Mayo Clinic RNA Discoveries and Therapeutics Conference 2025

Jacksonville, FL US
May 30, 2025 to May 31, 2025

This course offers Live (in-person) and Livestream (virtual) attendance options

Course Directors: Alfredo Quinones-Hinojosa, M.D.Nilufer Ertekin-Taner, M.D., Ph.D., and Himakshi Jhala, M.P.H., M.B.A., FACHE

May 30 - 31 - 2025 - Mayo Clinic Florida - Jacksonville, Florida

The Mayo Clinic RNA Discoveries and Therapeutics Conference 2025 will convene experts in the areas of RNA biology and RNA therapeutics development and application across various disease areas, including but not limited to neurological diseases, cancer, and rare diseases. This conference aims to identify gaps and opportunities in this field, leveraging RNA therapeutics from discovery to translation to clinical trials.

Registration Fee for Live and Livestream
MD, DO, PhD and Scientists$675
Allied Health, Trainees, Residents/Fellows

$500

 

Call for Abstracts

Abstract submissions are open for the Mayo Clinic RNA Discoveries and Therapeutics Conference.  Abstracts will be evaluated for poster display at the course.  Abstract topics may include but are not limited to RNA biology; applications of RNA therapeutics in neurologic, metabolic, rare diseases, or other conditions; computational design; and innovations in RNA therapeutic deliveries.  Abstracts should be 300 words or less and may include one figure or table.

Abstract Eligibility: Original abstracts that either have or have not been presented at other meetings will be considered. Previously published abstracts should be sufficiently modified.

Submission Criteria

  • Register for the conference, create a profile if you have not done so already, then log into your account
  • Complete and submit abstract form no later than March 28, 2025
  • Abstracts are limited to 300 words
  • Complete the form below

Notification of Results:  All primary authors will be notified no later than April 18, 2025 if their submission has been selected for presentation.  Details regarding presentation will follow.

Presentation of Abstracts: Abstracts chosen for presentation will be uploaded to the conference website and presented during the Mayo Clinic RNA Discoveries and Therapeutics Conference 2025 on May 30-31, 2025

Click here to Submit Abstract.  E-mail collins.kalene@mayo.edu with any questions.

Target Audience

This course is relevant for physicians and other healthcare providers specializing in oncology, neurology, rare diseases, and metabolic specialties.  The content also benefits individuals involved in cancer research, neurological studies, the development of RNA-based therapeutics, and clinical trial coordinators.

Learning Objectives

Upon completion of this activity, participants should be able to:

  • Review the current state of RNA therapeutics to include their discovery and translation into practice
  • Assess existing approaches to the discovery of RNA targets
  • Identify gaps in the development of RNA therapies for common complex and rare diseases
  • Assess existing approaches to the development of novel therapies to include treatment and delivery modalities
  • Identify future opportunities for development of RNA therapies in common complex and rare diseases


Attendance at any Mayo Clinic course does not indicate or guarantee competence or proficiency in the skills, knowledge or performance of any care or procedure(s) which may be discussed or taught in this course.

Course summary
Available credit: 
  • 12.50 AMA PRA Category 1 Credit
  • 12.50 Attendance
Event starts: 
05/30/2025 - 7:00am
Event ends: 
05/31/2025 - 5:00pm

Schedule reflects Eastern Time Zone.  Program subject to change.

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Friday - May 30, 2025

7:30 a.m.

Registration, Breakfast and Exhibits

8:10 a.m.

Welcome 
Kent Thielen, M.D., & AJ Dunn

8:20 a.m.

Introduction
Nilüfer Ertekin-Taner, M.D., Ph.D. & Alfredo Quinones-Hinojosa, M.D.

8:30 a.m.

Discovery of RNA-Targeting Compounds and Implications for Human Diseases
Blanton Tolbert, Ph.D.
Jacob Gershon Cohen Professor of Biochemistry and Biophysics at the University of Pennsylvania, HHMI

 

9:30 a.m.

Break and Exhibits

9:45 a.m.

RNA Biology Panel
Haidong Dong, M.D., Ph.D. (Mayo Clinic)
Jane Zhu, M.D., Ph.D. (Mayo Clinic)
Jeff Coller, Ph.D.
Xiling Shen, Ph.D. (MD Anderson Cancer Center)

10:45 a.m.

Short talks by RNA grantees 

11:45 a.m.

Lunch/Exhibit Hall 

12:45 p.m.

