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. 

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.

Jonathan is co-founder and CSO of EnPlusOne Bio. He did his postdoc in George Church’s Research Group at Harvard Medical School and the Wyss Institute for Biologically Inspired Engineering, where he co-developed the enzymatic RNA synthesis platform that formed the basis of EnPlusOne technology. During his postdoc, he jointly developed methods for tRNA charging and chemically accessing new, nonstandard amino acids. Jonathan earned his BS in Chemistry from Indiana Wesleyan University and a PhD in Organic Chemistry from Indiana University under Professor Michael Vannieuwenhze.

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.

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.

Dr. Thazha P. Prakash is an Executive Research Fellow of Medicinal Chemistry at Ionis Pharmaceuticals and is a well-recognized chemist specializing in RNA therapeutics. Ionis is a biotechnology company dedicated to the development of RNA-based treatments. Throughout his career at Ionis, he has held various research and development positions, making significant contributions to the field. He is a co-inventor of the ligand-conjugated antisense (LICA) technology, which enables tissue-specific targeting of antisense oligonucleotide therapeutics. Currently, more than ten LICA ASO drugs are in clinical development. Some of his key innovations include the development of: 2’-O-NMA and cEt BNA for antisense oligonucleotides (ASOs),  5’-vinylphosphonate and fully modified RNAs for small interfering RNAs (siRNAs),  chemical strategies to enhance the selectivity of siRNAs, monovalent and trivalent GalNAc conjugates for targeted delivery of ASOs to hepatocytes, GLP-1 conjugate for targeted delivery of ASOs to pancreatic β-cells, lipid and Ang II peptide conjugates for targeted delivery of nucleic acids to muscle,  neurotensin peptide conjugate for targeted delivery of nucleic acids to the brain, chemically modified guide RNA for CRISPR/Cas9-based genome editing. His research has contributed to the development of several notable drugs, including Spinraza, Waylivra, Wainua, Qulsody, and Tryglolza. Additionally, he contributed to early research that led to the discovery of 2’-methyl nucleosides for anti-HCV drugs. He is the author/inventor of more than 225 peer-reviewed publications, issued patents, and pending patent applications.

Xiao Wang is an associate professor in the Department of Chemistry at MIT and a core institute member at the Broad Institute of MIT and Harvard. Her lab develops and applies new chemical, biophysical, and genomic tools to better understand tissue function and dysfunction at the molecular level. Wang joined MIT and the Broad after conducting postdoctoral research at Stanford University with Professor Karl Deisseroth, where she was a postdoctoral fellow of the Life Sciences Research Foundation. At Stanford, she developed comprehensive methods for analyzing RNA in intact tissues that merge sequencing with imaging, in order to reveal the locations of various cell types in the brain. Wang received her B.S. in chemistry and molecular engineering from Peking University in 2010 and her Ph.D. in chemistry from the University of Chicago in 2015, where she elucidated the cellular functions of RNA modifications with Professor Chuan He.

Dr. Masad J. Damha is Distinguished James McGill Professor of Chemistry at McGill University. He is a leading expert in nucleic acid chemistry and has made significant contributions to the development of new methods for synthesizing and modifying nucleic acids.  His ‘chemistry toolbox‘ is being applied by multiple research laboratories and industries to oligonucleotide manufacturing and therapeutic development in gene silencing, and DNA and RNA gene editing strategies.  With his students, he has authored more than 230 publications, and filed/received several patents worldwide.

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.

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 (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.

Stanley T. Crooke, M.D., Ph.D. is founder, chairman and CEO of n-Lorem Foundation, a nonprofit focused on discovery, developing and providing personalized, experimental treatments for nano-rare patients (1 to 30 patients worldwide). Prior, Dr. Crooke founded, was Chairman, CEO and Lead Scientist of Ionis Pharmaceuticals, where he led the scientific development of a new platform for drug discovery, antisense oligonucleotide (ASO) technology and the creation of one of the largest development pipelines in the biotechnology industry. Dr. Crooke has received a number of awards, most recently, the Indiana University School of Medicine Steven C. Beering Award, the Prix Galien Roy Vagelos Pro Bono Humanum Award, the American Chemical Society’s E.B. Hershberg Award for Important Discoveries in Medicinally Active Substances, the Lifetime Achievement Award presented by the Oligonucleotide Therapeutics Society, the Scrip Lifetime Achievement Award and the 2019 Massry Prize. He received his M.D. and Ph.D. degrees and house staff training at Baylor College of Medicine, where he currently serves on the Board of Advisors.  He has published >600 scientific publications, edited more than 20 books, has numerous patents, and led the development of more than 23 commercialized drugs.

Dr. Kerr is the Chief Medical Officer at Dyne Therapeutics.  He is a neuroscientist and neurologist by training with a ten-year career as a faculty member at the Johns Hopkins School of Medicine before a 20-year career at present in the Boston area creating and serving in a series of biotechnology companies in the genetic disease and neurology/neuromuscular space. 

Doug has over 130 publications in medical journals many of which involved stem cell transplantation for preclinical models of neurologic disorders.    He has led the development of several therapies for neurologic and rare genetic diseases while working in the biotechnology industry.  He has participated on the boards and SABs of several non-profit organizations, including the Transverse Myelitis Association, CureSMA and the ALS Association as well as several biotechnology companies including Disarm Therapeutics, Triplet Therapeutics and FAZE Medicines.  Doug has been actively involved in gene and cell therapy programs and is on the Scientific Review Panel of the California Institute of Regenerative Medicine.

Dr. Muthiah (Mano) Manoharan serves as the Senior Vice President of Drug Innovation, a Scientific Advisory Board Member, and has earned the title of  Alnylam Distinguished Research Scientist at Alnylam Pharmaceuticals, Cambridge, Massachusetts. In 2003, he was the founding chemist hired at Alnylam as the Head of the Drug Discovery. He and his team pioneered the discovery and development of numerous chemical modifications, GalNAc conjugation chemistry, lipid conjugates (the first in vivo demonstration of RNAi in 2004) and Lipid Nanoparticles (LNP) delivery platform that made RNA interference-based human therapeutics possible.

Prior to joining Alnylam, he worked at Isis (Ionis) Pharmaceuticals in the field of antisense oligonucleotides and contributed to many clinical candidates. He is an author of more than 250 publications (more than 71,500 Google Scholar citations with an h-index of 122  and an i10-index of 452) and over 600 abstracts, as well as an inventor of over 300 issued U.S. patents. 

Among the numerous awards Dr. Manoharan has earned, two are worth pointing out: He is the winner of the Lifetime Achievement Award of the Oligonucleotide Therapeutics Society (OTS, 2019), and has been honored with the Professor Ronald Breslow Biomimetic Chemistry National award by the American Chemical Society for the year 2024.

Dr. Manoharan was born in Madurai, Tamil Nadu, India and received his B.Sc. and M.Sc. degrees in chemistry at the American College, Madurai, Tamil Nadu, India. He earned his Ph.D. in chemistry at the University of North Carolina, Chapel Hill, (Professor Ernest L. Eliel) and learned the field of oligonucleotides at Yale University and University of Maryland as a post-doctoral research associate (Professor John A. Gerlt).

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.