The oligonucleotide field is showing great potential to provide novel therapeutic opportunities to treat patients both in rare and more broader diseases. For the last 7 years I have been working with different types of oligonucleotides including mRNA, antisense oligonucleotides and siRNAs and really appreciate the wealth of knowledge that the oligonucleotide therapeutic community has shared with me and others. The importance of communicating the nucleotide research more broadly and sharing learnings as nucleotide therapeutics are discovered, tested in patients and reach the market is I feel a key objective of the OTS. I also believe that the OTS represents a healthy mixture of academia, biotech and big pharma representing the science diversity and breath/depth of knowledge required to push the scientific boundaries in the field. Currently, I have a leadership role in AstraZeneca with a focus on oligonucleotide discovery including nucleotide therapeutics and targeted delivery of RNA molecules as well as driving collaborations in the field with academic and industrial partners.Championing the area both within AstraZeneca and in the European academia has given me many insights into people’s perceptions of the field and activities the OTS may want to engage in to promote the area to a wider community.
As member of the BOD during the past 2 years I have contributed to the society by actively participating in board and OTS meetings including reviewing proposals for OTS local delivery events. I have also promoted OTS within Sweden and Europe to increase the engagement and participation of scientist into the OTS.
I have also engaged in different activities to promote oligonucleotide field such as Nucleic Acid Therapy Accelerator in UK; DARTER network in EU and OligoNova in Sweden.
Helping to mentor and coach young scientists is important to me. Within AstraZeneca, I try to provide scientific leadership in the oligonucleotide filed and I spend a significant part of my time mentoring young talent and support training of post-docs, graduates and PhD students and would have the support of the younger members of the OTS society high on my agenda.
I would be very happy to continue to contribute strongly to the Oligonucleotide Therapeutics Society such that the society and OTS meetings continue to thrive and are attractive for scientists to attend.
I have more than 20 years of experience in the Pharmaceutical industry in research from discovering and developing small molecules and during the last 7 years also from RNA based therapeutics. I have broad experience from both management of departments and teams as well as setting and delivering business strategies. I am also involved in several collaborations with biotech companies as well as with academic groups around the globe. I obtained a PhD in organic and analytical chemistry in 1989 from Linköping University. After a post-doc in Germany, I joined Linköping University as a Senior lecturer in Organic Analytical Chemistry and became an Associate Professor (Docent) in 1996 during which time I also gave birth to 2 children. In 1997, I joined AstraZeneca as a senior researcher and during my career I have held numerous management roles including Senior Director Drug Metabolism and Pharmacokinetics, Head of Enabling Technologies and Head of Lead Optimization, Medicinal Chemistry. I am currently Chief Scientist and Head of Oligonucleotide Discovery in Discovery Sciences at AstraZeneca. I have strong experience as a project leader for research and business change projects locally and globally within AstraZeneca. I am currently involved in several collaborations e.g. with Ionis Pharma, Moderna Therapeutics, Silence Therapeutics and OligoMed (EU funded) within the RNA field. I have over 60 peer reviewed publications, 2 book chapters and 6 patents and have presented my work at conferences throughout my career including chairing sessions at conferences.
Member of the board for OligoNova, Swedish national centre for research and development of therapeutic oligonucleotides
Member of Scientific Advisory Board Nucleic Acid Therapy Accelerator (NATA), UK
Member of the management committee for European Cooperation in Science & Technology action: Delivery of antisense RNA therapeutics
Board member, Swedish Chemical Imaging Infrastructure
Member of several Joint steering Committees for collaborations with AstraZeneca partners
Isabel Aznarez, PhD
Co-founder & VP, Discovery Research Stoke Therapeutics
For the last two years, I have served in the OTS board of directors, and I would like to serve for an additional term to continue to spread the word regarding the mission of the OTS and the potential of nucleic acids therapeutics. During my membership in the BOD, I co-chaired the Outreach/Website committee whose goal was to review the website and make recommendations for improvements, liaise with online and social media content consultant regarding Webinars, Hot Papers/Paper of the Month and Social Media posts, and liaise with Event Innovations to establish a new program to allow members to submit articles on ‘hot topics’ for website publication. In addition, I contributed to the OTS N-of-1 briefing document and have been a member of the N-of-1 task force. In the past year, I have led the new “Education and Outreach” committee, whose mission is to educate junior and new OTS members, and to also establish and maintain connections with stakeholders such as patient organizations and regulators. In addition, the committee is planning to set up mentoring programs for undergraduate and graduate students to promote careers in nucleic acids therapeutics. My goal for the next year is to continue to push on education through the Education and Outreach committee. Thank you for the opportunity to continue to serve on the BOD of the OTS.
Dr. Isabel Aznarez is an expert in modulation of RNA processes using antisense oligonucleotides. Isabel and Professor Adrian Krainer discovered a scientific approach to increase full length, fully functional protein production. The resulting technology platform – TANGO (Targeted Augmentation of Nuclear Gene Output) – represents an entirely new way of treating the underlying causes of severe genetic diseases. The acronym is a nod to Isabel’s Uruguayan roots. Isabel co-founded Stoke Therapeutics in 2014 with the goal of using TANGO to make medicines that can amplify protein production and restore human health. She has extensive experience in human genetics, RNA metabolism and modulation of RNA processes using antisense oligonucleotides and holds numerous patents and publications for this foundational work. Isabel plays a central leadership role at Stoke as Head of Discovery Research. Prior to founding Stoke Therapeutics, she was a research investigator with Professor Adrian Krainer, whose lab she joined as a postdoctoral fellow in 2008. Previously, Isabel was a researcher at the Hospital for Sick Children with Professor Lap-Chee Tsui, where she focused on the effect of cystic fibrosis mutations on the splicing of the CFTR gene. Isabel holds a Ph.D. in molecular and medical genetics from the University of Toronto and a B.Sc. in biology and human genetics from the University of Uruguay.