Translation Panel
Keith Gagnon, Ph.D. (Wake Forest University School of Medicine)
Julie Allickson, Ph.D. (Mayo Clinic)
Keith Knutson, Ph.D. (Mayo Clinic)
Ran Zheng

1:45 p.m.

RNA Therapeutics from Bench to Bedside: Moderna Experience
Patrick Finn, Ph.D.

2:30 p.m.

RNA Therapeutics from Design to ASO Candidates
Jeff Milton

3:15 p.m.

Group Picture/Break/Exhibit Hall

3:30 p.m.

Industry Panel
Jay Parrish (Pretzel Therapeutics)
Susan Catalano, Ph.D.
Ali Khademhosseini, Ph.D. (Terasaki Institute)
Andrew J. Geall, Ph.D.
Kate Broderick, Ph.D.

4:15 p.m.

Reception & Poster Exhibition (Non-CME)

6:15 p.m.

Dinner (Non-CME)

7:45 p.m.

Adjourn

Saturday - May 31, 2025

7:30 a.m.

Breakfast 

7:55 a.m.

Welcome 
Nilüfer Ertekin-Taner, M.D., Ph.D. & 
Alfredo Quinones-Hinojosa, M.D.

8:00 a.m.

Clinical Translation of Therapeutic RNA Nanovaccines for Cancer
Natalie Silver, M.D.
Director, Head and Neck Cancer Research, PI: Center for Immunotherapy and Precision Immuno-Oncology, Cleveland Clinic 

9:00 a.m.

Break/Exhibit Hall 

9:15 a.m.

Disease Application Panel
Ken Yamada, Ph.D. (UMass Chan Medical School)
Marlen Lauffer, M.D.
Margot Cousin, Ph.D. (Mayo Clinic)
Minerva Carrasquillo, Ph.D. (Mayo Clinic)

Tsuneya Ikezu, M.D., Ph.D. (Mayo Clinic) 

10:15 a.m.

Short talks selected from submitted abstracts

10:45 a.m.

Industrializing the Non-Profit ASO Treatment of Nano-Rare Patients
Stanley Crooke, M.D., Ph.D.
n-Lorem Founder and CEO

11:45 a.m.

Lunch/Exhibit Hall 

12:45 p.m.

Argonautes and RNA Therapies

Phillip Zamore, Ph.D.
UMass Chan School of Medicine, RNA Therapeutics Institute, HHMI

1:45 p.m.

Disruption of RNA Metabolism in Neurodegeneration and Pathway to Treatments
Don W. Cleveland, Ph.D.
Department Chair and Distinguished Professor UCSD

2:45 p.m.

Break/Exhibit Hall 

3:00 p.m.

Analytics Panel
Alper Kucukural, Ph.D. (UMass Chan Medical School)
Xue Wang, Ph.D. (Mayo Clinic)
Yan Asmann, Ph.D. (Mayo Clinic)
Richard Braatz, Ph.D

4:00 p.m.

Short talks selected from submitted abstracts 

5:00 p.m.

Closing Remarks/Adjourn

 

 

 

 

 

 

Mayo Clinic Florida
4500 San Pablo Rd
Kinne Auditorium
Jacksonville, FL 32224
United States

The course will take place in the Kinne Auditorium which is located on the 1st floor of the Cannaday Building.

Please visit HERE to access a campus map and additional information about the Mayo Clinic Florida campus.

Travel

Please note that this course does not have a designated room block. Please see below for hotel options that are near the Mayo Clinic Florida campus.

Residence Inn Jacksonville/Mayo Clinic Area
Distance to Mayo Clinic: 0.8 miles
Distance to Jacksonville International Airport: 27 miles
This hotel offers a daily shuttle to Mayo Clinic from 7am-5pm

Holiday Inn Express & Suites Jacksonville SE- Med Center Area
Distance to Mayo Clinic: 3.2 miles
Distance to Jacksonville International Airport: 26 Miles

The Ponte Vedra Inn & Club
Distance to Mayo Clinic: 5.3 miles
Distance to Jacksonville International Airport: 32 miles

Sawgrass Marriott Resort
Distance to Mayo Clinic: 7.6 miles
Distance to Jacksonville International Airport: 33.8 miles

One Ocean Resort
Distance to Mayo Clinic: 7.2 miles
Distance to Jacksonville International Airport: 25.2 miles

** All travel and lodging expenses are the sole responsibility of the individual registrant.**

 

Course Directors  

Nilufer Ertekin-Taner, M.D., Ph.D., is a neurogeneticist and behavioral neurologist at Mayo Clinic Hospital in Jacksonville, Florida. Her laboratory aims to discover and characterize genetic factors underlying the complex genetics of Alzheimer's disease (AD) and related neurodegenerative conditions.

