creating a
healthier future

1st Symposium on
Oligonucleotide Technologies and Therapeutics@Portugal – OTP2021

8-9 July 2021 | ONLINE EVENT


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The first Symposium on Oligonucleotide Technologies and Therapeutics in Portugal (OTP2021) wants to bring together national and international scientists from academia and industry, as well as the medical community, interested in nucleic-acids-based technologies and therapeutics. We expect to cover a wide range of topics in different sessions dedicated to:

I- Nucleic Acids Chemistry, Structures and Technologies
II- Delivery and Targeting
III- Discovery and Development (pre-clinical and clinical)

We aim to contribute to the creation of a network and forum by gathering national young and established scientists in the country, both from academia and industry, together with leading international guest speakers. We encourage you to take this opportunity to discuss your research and to submit your abstract for a poster presentation.


Abstract Book download >>


Organizing Committee
Pedro Moreno (INEB/i3S)
Sofia Santos (INEB/i3S)
Nuno Azevedo (FEUP, LEPABE)
Rita Sobral Santos (FEUP, LEPABE)
Nuno Guimarães (FEUP, LEPABE)
Sandra Alves (Instituto Nacional de Saúde Dr. Ricardo Jorge/CECA-ICETA)
Liliana Matos (Instituto Nacional de Saúde Dr. Ricardo Jorge/CECA-ICETA)
Daniel Vasconcelos (Faculty of Medicine, Biomedicine department, University of Porto)


Scientific Committee
Pedro Moreno (INEB/i3S)
Sofia Santos (INEB/i3S)
Ana P. Pêgo (INEB/i3S)
Nuno Azevedo (FEUP, LEPABE)
Rita Sobral Santos (FEUP, LEPABE)
Nuno Guimarães (FEUP, LEPABE)
Sandra Alves (Instituto Nacional de Saúde Dr. Ricardo Jorge/CECA-ICETA)
Liliana Matos (Instituto Nacional de Saúde Dr. Ricardo Jorge/CECA-ICETA)
Daniel Vasconcelos (Faculty of Medicine, Biomedicine department, University of Porto)
Bruno Godinho (UMass, RNA Institute, USA)



DAY 1 | 8 JULY 2021

All indicated times are Lisbon/ London time.


10:30 – 10:45 Login Start 

10:45 – 11:00 Welcome note 
Pedro Moreno, INEB, i3S


Session I – Nucleic acids chemistry, structures and technologies 

specially supported by: Alnylam 

Synthesis and modifications of nucleoside, nucleotides and oligonucleotides; conjugates; DNA/RNA nanotechnology; aptamers; sensors/diagnostics. 

Chairs: Nuno Azevedo, FEUP, LEPABE & Daniel Vasconcelos, Faculty of Medicine, Biomedicine department, University of Porto

11:00 – 11:30 Targeting of cells and viruses using nano-engineered ligand displays
Jørgen Kjems, iNANO, Arrhus University, DK 

11:30 – 12:00 Chemical methods to isolate modified aptamers
Marcel Hollenstein, Coordinator of Bioorganic Chemistry of Nucleic Acids Group, Department of Structural Biology and Chemistry, Institut Pasteur, FR


12:00 – 13:00 Lunch Break  


13:00 – 15:00 Poster Session I (all odd posters) / Networking  


Chairs: Pedro Moreno, INEB/i3S & Nuno Guimarães, FEUP, LEPABE

15:00 – 15:30 Living in the World of RNAi Therapeutics: A Triumph of Biomimetic Chemistry
Muthiah Manoharan, Alnylam Pharmaceuticals, USA 

15:30 – 16:00 Exploring the synthesis and the therapeutic potential of structurally new nucleoside and nucleotide analogs
Nuno M. Xavier, Univ. Lisboa, PT 

16:00 – 16:30 Aptamers for toxin detection
Carina Almeida, INIAV, PT 


16:30 – 17:00 Coffee break  


Session II – Delivery and Targeting 

Mammalian and bacterial cell uptake; nanoparticle-based delivery; conjugate-based delivery; tissue targeting; intracellular trafficking. 