OTS, AES, ASGCT. Member of the board of directors of Reaching U.
My introduction and interest in the oligonucleotide therapeutics world began in 2012 during my PhD at the University of Montpellier, France. When I started my PhD I did not know much about RNA therapeutics but it took me only few months to become fascinated by the field and realize the transformative nature of RNA therapeutics research. After my graduate studies, I was fortunate to be able to carry out my research in the field as a postdoc in Anastasia Khvorova’s lab at University of Massachusetts Medical School, USA. This incredible opportunity reinforced my strong interest and fascination about the RNA therapeutics field.
I attended my first OTS meeting in 2016 in Montreal when I joined Anastasia Khvorova’s lab and I have been attending the annual meetings since then. I discovered an excellent meeting with exceptional science and friendly interaction between academia and industry as well as young and senior scientists. At each OTS meeting I am always fascinated on how the society does an excellent job of covering the latest breakthrough in the field from chemistry all the way to oligonucleotides in clinic. I feel very lucky to be part of the amazing community and be able to share my research at OTS events. I had the great opportunity to present my work via poster presentation (poster award in 2016) and talks (invited 2019 and selected from abstract 2017) as well as to give a OTS webinar on siRNA design in January 2021.
It will be an honor to serve on the OTS Board of Directors for multiple reasons. First, I believe that the OTS plays a major role in advancing the field by bringing together the community to share science and recent developments. Secondly, I admire the society values and in particular the will to make the field accessible to everyone by organizing annual meeting with educational seminars and proposing educational webinars. Last but not least, I had the great opportunity to be part of the organizing committee of the virtual annual meeting this year that gave me a view of the inside of the OTS organization and a taste of contributing to the society which I enjoyed a lot.
Finally, I believe that I could bring valuable insights to the Board as I acquired skills and expertise in oligonucleotide chemistry, design and delivery. I made contributions to the field by engineering conjugated fully chemically stabilized oligonucleotides to enhance their delivery into extrahepatic tissues. I was able to identify conjugates that allow functional delivery in several tissues beyond liver such as lung, heart, muscles, fat, adrenal glands. These findings provide examples of how rational lipid engineering may be used to fine-tune the properties of therapeutic oligonucleotides and their extrahepatic delivery. I was honored to receive Dr Alan Gewirtz memorial scholarship in 2020 for my research work as well as be a OTS featured member in June 2021.
I would like to thank you for considering my application and really hope that we could work together to advance the field.
I am an Associate Principal Scientist in the Oligonucleotide Chemistry team at AstraZeneca R&D, Sweden. My work mainly focuses on the development of safe and efficient oligonucleotide therapeutics to treat diseases with unmet clinical needs. Over the past 9 years, I acquire a robust expertise in designing and developing novel chemical modalities to improve oligonucleotide therapeutic delivery. Before joining AstraZeneca in 2020, I was a postdoctoral associate at the RNA Therapeutics Institute, USA in Anastasia Khvorova’s lab, one of the pioneers in oligonucleotide therapeutics research. There, I made major contributions to the development of novel chemical platforms for enhancing the delivery and uptake of siRNA therapeutics. I was able to design and identify novel conjugated siRNAs that enable safe, sustainable and robust silencing in various extra-hepatic tissues including muscle, heart, and lung. This crucial discovery allows for the targeting of several new targets for therapeutic intervention and advance the oligonucleotide therapeutics field. In 2015, I obtained my PhD in chemistry from the University of Montpellier (France), where my work focused on the chemical synthesis of RNA prodrugs for the development of new therapeutic oligonucleotides. I established and optimized synthetic methods to successfully synthesize novel 2’-modified siRNAs and demonstrated that incorporation of 2’-biolabile moieties can be efficiently used to improve siRNA stability and uptake without major toxicity.
Masad J. Damha, PhD, FCIC
Distinguished James McGill Professor McGill University
I have associated with the OTS since its 1st OTS meeting in 2005 (Rockefeller U, NY) where I met some of the most prominent figures in our field. It was then when I knew I have found a Society to which I could contribute. First as poster and oral presenter, and later as board member, eventually serving as President of the OTS (2012-13). Those were exciting years; new committees started to be formed under the leadership of our Board and subsequent Presidents (Brett Monia, Art Krieg, Annemieke Aartsma-Rus) and our Society witnessed great growth. When my term in the Board ended in 2014, I continued to serve in the Scientific Advisory Council, and co-hosted of the 12th Annual Meeting (Montreal, 2016). I have also Chaired our Chemistry Sessions at OTS meetings for the past several years. Now that my term in the Board of the International Society of Nucleosides, Nucleotides & Nucleic Acids is ending, I would like to join the OTS Board of Directors to propose new activities such as building a mentorship program for new investigators (and members at large), facilitating their growth at their workplace, serving as ‘service point’ that connect them to academic and industrial centers. One of my best experiences as an academic and graduate supervisor is sharing my own personal experiences with new students and equipping them with tools that will help them move and succeed at the workforce. I would like to do this not only at McGill but also at my “OTS “University!
