at Alnylam Pharmaceuticals, Inc., a leader in the development of RNAi Therapeutics. At Alnylam, she was responsible for the Hepatic Infectious Disease Strategic Therapeutic Area, championed extra hepatic siRNA delivery, and was active in licensing and partnering. Laura spent 14 years at Merck & Co., most recently having served as executive director and department head, RNA Therapeutics Discovery Biology. In this role, she was responsible for identification and optimization of siRNAs and delivery vehicles, advancement of preclinical candidates, and development of an siRNA-conjugate platform to expand the repertoire of tissues accessible to in vivo siRNA delivery. Laura also has expertise in oncology drug discovery and development acquired earlier in her career by leading the Cancer Research Department at Merck West Point and working as an assistant lab member and assistant attending molecular biologist at Memorial Sloan-Kettering Cancer Center. She received her professional degree in Biochemistry from the University of Buenos Aires, Argentina and both her MS and PhD in Biochemistry from New York University. Laura holds professional affiliations with key scientific organizations, including the Oligonucleotide Therapeutics Society, the American Society for Gene and Cell Therapy, the European Society of Gene and Cell Therapy, and the New York Academy of Sciences, as well as a number of oncology societies. She also sits on the scientific advisory board of Thermo Fisher Scientific, Lodo Therapeutics, the U.K. Nucleic Acid Therapy Accelerator and is a member of Taysha Gene Therapies’ board of directors.

Kristy Szretter is a Scientific Director in the Nonclinical Safety and Pharmacology department at Takeda. Prior to joining Takeda, she served as a nonclinical lead at a number of organizations in the biotech industry. With over a decade of experience in nonclinical development, she has supported early and clinical development of biologics, cell therapies, vaccines, and viral and non-viral gene therapies in oncology, autoimmunity, infectious disease, neurology, and rare disease indications. She holds a PhD in Immunology and Molecular Pathogenesis from Emory University and is a Diplomat of the American Board of Toxicology.

Sebastian Trousil is a co-founder and chief operating officer of City Therapeutics. As head of corporate operations, business development and finance, he is enabling the organization to achieve the full impact of our RNAi technology. Prior to City, Dr. Trousil worked at RTW Investments and Flagship Pioneering, where he led multiple company creation efforts at both firms. He is a co-founder of Harbinger Health, a liquid biopsy company for early cancer detection, which he led from initiation to early proof of concept. Dr. Trousil received a master’s degree in pharmacy from the University of Vienna and a Ph.D. from Imperial College London. He conducted postdoctoral research at Harvard Medical School and Massachusetts General Hospital, where he received the prestigious MGH Tosteson Fellowship.

Professor Rakesh N. Veedu is an internationally renowned expert in RNA/DNA therapeutic and diagnostic development including the design, screening, lead molecule identification, preclinical studies and manufacturing chemistries. He completed PhD from the University of Queensland (UQ) in May 2006 after MSc from Griffith University. Later, he joined the Nucleic Acid Centre at the University of Southern Denmark (2006-2010) as a Postdoctoral Fellow. In July 2010, he returned to UQ and established an independent research group. In 2015, he moved to Murdoch University as a Research Head of Nucleic Acid Therapeutics Group through Perron Institute. He has published >115 research articles in internationally reputed journals, published a book, and also an inventor on multiple patents/patent applications. Prof. Veedu has been extremely successful in commercializing research discoveries through the establishment of two spin-out companies and played a pivotal role in the last 10 years to establish a vibrant biotechnology industry landscape in Australia, in addition to mining. He is the Founder, Director & CTO of ‘SynGenis Ltd’ (www.SynGenis.com)—Australia’s commercial oligonucleotide and diagnostic manufacturing company, and the Co-Founder & MD of "ProGenis Pharmaceuticals" (www.progenispharma.com), a precision RNA drug development company head quartered in Perth, Australia.

Since joining Wave in 2017, Dr. Wagner has spearheaded multiple programs focused on the development of steropure oligonucleotides therapies for several neurodegenerative and neuromuscular diseases. Her expertise extends from the early stages of target identification to lead optimization and through to IND enabling studies. She earned her PhD at the University of Chicago in 2016 in Cell and Molecular Biology.

Mike received his PhD from imperial college in London. He spent most of his career at GSK and its legacy companies primarily as an analytical scientist and then becoming the Vice President of Development Chemistry and Analysis for GSK in the UK. He was heavily involved in establishing initial GSK’s oligonucleotide CMC development efforts. Mike has edited 3 books on the analysis of pharmaceuticals, including one on the analysis of oligonucleotides. Since leaving GSK in 2016, he has been consulting with Big Pharma and Biotechnology companies in the development, analysis and manufacture of therapeutic oligonucleotides from pre-clinical to marketing submission.

Nick Yoder is currently Executive Director in the Platform Development group at Dyne Therapeutics. There his work focuses on discovery of receptor-targeted oligonucleotide therapies for genetic neuromuscular diseases, and enhancing Dyne's FORCE delivery platform. Prior to joining Dyne, Dr. Yoder worked for more than a decade on the design, synthesis, and development of therapeutic antibody-drug conjugates, at Magenta Therapeutics and at ImmunoGen, Inc.

Fengjiao is Director of Toxicology in the Preclinical & Clinical Discovery & Development Team at Wave Life Sciences. Prior to joining Wave, Fengjiao served as Principal Investigator (PI) and Professor in Shenyang Pharmaceutical University with extensive Drug Discovery & Development experience. She did her postdoctoral training at University of Arizona. She holds a PhD in Pharmacology and Toxicology and is a Diplomate of the American Board of Toxicology.

With over ten years of experience in oligonucleotide solid-phase and liquid-phase synthesis process development, Xuan Zhou is an accomplished scientist in antisense oligonucleotide (ASO) chemistry. Currently serving as Associate Director and ASO Process Chemistry Lead at Biogen, Xuan has pioneered advancements in oligonucleotide process development, including the successful creation of a scalable liquid-phase synthesis process. They have also published multiple patents and manuscripts contributing to the advancement of therapeutic oligonucleotide manufacturing. Xuan joined Biogen in 2016 as Scientist I and has since progressed through roles of increasing responsibility, including Scientist II (2019-2022), Senior Scientist (2022-2024), and now ASO Process Chemistry Lead. In each role, Xuan has focused on developing innovative methods to enhance process efficiency and scalability in oligonucleotide synthesis. Prior to joining Biogen, Xuan completed a Post-Doctoral Research Fellowship in Organic Chemistry at the University of Texas at Austin, following a Ph.D. in Medicinal Chemistry from Sichuan University in China.

Alex Zinoviev is an mRNA platform leader and RNA biologist with more than a decade of experience in RNA biology and technology. Most recently, she led the mRNA platform at Eli Lilly, spanning mRNA design and engineering, AI-enabled sequence optimization, technology development, and preclinical applications across therapeutic programs. Previously, Alex worked on RNA technology platforms at GreenLight Biosciences and Alltrna, including high-throughput mRNA screening and the development of emerging RNA therapeutic modalities. She now advises biotechnology companies on mRNA technology, platform development, and scientific strategy. Alex received her PhD from Ben-Gurion University in Israel, where she studied translation initiation, and conducted postdoctoral research at SUNY Downstate Medical Center focused on mammalian translation and mRNA quality control.