Chairs: Daniel Vasconcelos, Faculty of Medicine, Biomedicine department, University of Porto & Rita Sobral Santos, FEUP, LEPABE

17:00 – 17:30 Retinal gene delivery by intravitreal injected nanoparticles: the obstacle course to the inner retina
Katrien Remaut, Univ. Gent, BE

17:30 – 18:00 Delivery of RNA Therapeutics: Overcoming the Billion Year Old Endosomal Fortress
Steven Dowdy, Univ. California, San Diego School of Medicine, USA 




DAY 2 | 9 JULY 2021

All indicated times are Lisbon/London time.


Continuation, Session II – Delivery and Targeting 

Chairs: Sofia Santos, INEB/i3S & Rita Sobral Santos, FEUP, LEPABE & Liliana Matos, Instituto Nacional de Saúde Dr. Ricardo Jorge/CECA-ICETA 

09:30 – 10:00 Promise only what you can deliver
Ana P. Pêgo, i3S, Univ. Porto, PT 

10:00 – 10:30 Design strategies for protein-mediated delivery of therapeutic cargo
João Gonçalves, iMed-Univ.Lisboa, PT 

10:30 – 10:37 Special Outreach Session: How can the COST network DARTER help researchers in the therapeutic oligonucleotide field?
Virginia Arechavala-Gomeza, Biocruces Bizkaia Health Research Institute, ES


10:37 – 11:00 Coffee break  


Session III – Discovery and Development 

specially supported by: IONIS

Chairs: Sofia Santos, INEB/i3S & Liliana Matos, Instituto Nacional de Saúde Dr. Ricardo Jorge/CECA-ICETA 

11:00 – 11:30 Antisense oligonucleotide-mediated correction of aberrant pseudoexon splicing in Inherited Metabolic Diseases
Lourdes Ruiz Desviat, Universidad Autónoma de Madrid, ES 

11:30 – 12:00 RNA-based therapies for Machado-Joseph disease
Luís Almeida, CNC, Univ.Coimbra, PT 


12:00 – 13:00 Lunch break  


13:00 – 15:00 Poster Session II (all even posters) / Networking 


Chairs: Sandra Alves, Instituto Nacional de Saúde Dr. Ricardo Jorge/CECA-ICETA & Pedro Moreno, INEB/i3S

15:00 – 15:30 Application of nucleic acid mimics to control Candida albicans virulence
Sónia Silva, CEB , UMinho, PT 

15:30 – 16:00 Preclinical development of tcDNA-ASO for neuromuscular disorders
Aurélie Goyenvalle, University of Versailles, FR 

16:00 – 16:30 Antisense Therapeutics To Treat Neurological Diseases
Brett P. Monia, IONIS Pharmaceuticals, USA 


16:30 – 16:45 Coffee break  


Chairs: Sofia Santos, INEB/i3S & Sandra Alves, Instituto Nacional de Saúde Dr. Ricardo Jorge/CECA-ICETA

16:45 – 17:15 Oligonucleotide therapeutics: the experience of clinical development of disease modifying drugs for ATTR amyloidosis
Teresa Coelho, Hospital Sto António, Centro Hospitalar do Porto, PT 


17:15 – 17:30 Closing note  
Pedro Moreno, INEB/i3S



Confirmed speakers:

SESSION I - Nucleic Acids Chemistry, Structures, and Technologies

Muthiah (Mano) Manoharan 

Senior Vice President of Drug Innovation Chemistry, Scientific Advisory Board Member, and Distinguished Research Scientist at Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA.