I teach Chemistry at McGill, and I work with MSc, PhD and postdoctoral students pursuing studies in Nucleic Acid Chemistry & Biology. With them, I have been developing chemically modified oligonucleotides and methods for their synthesis. Through our work, we have discovered many of them have properties that render them adaptable for numerous biological applications. In some cases, these studies have been essential in deciphering structure-function relationships between our oligomers and enzymatic machinery associated with the antisense, RNAi, and CRISPR-Cas9 pathways. We start with chemistry to design and construct molecules, test them biochemically/biophysically in vitro and then, through collaborative work, we apply them in live-cells and animal disease models (Duchenne Muscular Dystrophy, Brain Cancer, etc.). For more information about our work, students, and scientific publications, please visit our website: http://damha-group.mcgill.ca/
Academies, Societies & Foundations:
International Society of Nucleosides, Nucleotides & Nucleic Acids (IS3NA)
(Board Member, Immediate Past President).
Foundation for the Advancement of Science, Technology and Education of Nicaragua (FASTEN)
National Academy of Science of Nicaragua (ACN)
Why do you want to serve on the OTS Board of Directors?
The Oligonucleotide Therapeutics Society has been a great platform for me, and other young scientists alike, to learn and network with the Titans in the field. The simple, informal at many times, and rich environment of the annual meetings have been extremely enjoyable and inspiring. Thus, the primary reason for me to serve on the board of this society, is the opportunity to serve THE SCIENTISTS that make up the fabric of this tightly knit community. Furthermore, it would be a great honor and privilege to help shape the present and future of the OTS and share the responsibility of maintaining it as stimulating as it has been to better educate the next generation of oligonucleotide scientists.
What talents and skills will you bring to the Board? Qualification and past service
I am a trained pharmacologist with 12 years of experience in delivering oligonucleotides to the brain, yet “I am nothing… Apart from that, I have in me all the dreams in the world.” ― Fernando Pessoa. The quote from this famous Portuguese author portrays an important aspect that I generally like to focus on, more than any of my hard skills: the commitment, motivation, curiosity, and passion that drives the scientist in me. I believe I can be an asset in many ways to the BOD, and I am confident that my organizational and managerial skills, as well as my scientific knowledge, will be appreciated and useful to help fulfill OTS’s mission.
To highlight some of the service to the scientific community:
I currently serve as a scientific advisor at the Health & Technology Research Centre (ESTESL), and I have also served on organizing committees for scientific meetings, including: the 17th annual meeting of the OTS (virtual meeting, 2021), where I participated by co-chairing a session (Preclinical session 2); and the 1st Symposium on Oligonucleotide Technologies & Therapeutics in Portugal.
I serve as editorial board member for Frontiers in Drug Delivery (Frontiers) and Saúde & Tecnologia (ESTESL), and I am an Ad-hoc reviewer for more than 12 scientific journals (including: Nucleic Acids Research; Nucleic Acids Therapeutics; Scientific Reports; Biomaterials; and ACS Chemical Neuroscience).
Bruno Godinho received his foundational pharmacy training at the Lisbon School of Health Technology (ESTESL, Portugal) where he obtained his Bachelor’s degree in Pharmacy. During his time in college he undertook research internships at Utrecht University (Utrecht, The Netherlands) and Universidade NOVA de Lisboa (Caparica, Portugal), which were key shaping his interest in pursuing a career in scientific research. His Master degree in Clinical Pharmacology obtained at the University of Glasgow (Scotland, UK) fostered, for the first time, his interest in gene therapy and gene silencing. He later received his Doctorate degree from University College Cork (Cork, Ireland) where he studied formulation-based approaches for the delivery of therapeutic oligonucleotides to the central nervous system (CNS). He then joined Prof. Khvorova’s Lab at the RNA Therapeutics institute (UMass Medical School) as a postdoctoral fellow to learn and gain experience in the design and delivery of fully-modified conjugated therapeutic oligonucleotides. During his training he made significant contributions towards the identification of neuroactive siRNA scaffolds that enable potent and sustained gene silencing in the CNS, without the need for synthetic formulation. In 2016, he received the Milton-Safenowitz Post-Doctoral Fellowship Award from the Amyotrophic Lateral Sclerosis Association (ALSA) to support his research. Bruno is also the recipient of other career awards, including the Dr. Alan M. Gewirtz Memorial Scholarship Award (Oligonucleotide Therapeutics Society), the STAT 2019 Wunderkind Award (STAT News) and a Silver Medal from the Polytechnic Institute in Lisbon (Instituto Politécnico de Lisboa). While a postdoc, Bruno acted as an Adjunct Professor at the Lisbon School of Health Technology (ESTESL, Portugal) and Guest Lecturer at the Instituto de Engenharia de Lisboa (ISEL), teaching pharmacy-related subjects and advanced therapeutics.
At Atalanta Therapeutics, a CNS-focused RNAi therapeutics company, Bruno leads platform development and innovation efforts, and is the lead in vivo pharmacologist for several internal and partnership programs.