Alfredo Quinones-Hinojosa, M.D., is a consultant and serves as chair of the Department of Neurologic Surgery at Mayo Clinic's campus in Florida, and he is recognized with the distinction of a named professorship, the William J. and Charles H. Mayo Professorship.

Himakshi Jhala, M.P.H., M.B.A., FACHE, is an Administrator at Mayo Clinic and works on driving innovative strategic initiatives focusing on discovery research, impactful translation, and synergistic growth areas that meet the unmet needs of the patients across the Mayo Clinic enterprise. She is board certified in healthcare management and is recognized as a Fellow of the American College of Healthcare Executives. 

 

Mayo Clinic Faculty  

               

Julie Allickson, Ph.D., is the Chief Technology Officer at Mayo Clinic’s Center for Regenerative Biotherapeutics and also directs Biomanufacturing and Process Development. She's a consultant for Laboratory Medicine and Pathology and an associate professor of regenerative medicine. Dr. Allickson focuses on product development for the center in line with Mayo Clinic's 2030 vision. With decades of experience, she specializes in Cell and Gene Therapies, Tissue Engineering, and 3D Bioprinting, aiming to position Mayo Clinic as a leader in regenerative medicine. She leads biomanufacturing strategy, new therapy introductions, and external relationships. With over 25 years in cellular therapy and regenerative medicine, Dr. Allickson has expertise in business management, strategic planning, and project management. She has worked in both industry and academia and was an executive officer at a cell banking company.

Yan Asmann, Ph.D.

                 

Minerva Carrasquillo, Ph.D., is an Assistant Professor in the Department of Neuroscience at Mayo Clinic Florida. She was born and raised in the beautiful Caribbean Island of Puerto Rico. She earned a PhD in Human Genetics from Case Western Reserve University and completed postdoctoral training at Johns Hopkins University and Mayo Clinic Florida. She now leads a research laboratory focused on the identification of genomic and environmental risk factors that may inform the development of effective therapies and accessible blood biomarkers for Alzheimer’s disease (AD), with special emphasis on underrepresented populations. She serves as Chair of the Department of Neuroscience Equity, Diversity and inclusion Committee, and as Co-Director for the Undergraduate Biomedical Research Internship (U-BRI) and Summer Undergraduate Research Fellowship (SURF) programs at Mayo Clinic Florida. Dr. Carrasquillo has authored/co-authored over 120 peer-reviewed publications on AD and related dementias. She is PI/co-Principal Investigator of 4 grants, including 2 large NIH/NIA grants that support the Mayo Advancing Research Equity in ADRD Study in Jacksonville (MAREAS-Jax) and the Centrally-linked longitudinal peripheral biomarkers of AD in multi-ethnic populations (CLEAR-AD) programs.

Margot Cousin, Ph.D.

Haidong Dong, M.D., Ph.D.

                 

Tsuneya Ikezu, M.D., Ph.D., is a Professor and Consultant in the Department of Neuroscience and Director of Molecular NeuroTherapeutics Laboratory at the Mayo Clinic in Florida.   Researchers in Dr. Ikezu’s lab are particularly interested in how the innate immune-related cells, extracellular vehicles (EVs), and molecules in the central nervous system (CNS) influence the pathology and progression of select neurodegenerative disorders such as Alzheimer’s disease (AD) and frontotemporal dementia. He has authored more than 140 journal articles, edited the textbook Neuroimmune Pharmacology and Therapeutics (Springer Nature) and served on several international boards including Associate Editor of Journal of Extracellular Vesicles and co-chair of ISEV EV in Nervous System Special Interest Group. Over his career, Dr. Ikezu has received Vada Kinman Oldfield Alzheimer’s Research Award (2000), Inge Grundke Iqbal Award from Alzheimer’s Association (2016), Jack Spivack Excellence in Neuroscience Award (2018), and Investigator of the Year Award from Mayo Clinic Florida (2023). Dr. Ikezu received his M.D. and Ph.D. from University of Tokyo School of Medicine and was Professor of Pharmacology and Experimental Therapeutics at Boston University School of Medicine (2010-2020). Dr. Ikezu will talk about development of EV-based biomarkers in neurodegenerative disorders.

Keith Knutson, Ph.D.

Xue Wang, Ph.D.

Jane Zhu, M.D., Ph.D.