Dr. Muthiah (Mano) Manoharan serves as the Senior Vice President of Drug Innovation Chemistry, a  Scientific Advisory Board Member, and a Distinguished Research Scientist at Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA. Dr. Manoharan has had a distinguished career as a world-leading chemist in the areas of oligonucleotide chemical modifications, conjugation chemistry, and delivery platforms (lipid nanoparticles (LNPs), polymer conjugates, and complex-forming strategies).  Dr. Manoharan and his research group designed, synthesized, and demonstrated for the first time the human therapeutic applications of GalNAc-conjugated oligonucleotides at Alnylam, a platform that has revolutionized the nucleic acid-based therapeutics field with several compounds currently in the advanced clinical trials.   Under his leadership, the Alnylam team developed the ionizable lipid nanoparticles (LNPs), the formulation for ONPATTRO (patisiran), the first RNAi therapeutic approved by FDA. ONPATTRO was approved in 2018 for treating TTR amyloidosis-mediated polyneuropathy. The GalNAc platform has been validated by the first clinically approved GalNAc-siRNA conjugate, GIVLAARI (givosiran), which was approved for the treatment of acute hepatic porphyria by the FDA in November 2019. 
Prior to Alnylam, Dr. Manoharan worked at Ionis  (formerly Isis) Pharmaceuticals and  LifeCodes Corporation in the field of antisense oligonucleotide therapeutics. He is an author of more than 215 publications (nearly 43,000 Google Scholar citations with an h-index of 94 and an i10-index of 380) and over 400 abstracts, as well as an inventor of over 245 issued U.S. patents.  
Dr. Manoharan has been recognized as the Lifetime Achievement Awardee of the Oligonucleotide Therapeutics Society for the year 2019.  He received the M. L. Wolfrom Award from the American Chemical Society in 2007.

Marcel Hollenstein

Coordinator of Bioorganic Chemistry of Nucleic Acids Group, Department of Structural Biology and Chemistry, Institut Pasteur, France

Marcel Hollenstein studied chemistry at the University of Lausanne (Switzerland) where he received his Masters's degree in 1999. He then earned his PhD in 2004 working in the group of Prof. Christian Leumann (University of Bern, Switzerland) on fluorinated peptide nucleic acids. In 2005, he joined the group of Prof. David Perrin (University of British Columbia, Vancouver, Canada) to work on in vitro selection of modified DNA enzymes. He started his independent scientific career at the University of Bern in 2009 as a Swiss National Science Foundation (SNSF) Ambizione Fellow. Since 2016, he has been an independent junior research group leader at Institut Pasteur (Paris, France) in the Department of Structural Biology and Chemistry. His research interests focus on the synthesis of modified nucleoside triphosphates for their use in SELEX for the identification of modified aptamers and DNAzymes for therapeutic and biosensing purposes. 



Nuno M. Xavier

Assistant Researcher at Centro de Química Estrutural (CQE), Faculty of Sciences, University of Lisbon (FCUL), Portugal

Nuno M. Xavier (b. Nov. 1982, Vila Real, Portugal) is a Researcher at Centro de Química Estrutural (CQE), Faculty of Sciences, University of Lisbon (FCUL). He was awarded a double Ph.D. degree in Chemistry (Organic Chemistry) from the University of Lisbon and from the National Institute of Applied Sciences of Lyon in 2011. He was afterward a Postdoctoral Research Fellow at the University of Natural Resources and Life Sciences of Vienna and then at FCUL. In 2014 he was awarded an Investigator Starting Grant at FCUL and recently he has been selected as Assistant Researcher at CQE, both of which under highly competitive calls from the Portuguese Foundation for Science and Technology (FCT). His research has been devoted to the development of efficient synthetic methodologies for novel bioactive carbohydrate-based molecules, for which he has been internationally recognized with various Young Scientist Awards (e.g. IUPAC, Alberta Ingenuity Centre for Carbohydrate Science - Canada, Groupe Lyonnais des Glyco-Sciences - France) and an Innovation Award at the International Carbohydrate Symposium 2018. He has been particularly focused on nucleoside and nucleotide chemistry towards new molecules of potential therapeutic application. He was the PI of an FCT-funded R&D exploratory project and a Team Member of 8 projects funded by national or international entities. He has acted as Guest Editor for Pharmaceuticals and Frontiers in Chemistry (Section “Medicinal and Pharmaceutical Chemistry”) and as Topic Editor for Frontiers in Chemistry and Antibiotics. His research activities have been reported in ca. 40 publications and he has been the Speaker of more than 30 lectures in international and national Symposia. 


