


The conference will discuss the development of nucleic acid medicines including recent clinical successes, and current and upcoming challenges. By bringing together academics in basic and translational research and their peers in thriving biotechnology companies and promising start-ups, the conference aims to foster dialogue and to spearhead collaborations. The first day (December 8th, 2025) will cover the areas of basic nucleic acid biology and translational developments. The second day will discuss clinical work using nucleic acid gene therapies, along with a panel discussion. The final day will cover nucleic acid chemistry and include a meet the editors session over lunch.
Event details
Speakers

Rebecca Ahrens-Nicklas
CHOP, USA

Julia Alterman
University of Massachusetts Chan Medical School, USA
Julia Alterman is an Assistant Professor at the RNA Therapeutics Institute at UMass Chan Medical School. She began her scientific career in chemical biology and pharmacology, contributing to the development of small molecules and peptide-lipid conjugates at biotech companies in the Boston area. She earned her PhD in Dr. Anastasia Khvorova’s lab, where she developed a novel branched siRNA scaffold enabling potent, widespread gene silencing in the central nervous system—technology now licensed to Atalanta Therapeutics for treating neurodegenerative diseases.
Julia’s research focuses on overcoming the challenge of oligonucleotide delivery to extrahepatic tissues, with active programs targeting skin, muscle, bone, and eye. Her lab develops hyper-functional siRNA sequences and delivery platforms, including a skin-targeted siRNA currently in Phase 2a trials for alopecia areata in collaboration with Alys Pharmaceuticals. She also serves the oligonucleotide therapeutics community as a board member of the Oligonucleotide Therapeutics Society, on the editorial board of Molecular Therapy – Nucleic Acids, and on several scientific advisory boards.

Maria Barna
Stanford University, USA
Dr. Barna obtained her B.A. in Anthropology from New York University and her Ph.D. from Cornell University, Weill Graduate School of Medicine. Dr. Barna was subsequently appointed as a UCSF Fellow through the Sandler Fellows program, which enables exceptionally promising young scientists to establish independent research programs immediately following graduate school. She is presently an Associate Professor in the Genetics Department at Stanford University. Dr. Barna has received a number of distinctions including being named a Pew Scholar, Alfred P. Sloan Research Fellow, and top ’40 under 40’ by the Cell Journal. She has received the Basil O’ Connor Scholar Research Award and the NIH Directors New Innovator Award. She is the recipient of the Elizabeth Hay Award, H.W. Mossman Award, Tsuneko and Reiji 'Okazaki Award', American Society for Cell Biology Emerging Leader Prize, the Rosalind Franklin Young Investigator Award, and the RNA Society Early Career Award. She is presently a NYSCF Robertson Stem Cell Investigator.

Howard Chang
Stanford University, USA

Grace Chen
Yale University, USA
Dr. Grace Chen obtained a BS in Chemical Biology from the University of California, Berkeley and received her PhD in Chemical Biology from Harvard University, where she worked in Dr. David Liu’s lab studying RNA modifications. Dr. Grace Chen was a postdoctoral fellow with Dr. Howard Chang at Stanford University researching engineered and endogenous circular RNAs. Dr. Grace Chen joined the faculty of the Yale University Department of Immunobiology in 2019 as an Assistant Professor and the Robert T. McCluskey Yale Scholar. She is a Rita Allen Foundation Scholar and an Allens Distinguished Investigator. The Chen Laboratory investigates the diverse functions and applications of circular RNAs and RNA modifications in health and disease.

David Corey
UT Southwestern, USA
As a Ph.D. student in the laboratory of Dr. Peter Schultz, I learned that simple chemical modifications could dramatically change the ability of oligonucleotides to recognize complementary RNA and DNA targets. During my postdoctoral work with Dr. Charles Craik, I studied the interplay of amino acid chemistry and function, and how interdisciplinary collaborations enable biomedical science to be conducted at a high level. I began my independent career at UT Southwestern Medical Center because it had a culture of collaboration and the resources necessary to make those collaborations succeed. In the thirty-two years I have spent at UTSW, I have found colleagues who have taught my lab new techniques, provided critical ideas and feedback, and acted as close research partners in multi-year projects at the interface of cell biology, biochemistry, and nucleic acids.