External Scientific Advisor at the Health & Technology Research Centre (Escola Superior de Tecnologia da Saúde de Lisboa (ESTESL), Lisbon, Portugal)
Guest Lecturer in Advanced Human Therapies, Master Program in Biomedical Engineering (Instituto Superior de Engenharia de Lisboa/ESTESL, Lisbon, Portugal)
I am a molecular biologist by training, and I became fascinated with small RNAs very early in my career, earning a PhD in microRNA mechanisms and completing a postdoctoral training in the RNAi Therapeutics group at the Novartis Institutes for BioMedical Research. After this extensive training in RNA biology, I joined the investigative toxicology group at Alnylam Pharmaceuticals in 2014 as a scientist, becoming the head of the group and a Diplomate of the American Board of Toxicology (DABT) in 2016. During my tenure at Alnylam, I have been fortunate to be able to contribute to multiple projects where molecular understanding of an issue enabled finding a mitigation strategy, and successfully translating it in humans, from bench to bedside and back.
The first OTS meeting I participated in was in 2016 in Montreal. I was very impressed with the quality of the science and the passion of the participants. Since then, I enjoyed attending the meeting annually and giving multiple oral presentations, including on the safety of 2’F monomers in 2018. In addition, I have been a speaker, a co-organizer, and/or a moderator at multiple other oligonucleotide-focused conferences and workshops, including TIDES, the Annual Meeting of the American College of Toxicology (ACT), Cold Spring Harbor, DIA/FDA Oligonucleotide-Based Therapeutic Conference, AAPS National Biotechnology Conference (NBC), and Pharmaceutical & BioScience Society International (PBSS). I have co-authored 19 peer-reviewed papers in the field, including on increasing the specificity of RNAi therapeutics (Nat Commun. 2018) and on the safety of 2’F monomers (Nucleic Acids Res. 2019; March 2019 OTS Paper of the Month). I would welcome the opportunity to leverage the network I built through these experiences to support the mission of the OTS.
I believe I can bring a valuable perspective to the board as a female scientist with both basic oligonucleotide research and therapeutic development experience, and a proven track record of cross-functional leadership and teamwork. My experience in investigative toxicology, in particular, may nicely complement the existing expertise of the board. I would be honored channel my passion for the field as a member of the OTS Board of Directors to provide strategic input on the direction of the field and to support its important mission to facilitate the discovery and development of efficacious and safe oligonucleotide treatments for the patients in need.
I am currently a Director, Investigative Toxicology, at Alnylam Pharmaceuticals where I am leading mechanistic efforts related to nonclinical safety evaluation of RNAi therapeutics targeting a variety of tissues, including the liver, eye, and CNS. I earned a Bachelor of Arts in Chemistry and Biochemistry from the University of Colorado in 2007, and a PhD in Biological and Biomedical Sciences from Harvard University in 2012, working with Dr. Carl D. Novina on novel regulators of microRNA biogenesis and function. After completing a postdoctoral fellowship in the RNAi Therapeutics group at Novartis Institutes for BioMedical Research, I joined Alnylam Pharmaceuticals in 2014, and assumed positions of increasing responsibility in the Early Development organization. In addition to leading the investigative toxicology function, I co-led cross-functional CNS platform efforts to optimize and de-risk novel conjugates for CNS diseases, enabling the advancement of our first CNS program to the clinic. More recently, I have been co-leading the RNA Sciences platform team tasked with optimizing siRNA designs for RNAi and beyond. I have successfully passed the exam to become a Diplomate of the American Board of Toxicology (DABT) in 2016 and was awarded the OTS Young Investigator Award in 2020.
One of my most notable contributions to the RNAi therapeutics field includes demonstrating for the first time that the main driver of GalNAc-siRNA hepatotoxicity in vivo is seed-based off-target activity and that thermally-destabilizing seed modifications can mitigate this liability (Nat Commun. 2018). This discovery fundamentally changed our siRNA lead selection paradigm and provided the framework for the ESC+ platform, with multiple programs now in the clinic. In addition, I have been leading the efforts to establish and implement a de-risking paradigm for unnatural monomers utilized in our RNAi therapeutics, including publishing the first comprehensive in vitro and in vivo de-risking of 2′-fluoro nucleotides (Nucleic Acids Res. 2019; March 2019 OTS Paper of the Month). Using this de-risking paradigm, we have been able to transition multiple other unnatural nucleotides to development to support platform advances.
As someone who has spent his entire professional life working on therapeutic oligonucleotides, I highly appreciate the mission of the Oligonucleotide Therapeutics Society to promote research in the field, provide a forum for open scientific exchange, and foster communication and cooperation among scientists across academia and industry, across disciplines and globally across borders.As part of this framework, the OTS is also providing a platform and support for the next generation of scientists, who are entering this field.
Throughout my professional careerin the biopharmaceutical industry, I had the opportunity to workon the advancement of different classes of therapeutic oligonucleotides, such as antisense oligonucleotides and siRNAs. Co-leading Alnylam’s delivery and platform technology efforts, I contributed to the development of lipid nanoparticles and GalNAc conjugates, two clinically validated platforms for thedeliveryof nucleic acids. This platform advancements resulted in the regulatory approval of the first four RNAi therapeutics with many more currently advancing through various stages of development.