Carina Almeida

Associate Researcher of the National Institute of Agrarian and Veterinary Research (INIAV), Portugal. Co-founder of Biomode S.A.

Carina Almeida finished her Ph.D. degree in Biomedical Engineering in 2010, at the University of Minho (UM), in collaboration with the University of Southampton, United Kingdom. From 2010 to 2014 C. Almeida worked as a Post-doctoral at the Center of Biological Engineering (UM) and the Faculty of Engineering of the University of Porto. From 2014 to 2016 she was the CSO at Biomode, a startup company that exploits the commercial potential of technological solutions to the diagnostic market. In 2017, C. Almeida has moved to the National Institute for Agrarian and Veterinary Sciences (INIAV) as a Senior Scientist. Since 2020, she also serves as the Head of the Food Safety Unit of INIAV.
C. Almeida's main research interests are to explore the potential of nucleic acid mimics, in particular for food safety, agrarian and clinical applications. She is also interested in the epidemiological aspects of pathogenic bacteria with human and veterinary impact. During her career, C. Almeida has authored/co-authored 40 papers in peer-reviewed international journals, 12 book chapters, and 5 patents. She is currently supervising/co-supervising 6 Ph.D. students, and she participates (or participated) as a PI and as a researcher in 7 FCT-funded projects and in 4 European/International projects.


rgen Kjems

Professor, Interdisciplinary Nanoscience Centre (iNANO), Department of Molecular Biology and Genetics, Aarhus University, Denmark

Jørgen Kjems studied chemistry and physics at Aarhus University from where he received his Ph.D. degree in the field of RNA in 1989. After being a postdoctoral fellow at Harvard Medical School, Boston, USA 1989-1990 and at MIT, Cambridge, USA 1990-1991 he returned to the Department for Molecular Biology, the University of Aarhus where he became an associate professor in 1994. In 2003 he was appointed as a full professor in Nanoscience and was co-founder of the Interdisciplinary Nanoscience Centre (iNANO), a truly interdisciplinary, physically assembled entity at the University of Aarhus with the active participation of more than 300 scientists, which he headed in the period of 2014-2018. Since 2017 he has headed the Centre of Excellence for Cellular Signal Patterns, CellPat. He has published more than 400 international research papers (H-index of 80) and received several prizes including the Novo Nordisk Prize in 2018. His research is centered around RNA/DNA and protein-based nanotechnologies.



SESSION II - Delivery and Targeting


Steven F. Dowdy

Professor, Univ. of California San Diego, School of Medicine, USA. Board member of Oligonucleotide Therapeutics Society

Professor Steven Dowdy, from the University of California San Diego School of Medicine, has focused his research on the delivery of novel therapeutics into cells, especially RNAi therapies. He is the founder and serves on the Scientific Advisory Boards of several Biotech companies and is a member of the Board of Directors of the Oligonucleotide Therapeutics Society. He is a co-author of more than 100 peer-reviewed high-impact publications in cell and molecular medicine.



Katrien Remaut
Univ. Gent, BE

Katrien Remaut was born on the 10th of November 1978 in Ghent. In 2001, she graduated as a Pharmacist with the greatest distinction. She then started research work in the Lab of General Biochemistry and Physical Pharmacy under the guidance of Prof. De Smedt and Prof. Demeester. In 2007, she received the title of doctor in pharmaceutical sciences with her dissertation ‘Exploring the relation between the intracellular fate and biological activity of nucleic acid nanoparticles’ and continued research work in the lab as a postdoctoral fellow of the Research Foundation Flanders. In 2009, Katrien joined the Directors Research Lab under the guidance of Prof. Ian Mattaj at the European Molecular Biology Laboratory for 6 months. Katrien is the author and co-author of several peer-reviewed scientific publications in e.g. Advanced Drug Delivery Reviews, Biochemistry-US, Biomacromolecules, Biophysical Journal, Journal of Controlled Release, ASC Nano, Nanomedicine and Macromolecules. She received several scientific prices (the scientific price 'Bank van Breda' in 2001; the Highlights of student posters Award in 2003; winner of the AIO competition in 2004; Jan Feijen Poster Award in 2010 and Price of the Royal Academy of Medicine for Scientific Research in Pharmacy, period 2008 – 2011). In 2013, Katrien was elected as a member of the Young Academy in Flanders. She was appointed tenure track professor at the Lab General Biochemistry and Physical Pharmacy, Faculty of Pharmaceutical Sciences in 2014.