Stanley Crooke
n-Lorem Foundation, USA

James Dahlman
Emory University, USA

Masad Damha
McGill University, Canada
Dr. Damha has served as the President of both the International Society of Nucleosides, Nucleotides and Nucleic Acids and the Oligonucleotide Therapeutic Society, and currently serves on the Editorial Board of the journal Nucleic Acids Therapeutics. He has received numerous awards and distinctions for his research, including the Ray Lemieux and the Bernard Belleau Awards, both from the Canadian Chemical Institute, and the Queen Elizabeth II Diamond Jubilee Medal from the Governor General of Canada. Damha received his B.Sc. and Ph.D. in organic chemistry from McGill University.

Mitch Guttman
CalTech, USA

Chris Hart
Eli Lilly, USA

Doug Kerr
Dyne Therapeutics, USA

Arthur Krieg
Zola Therapeutics, USA
Arthur M. Krieg, MD has worked in the oligonucleotide field since the 1980s. Art graduated from Haverford College in 1979, received his MD from Washington University in 1983, and completed a residency in Internal Medicine at the University of Minnesota in 1986. He was a Staff Fellow at the NIH in the Arthritis Institute from 1986 to 1991, when he joined the University of Iowa, becoming Professor of Internal Medicine in the Division of Rheumatology. He has had 19 years of patient care experience, although his focus has always been on basic research and teaching. Art discovered the immune stimulatory CpG DNA motif in 1994, which led to a new approach to immunotherapy and vaccine adjuvants. Based on this technology he co-founded Coley Pharmaceutical Group in 1997, discovering and taking 4 novel oligonucleotides into clinical development, including the CpG 7909 in the approved vaccine, Cyfendus. Art was co-founder, CSO of Coley Pharmaceutical Group from 1997 until its acquisition and incorporation into Pfizer in 2008. He then served as CSO of Pfizer’s Oligonucleotide Therapeutics Unit from 2008 to 2011, becoming co-founder and CEO at RaNA Therapeutics from 2011 to 2013, and CSO at Sarepta until July 2014. In 2015 he founded Checkmate Pharmaceuticals to develop novel oligonucleotides for cancer immunotherapy, and then served as CSO until its acquisition by Regeneron in 2022. 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.
Art is currently an adjunct Professor in the UMass Chan Medical School RNA Therapeutics Institute, a mentor for the Termeer Foundation, and serves on the scientific advisory boards of several companies developing oligonucleotide therapeutics. Most recently Art founded his fourth biotech company, Zola Therapeutics, with the goal of developing a new generation of immunostimulatory oligonucleotides for cancer immunotherapy. He has published more than 250 scientific papers (h-index >130) and is an inventor on >50 issued US patents covering oligonucleotide technologies.

Mano Manoharan
Alnylam Pharmaceuticals, USA

Anne Pyle
Yale University, USA

John Rittichier
EnPlusOne Bio, USA

Sam Tsimikas
Ionis Pharmaceuticals, USA
Dr. Tsimikas is Senior Vice President and Cardiovascular Franchise Leader at Ionis Pharmaceuticals. He has spearheaded the development of multiple RNA therapeutics with regulatory approval of several drugs (Kynamro, Waylivra, Wainua) and has directed the design and execution of approximately 50 clinical trials in RNA therapeutics targeting Lp(a), APOC3, PCSK9i, ANGPTL3, angiotensinogen and TTR. He is a practicing Board-Certified Cardiologist and Professor of Medicine at the University of California San Diego School of Medicine. He is the Founding Director of the Vascular Medicine Program and the UCSD “Lp(a) Clinic”. He has published his work in major medical journals, including NEJM, Lancet, Nature, Cell, JACC, Circulation, EHJ, and has over 400 original manuscripts, review articles and book chapters.

Xiao Wang
Thomas D. and Virginia Cabot Associate Professor of Chemistry

Anne Willis
MRC Toxicology Unit, University of Cambridge, UK
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