Triggered by the significant clinical successes of the past few years as well as the rapid emergence of highly effective mRNA vaccines against SARS-COV2, we are currently witnessing a rapid expansion of the field of nucleic acid medicines. This has already resulted in severalnew and exciting challenges, such asthe need to educate a broader audience on the underlying technologies and, critically, the need to substantially increase the number of young scientists and researchers working ontherapeutic nucleic acids. Similarly,the OTS community is challenged to adapt to a field, which is rapidly increasing in size and complexity. Having already served on the OTS Board of Directors for the past term, I believe my experience in the discovery and development of therapeutic nucleic acidswill allow me to continue to support the OTS directly and significantly. Besides being a forum for scientific exchange and debate and fostering collaboration among the scientific community,the Society will have to play an increasingly important rolein educating the public about our science and encouraging open scientific dialogue to counter widespread misinformation. I would be honored to serve on the OTS Board of Directors for another term to support its important mission and to help maintain its focus, relevance, and integrity for the years to come.
Dr. Maier joined Alnylam Pharmaceuticals in 2006 where he currently serves as Vice President, Research heading the RNAi Discovery group and co-leading an interdisciplinary research team focused on RNAi platform technology. In these roles, Dr. Maier has contributed to the development of lipid nanoparticles and GalNAc conjugates, two clinically validated platforms for siRNA delivery, and the advancement of multiple therapeutic programs to development, which culminated in the approval of the first-four RNAi therapeutics (ONPATTRO™, GIVLAARI™, OXLUMO™ and LEQVIO™).Martin received his Ph.D. in Organic Chemistry in 1997 at the University of Tübingen, Germany with Professor Ernst Bayer. He moved to the U.S. for his postdoctoral research at Isis (Ionis) Pharmaceuticals, where he subsequently assumed a permanent position working on novel chemistries and delivery systems for antisense oligonucleotides. During Martin’s more than 20 years of experience in the field of oligonucleotide therapeutics, he has contributed to the advancement of therapeutic nucleic acids across both, ASO and RNAi platforms. He is the author of more than 70 peer-reviewed scientific publications, reviews and book chapters and inventor on more than 30 issued patents.
Current member of the OTS BoD
Member of the American Chemical Society
Member of the Scientific Advisory Board, ProQR Therapeutics
Rebecca R. Miles, PhD
Principal Research Scientist Lilly Research Labs, Indianapolis, IN
Indiana University Purdue University Indianapolis, IN
IU School of Medicine PhD in Biochemistry and Molecular Biology
College of William and Mary, Williamsburg, VA
Master of Arts in Biology
Principal Research Scientist, Lilly Research Labs, Indianapolis, IN
New Therapeutic Modalities, Pharmacology Team Leader, 23 years
I have had a long career (23+years) in pharmaceutical discovery research supporting many therapeutic indications. In addition to exploring mechanism of action studies by investigating differential gene regulation, I was an early adopter of RNAi as a screening tool for functionalizing genes and validating them as therapeutic targets. I expanded our RNAi screening platform to be able interrogate whole genome siRNA libraries. Over the years, I have watched and studied the RNAi space mature into a durable and efficacious modality of its own. The science and groundbreaking research that broke down the barriers to enable RNAi therapeutics has been supported by the OTS, and communicated through its journal, Nucleic Acid Therapeutics. My understanding of the oligonucleotide field would not have happened without the OTS annual meetings (attended 2017-2021) and the body of literature published by its members. I am passionate about RNAi and enabling it as a therapeutic modality to transform patient lives by drugging previously undruggable targets. As such, it would be a great honor to serve and “do the work” of the Society to continue to help advance the oligonucleotide field forward.
In addition to scientific passion and a humble desire to serve the OTS, I bring my years of discovery research and leadership in enabling new therapeutic modalities at Eli Lilly. Most recently, I provide scientific leadership and direction for our internal pharmacology team. I do not have experience serving on a board of directors for a scientific society – but would love to gain that experience. By serving on the board, it is my desire to expand my network and connect more directly with the members of the OTS.
I received a master’s degree in biology from the College of William and Mary and a Ph. D. in Biochemistry and Molecular Biology with a focus on translational control and the integrated stress response from the Indiana University School of Medicine. Over my 20+ year Lilly career as a cell and molecular biologist, I have supported small molecule, peptide, and large molecule discovery efforts in the areas of bone formation, osteoporosis, uterine leiomyomas, breast cancer, osteoarthritis, insulin secretion, atherosclerosis, metabolic syndrome, obesity, kidney disease, oncology, immunology, neurodegeneration, and pain. I have been recognized for leadership and implementation of genomic technologies to drive and impact drug discovery to identify and validate new targets for the Lilly portfolio. Over the last several years, I have continued to evaluate new targets for emerging therapeutic modalities. During my career, I have authored or co-authored 29 papers, given presentations at many national and international scientific meetings, and am a co-author on several patents.
Member of OTS Society 2020-2021
Member of the Society for Investigative Dermatology 2019
Board Member of a local parish in Pendleton, Indiana, 2016-2017
Over the last 15 years, oligonucleotide therapeutics have emerged as a new class of medicines, expanding the druggable genome and significantly shortening the timelines for drug development. As a field, we have benefitted by the excellent research by academics and industrial scientists addressing the key barriers that needed to be overcome to bring nucleic acid therapies to patients. Since 2004, OTS has been instrumental in bringing oligo researchers together, not only at our annual meeting and other venues, but also by encouraging connectivity between its members. Furthermore, as a society we stride to maintain high quality of science being presented at conferences and published in our journal and offering opportunities for mentoring and development of young scientists.