Ana P. Pêgo

Coordinator of the nBTT - nanoBiomaterials for Targeted Therapies Group at i3S - Instituto de investigação e Inovação em Saúde, Universidade do Porto, Portugal

Ana Paula Pêgo got her Ph.D. in Polymer Chemistry and Biomaterials from the University of Twente, the Netherlands, in 2002. In 2003 she moved to INEB – Instituto de Engenharia Biomédica, where she became a Principal Investigator in 2012. In 2015, INEB joined the i3S – Instituto de Investigação e Inovação em Saúde (Universidade do Porto), where Ana Paula Pêgo leads the nanoBiomaterials for Targeted Therapies (nBTT) Group.
By using nanomedicine strategies the nBTT Group, aims at providing in situ and in a targeted manner the required signals to promote nervous tissue regeneration. The research on new biomaterials for application in neurosciences includes the development of new polymers for the design of alternative vectors to viruses for efficient nucleic acid delivery and preparation of nerve grafts for spinal cord injury treatment. Societal and ethical issues that concern Regenerative Medicine and NanoMedicine are also topics in which Ana Pêgo is involved.
She has been appointed the Scientific Director of the Bioimaging Centre for Biomaterials and Regenerative Therapies of INEB and she is an Invited Associate Professor at the Instituto de Ciências Biomédicas Abel Salazar (ICBAS) and at the Faculty of Engineering (FEUP) of the University of Porto.
Currently, Ana Paula Pêgo is a member of the Board of Directors of i3S, serves as an Associate Editor of Biomaterials (Elsevier), and is the Secretary of the Council of the European Society for Biomaterials.

João Gonçalves

Head of the Biopharmaceutical and Molecular Biotechnology Unit at the iMed-Research Institute for Medicines and Pharmaceutical Sciences, Faculty of Pharmacy, University of Lisbon, Portugal.

Joao Goncalves is Head of the Biopharmaceutical and Molecular Biotechnology Unit at the iMed-Research Institute for Medicines and Pharmaceutical Sciences and Professor of Immunology and Biotechnology both part of the Faculty of Pharmacy, University of Lisbon, Portugal. He is also Head of Antibody Engineering Laboratory at iMed – Faculdade Farmacia Universidade Lisboa. Professor Joao Goncalves has a pharmaceutical degree and Ph.D. in Infectious Diseases/Immunology. He has postdoctoral experience at Harvard University and Scripps Research Institute, La Jolla. His research focuses on therapeutic antibody discovery and development ranging from virology to oncology. His group is interested in biomolecular and biopharmaceutical engineering aiming at the investigation, analysis, and integration of genomic and proteomic data towards the design of therapeutic proteins. This will lead us to guide protein engineering through molecular medicine and bio-nanotechnology in a mid-to-long-term timeframe. At the center of our biopharmaceutical development, we aim at rational antibody engineering as our approach to validating our theoretical concepts. We consider the use of intracellular and extracellular small-domain antibodies to be exemplary in our rational protein engineering strategies. We have applied our stability engineering strategies to the construction of hyper-stable and effective small antibodies. 
The focus on the discovery and development of a new class of therapeutic antibodies and efficient display technologies may allow the rapid identification of binding proteins with high affinity and specificity. The domain antibodies combine the advantages of conventional antibodies with the benefits of small-molecule drugs. 




Virginia Arechavala-Gomeza

Virginia Arechavala-Gomeza is an Ikerbasque Research Professor at Biocruces Bizkaia Health Research Institute. Chair of COST Action "Delivery of Antisense RNA ThERapeutics (DARTER).
Her main interest is the development of better methods to evaluate candidate treatments for neuromuscular disorders, such as RNA therapies. She also leads the international network DARTER, including over 350 researchers in 31 countries aiming to improve the delivery of RNA drugs to target tissues (
Prof. Arechavala-Gomeza studied pharmacy at the University of the Basque Country, Spain, followed by an MSc at the University of Strathclyde in Glasgow, before moving to London to complete a PhD studying SOD1 mutations that cause amyotrophic lateral sclerosis (King’s College London). After her PhD, she joined the laboratory of Prof Francesco Muntoni (Imperial College London and University College London) as a postdoctoral researcher where she participated in the preclinical development and the first clinical trials for eteplirsen, approved for the treatment of Duchenne muscular dystrophy.
She has authored many scientific publications (cited over 3000 times), collaborates as an expert with the European Medicines Agency, is a founding member of CERU/SRUK (Spanish researchers in the UK), and actively participates in science communication activities in English and Spanish.

* Picture: Iñigo Sierra

More information:



SESSION III - Discovery and Development


Brett P. Monia

CEO, IONIS Pharmaceuticals, USA

Dr. Monia is the chief executive officer, a member of the board of directors, and a founding scientist of Ionis Pharmaceuticals. His contributions at Ionis include research into the medicinal chemistry and mechanisms of action of RNA-targeting modalities to treat human diseases, most notably antisense-based therapeutic strategies. Dr. Monia has extensive experience across a range of therapeutic areas, including oncology, metabolic disease, inflammation, neurological disease, and cardiovascular disease, which have resulted in a broad range of successful clinical achievements and in marketing approvals for new medicines.
Dr. Monia has published more than 200 primary research manuscripts, reviews, and book chapters, and is an inventor on more than 100 issued patents. He serves as a senior editor for the journal Nucleic Acid Therapeutics. He is on the board of directors of Cognition Therapeutics and Dynacure. He has also served as president of the Oligonucleotide Therapeutics Society (OTS). Dr. Monia is an adjunct professor of biology at San Diego State University where he lectures at the graduate level on pharmacology.
Dr. Monia received his Ph.D. in Pharmacology at the University of Pennsylvania and B.S. degrees in Molecular Biology and Analytical Chemistry at Stockton State College in Pomona, New Jersey.


Luís Almeida

Assistant Professor at Faculty of Pharmacy, the University of Coimbra and Principal Investigator and head of Molecular Therapy of Brain Disorders group at CNC-Center for Neuroscience and Cell Biology, University of Coimbra, Portugal.

Luís Pereira de Almeida, is a tenured associate professor at Faculty of Pharmacy, University of Coimbra. His research is developed at CNC-Center for Neuroscience and Cell Biology, the University of Coimbra Portugal where he is Principal Investigator and President. Luis also coordinates the Center for Innovative Biomedicine and Biotechnology (CIBB).
Luis did his Ph.D. in the Gene Therapy Center of Lausanne, CHUV, Switzerland, and spent short sabbatical leaves at CEA, France(2005), and at Massachusetts Institute of Technology (MIT-2010) and has been vice-president of the Portuguese Society for Stem Cells and Cell Therapy (2013-2015). 
Luis leads a talented research group focused on gene and stem cell therapy approaches for brain disorders, particularly Machado-Joseph disease/spinocerebellar ataxia type 3. He has published over 100 papers/book chapters (h-factor=41). Luis mentored 12 pos doctoral fellows and 17 Ph.D. students and coordinated over 26 research projects, such as the transnational projects Synspread and ModelPolyQ within the European Joint Programme for Neurodegenerative Diseases (JPND), and presently including ViraVector - a Research Infrastructure for production and experimentation with viral vectors that integrate the national roadmap of scientific infrastructures.