However, there is more for us to do to realize the potential of oligo drugs.
As a BOD member, I am committed to:
Supporting collaborative research to overcome fundamental scientific barriers still facing our oligo drug development
Fostering open discourse, ensuring we maintain the highest quality of our research
Identifying and facilitating professional training and development opportunities for scientists entering the field across the globe
Enhancing communication among scientists, engineers, and the public
Increasing public awareness and understanding of the mechanism and safety of oligo drugs
Facilitating the development and access of oligo drugs for both rare and prevalent diseases
Interfacing with regulatory agencies to facilitate development of new oligo modalities
Championing diversity, equity and inclusion
Dr. Laura Sepp-Lorenzino is Executive Vice President, Chief Scientific Officer at Intellia Therapeutics, a company developing curative genome editing treatments to positively transform the lives of people with genetic diseases. She oversees all drug research across in vivo and engineered cell therapy areas at Intellia. Previously, she was VP, Head Nucleic Acid Therapies at Vertex Pharmaceuticals and before that, VP, Entrepreneur-in-Residence at Alnylam, a leader in the development of RNAi Therapeutics. At Alnylam, Laura was responsible for the Hepatic Infectious Disease Strategic Therapeutic Area, championed Extra Hepatic siRNA Delivery internally, and via a number of collaborations, was active in licensing and partnering. Before joining Alnylam, she spent 14 years at Merck & Co., having most recently served as Executive Director and Department Head, RNA Therapeutics Discovery Biology. In this role, Laura 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. Prior to her work with RNAi, Laura led the Cancer Research Department at Merck West Point, where she managed oncology drug discovery and development. She began her career in academia, as an Assistant Lab Member and Assistant Attending Molecular Biologist at Memorial Sloan-Kettering Cancer Center. Laura received her Professional Degree in Biochemistry from the University of Buenos Aires, and her M.S. and Ph.D. in Biochemistry from New York University.
Since its inception, the Oligonucleotide Therapeutics Society (OTS) has been the leading scientific society promoting the development of oligo-based therapeutics. Since joining the OTS in 2005, I have witnessed and experienced first-hand how our field has grown from a concept that many thought was an impractical if not impossible approach to combat diseases to a flourishing field leading the biomedical worlds response to many unmet medical needs ranging from treating rare genetic disorders to addressing a pandemic that is negatively impacting most of the planets nearly 8 billion human inhabitants.
I previously served on the OTS Board of Directors from 2007-2014 and as the Treasurer of the Society from 2008-2010. I did not run for re-election in 2014 so that others could have a chance to serve and impact the OTS and also so that OTS could help develop a broad base of scientific leaders in the oligonucleotide therapeutics field. Nevertheless I have continued to be highly engaged with the OTS serving as Co-Editor-in-Chief for the Society’s official journal Nucleic Acid Therapeutics from 2011 to the present, a time span over which our journal’s impact factor has steadily increased as our society has continuously grown. In addition, to my service to OTS, I have served on the Board of Directors for the American Society of Gene and Cell Therapy from 2013-2016 as well as multiple sub-committees for it including leading the ASGCT committee on oligonucleotide therapeutics. I have also been an organizer of multiple aptamer meetings and helped start the Cold Spring Harbor Meeting on RNA Therapeutics. Finally, I was asked to testify about the importance of biomedical research including the future use of oligonucleotide-based therapeutics by Senators Lamar Alexander and Patty Murray for the United States Senate in 2015. Thus I believe that I can bring considerable new experience and important insights about the biomedical research world in general and the therapeutic oligonucleotide field in particular to the OTS Board of Directors. Thus I seek election to the OTS Board to serve the members of our Society as additional oligonucleotide therapeutics progress from discovery in the lab to the clinic, to the market and ultimately to positively impact our world community.
I am a translational researcher who has been at Duke University for 26 years working on the discovery and development of RNA/oligonucleotide therapeutics. I obtained my PhD from Weill Cornell University working at Sloan-Kettering Cancer Institute and postdoctoral training in RNA biochemistry with Tom Cech at the University of Colorado. My research program focuses upon the generation and development of nucleic acid therapeutics for a variety of medical applications. In particular we have worked on 1.) translational medicine for treating thrombosis in the settings of cardiovascular disease, stroke and cancer and 2.) therapeutic targeting of malignancies with RNA aptamer-drug conjugates. More recently my research group has also worked upon developing the use of nucleic acid binding polymers to scavenge proinflammatory RNA and DNA-containing DAMP/PAMPs released from dead and dying cells to limit pathological inflammation and generating novel guide RNAs for improved CRISPR gene editing. Moreover three different RNA/oligo-based drugs discovered/invented in my laboratory have been translated into the clinic an accomplishment recognized by the awarding of the 2015 ASGCT Outstanding Achievement award and election into the National Academy of Inventors (2018) and the AAAS (2014). In addition, I have served as the Founding Director of the Duke Translational Research Institute (DTRI; now Duke CTSI) as well as the Associate Director for Translational Research for the Duke Cancer Institute. Finally, I am a scientific founder of five biotechnology companies two of which completed IPOs and one recently securing its initial series capitalization to translate an antiplatelet oligo drug from my lab into First in Human Studies Q4, 2021. Thus, I have extensive experience building research teams spanning the academic-private sectors to move important oligo/RNA discoveries and inventions from the laboratory to the bedside to improve patient care and health.
Board of Directors, Basking Biosciences Inc
Scientific Advisory Board, Rznomics Inc
Scientific Advisory Board, Ovid Therapeutics
Marie Wikström Lindholm, PhD
SVP, Head of Molecular Design Silence Therapeutics
The first time I attended the yearly meeting, at that point as a newbie in the oligo therapeutic field, it felt like coming home. The buzzing throughout the poster session, the engagement manifested in the Q&A for each and every session, the openness, dialogue, and strong scientific focus on subjects where we all have a common goal really resonated with me and I have always made an effort, as I have become more senior in this field, to ensure that my team members get a chance to experience the same thing.
14 years down the line I have both ASO and siRNA drug discovery under my belt, I have experienced an adverse event in the clinic, developed and shared screening tools to reduce the risk for that particular event happening again, and learned a lot about which diverse groups of scientists and science one needs to include at an early stage to get the right molecules to patients – safe and fast. We will never run out of pathways to map, and new ways to use the oligos. I see that many of us in the field keep asking similar questions, and I would like to use my experience together with the rest of Board to make sure OTS as an organization stays on track to enable good collaborations and openness in the pre-competitive space.
With respect to examples of past service I can refer to my role as scientific coordinator in two large EU-funded projects (AtheroBCell and AtheroFlux), and I also have been active as mentor to soon-to-be-ready PhDs at Lund University, guiding the mentees to the scientific world outside academia.
I started out with a BSc in chemistry, a Life Sciences PhD, and post-doctoral research projects in Berkeley and London. I then because an Assistant Professor at the Faculty of Medicine at Lund University to be part of a research team working on a vaccine against atherosclerosis. In 2006 I became an Associate Professor in Experimental Cardiovascular Medicine.
May 2007 I changed pace and started at Santaris Pharma, a biotechnology company developing RNA-targeting drugs using locked nucleic acid (LNA) technology, where I established a well-equipped lipid analysis laboratory. As group leader in Hyperlipidemia and project leader in Metabolic Diseases I worked on LNA oligonucleotide drug development from molecule design through in vitro screening, in vivo activity and metabolism studies, and finally as lipid metabolism expert when the apoB, PCSK9, and miR-122 targeting programs reached the stage of clinical development. I advanced to VP, Director in Discovery Biology. After Santaris was acquired by Roche I was appointed Expert Scientist in Discovery Technology and Head of Targeted Delivery of oligonucleotide conjugates. In that role I headed interdisciplinary teams across Roche sites, led collaborations with academia, and frequently participated in evaluations of outside opportunities for targeted delivery, including setting up PoC study criteria of targeting moieties such as aptamers, antibodies, and small molecule conjugates.
December 2017 I returned to biotech when I was recruited to Silence Therapeutics where I am leading a skilled team focusing on fine-tuning design of GalNAc-conjugated siRNA and exploring extra-hepatocyte siRNA targeting.
I have founded a consultancy, Maclawili, through which I can take on smaller consulting tasks building on my experience outside, and not in conflict with, my work at Silence.
Takanori Yokota,MD, PhD
Professor of Department Neurology Tokyo Medical and Dental University
At present, interest to oligonucleotide research in Asian countries has been rapidly increasing. Nine out of top ten pharmaceutical companies of Japan have started full-scale research and development of oligonucleotide drugs. In 2020, viltolarsen, antisense drug targeting Duchenne muscular dystrophy (DMD) was on the market from Japan, which is the first oligonucleotide drug generated from other than US and EU. In annual OTS meetings, thirteen to fourteen percent of attendees were originated from Asia countries. In the number of acquisition to OTS Website, Japan is the second (8.6%) following US (47.0%) and more than UK (6.1%) or Germany (5.0%). The number of attendees of annual meeting of Nucleic Acids Therapeutics Society of Japan (NATS-J) is reached to more than 700, which is even more than that of OTS.
I have been the BOD member since 2017 and been playing a role in expanding oligonucleotide research in Asia. As president of NATS-J, I have been asking Japanese academic and company scientists to join OTS and pharmaceutical companies to donate annual meeting of OTS with Marc, every year. In annual meeting of NATS-J, I managed OTS symposium since 2015. Total 23 outstanding scientists, many of them were from BOD OTS member, joined this symposium.
I hope to serve the followings as the OTS Board of Directors in the next two years.
1) Increasing OTS members and companies from Asian countries, especially Japan.
2) Asking Japanese companies to donate OTS.
3) Continuation and further development of OTS symposium in NATS-J annual meeting.
4) Expanding N-of-1 trial with oligonucleotide drugs to Asian countries.
5) Candidate to held annual meeting of OTS in Japan 2024.