Aurélie Goyenvalle

Group Leader in the Unit of Therapies for Neuromuscular Diseases at Université de Versailles St- Quentin U1179 INSERM, France

Dr. Aurélie Goyenvalle is directing a research group at the University of Versailles focused on gene and antisense therapies for the treatment of neuromuscular disorders. Aurelie received her Ph.D. in Virology at the University of Paris VII in 2006 from her work at Genethon in France, where she developed an exon-skipping gene therapy strategy for Duchenne muscular dystrophy (DMD) using adeno-associated virus vectors (AAV) encoding chimeric U7snRNA constructs carrying antisense sequences. To pursue her interest in gene therapy and splicing modulation approaches for DMD, she joined Prof. Kay Davies’ laboratory at the University of Oxford as a post-doctoral scientist supported by an EMBO fellowship. During her postdoc in Oxford and subsequent position at the Medical Research Council (MRC), Aurélie developed various splicing modulation approaches including the optimization of the U7 snRNA system for neuromuscular and neurodegenerative diseases and the evaluation of peptide-conjugated antisense oligonucleotides. In 2011, she joined the Institute of Myology in Paris to investigate splice switching approaches using novel antisense oligonucleotides and in 2012, she was awarded a Chair of Excellence program to establish her own group at the University of Versailles to develop novel RNA based technology for the treatment of neuromuscular diseases. In this context, she has notably demonstrated the therapeutic potential of a novel class of AONs made of tricyclo-DNA (tcDNA), which displays unique pharmacological properties and unprecedented uptake in many tissues after systemic administration. In 2015, Aurélie has been appointed a permanent research scientist position by the French national institute for health and medical research (INSERM) to pursue this line of research and she is currently head of a team evaluating splice-switching approach for various genetic disorders.


Lourdes Ruiz Desviat

Associate Professor at the Department of Molecular Biology, University Autonoma of Madrid; Director of the research institute “Centro de Biología Molecular Severo Ochoa”, Madrid, Spain. Group leader in the research line “Physiopathology studies and therapeutical approaches in animal and cellular models of neurometabolic diseases”.

Dr. Lourdes R Desviat is an Associate Professor at the Department of Molecular Biology, University Autonoma of Madrid. She is a group leader in the research line “Physiopathology studies and therapeutical approaches in animal and cellular models of neurometabolic diseases” and currently she is the Director of the research institute “Centro de Biología Molecular Severo Ochoa”, Madrid. Dr. Desviat and her research group have been working for several years in the field of inherited metabolic diseases. They focus mainly on organic acidemias and hyperphenylalaninemias, by generation and characterization of relevant animal and cellular models, to be used as research tools to understand the molecular and physiopathological mechanisms, to analyze potential biomarkers for prognosis and follow-up, and to identify new therapeutical targets. Their ultimate aim is to develop and perform for many of the diseases under study preclinical studies of personalized RNA targeted therapies as well as pharmacological therapies with antioxidant compounds and mitochondrial activators. The research group has ample experience in the in vitro and in vivo use of antisense therapy to revert splicing defects in different rare diseases, participating in EU-COST Actions within this topic. To date, they have generated iPS cells derived from patients’ fibroblasts and differentiated them to neurons and cardiomyocytes as disease-relevant cell lineages. Now, they are using gene-editing CRISPR/Cas9 technology for the generation of cellular and animal models with specific splicing mutations, to identify and test candidate therapeutical antisense oligonucleotides.

Dr. Desviat is an author of more than 136 publications (with an h-index of 29 and 79 in Q1) and co-author of 16 book chapters. She has also supervised several Master's (8) and Ph.D. thesis (13).