Dr. Yokota graduated from Tokyo Medical and Dental University in 1984 and completed his Residency and Fellowship in Musashino Red Cross Hospital in 1986. He studied molecular biology and gene therapy in in Bredesen’s lab in Burham Institute (CA, USA) and Buck Center for Aging Research (CA, USA) in 1998-2000. He accepted a position as Professor in 2010, and Chairman of Department Neurology and Neurological Science, Tokyo Medical and Dental University. Dr. Yokota published more than 200 outstanding scientific papers in basic and clinical neurology and molecular medicine. Dr. Yokota’s research has focused on gene therapy with oligonucleotide drug and the molecular mechanism of Neurodegenerative diseases. His group has developed DNA/RNA oligonucleotide (HDO) as a third class of oligonucleotide drug which is highly potent and can regulate extra-hepatic organs including brain by crossing blood-brain-barrier (BBB) and started “RENA Therapeutics”.
He also developed the technology for crossing of high molecular drugs through BBB using glucose transporter 1 recycling and funded “BRAIZON Therapeutics”.
In OTS, he has been the BOD member since 2017 and been playing a role in expanding oligonucleotide research in Asia. He has been the president of Nucleic Acid Therapeutics Society of Japan (NATS-J) as Japanese OTS since 2018.
Dr. Yokota is a leading scientist as well as neurologist for next generation of gene therapy with oligonucleotide drugs.
President of Nucleic Acid Therapeutics Society of Japan (NATS-J)
Division of Genetics & Genomics and Department of Neurology,
Boston Children’s Hospital
I owe a great debt to the Oligo Therapeutics Society community, which welcomed me in 2017 when I, brand-new to the antisense oligonucleotide field. Many members stepped up to advise us (Art Krieg, Susan Srivatsa, Fran Wincott, Frank Bennett, Frank Rigo, Firoz Antia, Sudhir Agrawal, Kim Tyndall, to name only an incomplete few) in our effort to develop a clinical-grade antisense oligonucleotide (milasen) for a young child with a fatal neurogenetic disorder. I’d like the opportunity to repay that favor through my service. In the years since that case, I have benefited greatly from collaborations with many additional OTS members too numerous to name.
I have a long track record of collaborative science, having taken active roles in large scientific consortia like the Autism Sequencing Consortium and the Simons Foundation for Autism Research for the past ten years.
If elected to the OTS Board, I look forward to learning from fellow board members, and will gladly offer my genetics and clinical expertise in return. In particular I hope to be able to bring a unique perspective due to my ongoing efforts to advance the use of oligonucleotide technologies for individualized / N-of-few medicines. I serve as one of the founding contributors to the n-Lorem foundation, the founder of the N=1 collaborative, and an advisor to several other relevant efforts (e.g. the Dutch Center for RNA Therapeutics, or the brand-new Ultra Rare Disease Gene Therapy Network sponsored by the National Institute of Neurological Disorders and Stroke). The OTS has a real opportunity to lead in this field as we grapple with the clinical, scientific, and regulatory opportunities and challenges in this space. Solving these challenges will not only benefit oligonucleotide work, but create a platform for individualized medicine using other nucleic acid-based therapies (siRNA, morpholinos, mRNA, CRISPR, etc.).
Dr. Yu is a neurogeneticist at Boston Children’s Hospital and Harvard Medical School. He obtained his undergraduate degree in Biochemistry at Harvard College. As a MD-PhD student at UCSF, he used molecular genetics to elucidate basic mechanisms of neuronal attraction and repulsion in the wiring of the C. elegans nervous system, and authored or co-authored papers in Science, Neuron, Nature Neuroscience and Nature Reviews Neuroscience. He then completed neurology residency at Massachusetts General Hospital and Brigham and Women’s Hospital. He joined the faculty in the Division of Genetics and Genomics at Boston Children’s Hospital, and is currently Associate Professor at Harvard Medical School and an Associate Member of the Broad Institute. He leads a cross-disciplinary research group that operates at the intersection of genomics, neurobiology, and bioinformatics. Tim’s group was one of the first in the field to deploy high throughput sequencing methods for human genetics, and today continues to use advanced sequencing and analytics to understand the genetic architecture of autism, and other disorders of brain development. His lab has also translated these tools to the hospital, conducting analyses of rapid-turnaround genomic sequencing in both the neonatal intensive care unit and the newborn nursery. Finally, Tim’s group has pioneered disruptive approaches to accelerating treatments for orphan genetic diseases. Beginning with a young girl with Batten disease (a progressive, fatal, neurodegenerative disorder), his team demonstrated it was possible to go from genetic diagnosis to development, testing, and deployment of a novel investigational drug, using an intrathecally delivered, patient-customized oligonucleotide, in just one year’s time. His laboratory continues to work with clinicians, scientists, industry, policymakers, rare disease advocates, and the FDA to streamline this path as an option for children suffering from rare, orphan diseases, and currently has five clinical therapeutic programs in the IND or pre-IND phase for individuals with ultra-rare, orphan neurogenetic conditions.
Staff Physician, Division of Genetics & Genomics and Department of Neurology, Boston Children’s Hospital
Associate Professor, Harvard Medical School
Associate Member, Broad Institute of MIT and Harvard
Scientific Advisory Board, Mila’s Miracle Foundation
Scientific Advisory Board, Dutch Center for RNA Therapeutics
External Consultant Board, Ultra Rare Disease Gene Therapy Network, National Institute of Neurological Disorders and Stroke
Current OTS members are encouraged to participate in the BOD election which will be open through December 12, 2021. All current members have been emailed a link to the ballot which can also be reached here.