Sónia Silva

Assistant Researcher at the Biofilm Research Lab, Dept. Biological Engineering, Univ. Minho, Braga

Sónia Silva received her graduation in Biology from Porto University in 2000 and in 2005 finished her Master in Molecular Genetics at the University of Minho (UM), in the field of yeasts metabolism and protein purification. Sónia Silva finished her Ph.D. in Biomedical Engineering in 2006 at the Center of Biological Engineering (CEB)-UM and at Cardiff University (UK). Her Ph.D. focused on the characterization of Candida spp virulence factors and antifungal resistance. Since 2013, Sónia Silva has been working as Assistant Researcher at CEB-UM, and her research focuses on two main research lines: i) identification of molecular mechanisms underlying Candida virulence and antifungal resistance; ii) exploration of NAMs technology to control Candida spp virulence and antifungal resistance. She has authored 76 original papers, 10 book chapters, and 1 book, with over 3600 citations and with an H-index of 29. Sónia Silva registered 2 patents and supervised 4 PhDs, 8 MScs, and 3 graduate students. She is managed 4 competitive national and international research projects all in the field of Candida infections. 


Teresa Coelho

Neurologist and Neurophysiologist, Andrade’s Unit for Familial Amyloidosis, Santo Antonio Hospital, Centro Hospitalar Universitário do Porto

Teresa Coelho, MD, PhD. is a Neurologist and Neurophysiologist at Santo António Hospital, Centro Hospitalar Universitário do Porto, where clinical investigation led to the first description of familial amyloid polyneuropathy (FAP), published in Brain in 1952. 
Since 1997 Dr. Coelho has been head of Andrade’s Unit for Familial Amyloidosis at Centro Hospitalar Universitário do Porto. This Unit is an outpatient multidisciplinary clinic and is a reference center for this disease. The hospital’s historical register includes about 3,000 observed subjects from 650 families. More than 1000 subjects are observed every year including 70 newly diagnosed subjects.
Dr. Coelho is also coordinator of the neuromuscular outpatient clinic and principal investigator of several pivotal clinical trials in transthyretin FAP, including those testing and leading to the approval of a siRNA and an ansisense oligonucleotide for the treatment of ATTR amyloidosis.



Abstract Submission

Authors are invited to submit abstracts for a virtual poster presentation. In the virtual poster sessions, besides the regular digital poster we will also encourage (optional) presenters to upload a short video presentation of their poster that can help to present the work in the most efficient way (detailed instructions will be sent later).


Abstracts should be structured according to the template, under the headings below:

Title should be in bold Times New Roman font (font size: 14; 1pt spacing between lines).

Type initials and surnames of the authors (Times New Roman; font size:12; centred; 1pt spacing between lines). Underline the name of the presenting author. Include superscripted letters by alphabetical order referring to the affiliations.

This should include the name of department or laboratory, followed by the name of the organisation (university/company), city and country (Times New Roman; font size:10; justified; 1pt spacing between lines and 6pt spacing between paragraphs). One affiliation per line.

Abstract body
This should be in Times New Roman font (font size: 12; justified; 1pt spacing between lines and 6pt spacing between paragraphs). Abstract must be structured as: introduction, materials and methods, results, conclusions.

Graphs and tables are optional.

References (if necessary)
Along the text the references should be numbered and in between square brackets. In the references list they should be in Times New Roman font (font size: 11; justified; 1pt spacing between lines) and in Vancouver style.

Acknowledgements (if necessary)
This should be in Times New Roman font (font size: 11; justified; 1pt spacing between lines). Include the title in bold.

Additional Rules
The margins should be set to (sup.: 2,5cm; Inf.: 2.5cm; Left.: 2.5cm; Right.: 1.5cm). Abstract length is limited to one page A4, including tables and graphs, and all of the topics above.

Abstract submission deadline: 21st May 2021
Communication of abstracts results: 24th May 2021 

Deadline for submission of the work for the virtual poster presentation: 21st June 2021 


- Undergraduate, MSc, PhD Student: 20€
- Academia/Clinicians: 20€
- Industry/Non-Academia: 50€

Deadline for registration and payment of participants with submitted abstracts: 7th June 2021 
Deadline for registration and payment of participants without abstracts: 25th June 2021







European Union’s Horizon 2020 research and innovation programme under grant agreement No 810685










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