 

Guest Faculty  

                     

Richard Braatz, Ph.D., is the Edwin R. Gilliland Professor of Chemical Engineering at MIT and the Director of the Center for Continuous mRNA Manufacturing where he conducts research in advanced biotherapeutics manufacturing systems. Dr. Braatz received an M.S. and Ph.D. from the California Institute of Technology and was the Millennium Chair and Professor at the University of Illinois at Urbana-Champaign and a Visiting Scholar at Harvard University before moving to MIT. Dr. Braatz has collaborated with more than 20 companies, has coauthored over 350 journal papers and three books, and has coedited two books on Digital Twins for Manufacturing. Dr. Braatz is a Fellow of IEEE, IFAC, AIChE, and AAAS and a member of the U.S. National Academy of Engineering.

                   

Kate Broderick, Ph.D., has more than 20 years of experience in the life science industry. A recognized vaccine expert, Dr. Broderick has a broad background product development in the nucleic acid therapeutic and drug delivery field. Dr. Broderick joined Maravai in 2022 as the Chief Innovation Officer and oversees the research and development of the company. Prior to joining Maravai, Dr. Broderick was the Senior Vice President, R&D. at Inovio Pharmaceuticals, running their R&D programs for a variety of DNA-based targets.  Dr. Broderick has served as a principal investigator for a variety of grants and awards from government agencies and non-profits, including the National Institutes of Health, Gates Foundation and CEPI. She received her PhD from the University of Glasgow in Scotland and completed her post-doctoral research at the University of California, San Diego. 

 

Susan Catalano, Ph.D.

 

Don W. Cleveland, Ph.D. (University of California San Diego)

 

Jeff Coller, Ph.D. 

 

Stanley Crooke, M.D., Ph.D. (n-Lorem Foundation), 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.

 

Patrick Finn, Ph.D.

 

Keith Gagnon, Ph.D. 

 

Andrew Geall, Ph.D., is the co-founder and Chief Development Officer at Replicate Bioscience, a San Diego based clinical-stage company pioneering novel self-replicating RNA (srRNA) technology for applications across infectious disease, immunology, and other therapeutic areas. He started his career in RNA vaccine in 2008, when he initiated and lead the self-amplifying RNA vaccines program at Novartis Vaccines. His clinical interest are in infectious disease, Replicate Bio just completed its 1st phase 1 clinical trial with a rabies vaccines and the data was published in Jan 2025 in Nature Communications.

                   

Ali Khademhosseini, Ph.D. (Terasaki Institute), is the CEO and Founding Director of the Terasaki Institute for Biomedical Innovation. Previously, he was a Professor at UCLA and Harvard Medical School, with faculty roles at Harvard-MIT HST, Brigham and Women’s Hospital, and the Wyss Institute. He has authored over 800 publications, holds more than 50 patents, and has an H-index of 193. His research has led to groundbreaking advances in biomaterials and hydrogel technologies for healthcare. He earned his Ph.D. in bioengineering from MIT and his MASc and BASc in chemical engineering from the University of Toronto. He has founded three companies, including Obsidio Medical (acquired by Boston Scientific), Omeat, and BioRAE.

 

Alper Kucukural, Ph.D. (UMass Chan Medical School)

 

Marlen Lauffer, M.D., is a senior researcher at the Dutch Center for RNA Therapeutics where she is responsible for identifying suitable candidates for individualized ASO treatments and overseeing the practical work at the lab in Leiden. She was trained as a medical doctor and human geneticist in Germany, Switzerland, Australia, and the UK. Marlen has been working on rare neurogenetic disorders for the past decade and has a special interest in neurodevelopmental disorders and neurodegenerative disorders in children. She is member of multiple international consortia working on individualized genetic therapy development and leading an international working group on patient identification with the N=1 Collaborative.

 

Jeff Milton, is Co-Founder and CTO of La Jolla Labs, a company developing technology for RNA therapeutics. Prior to this Jeff was Head of Data Sciences at Arcturus Therapeutics where he worked on several rare disease programs including both RNA-targeting and mRNA modalities. Jeff served as Director of Functional Genomics and Drug Discovery at Ionis Pharmaceuticals where he led teams in bioinformatics and screening software. Prior to this he worked in the Bioinformatics Department at Genentech. Jeff currently serves as an advisor to his alma mater, the Mellon College of Science at Carnegie Mellon University.

 

Jay Parrish

 

Xiling Shen, Ph.D., is a Professor and CPRIT Established Scholar in GI Medical Oncology and Co-Director of CRC Moonshot at MD Anderson and Chief Scientific Officer at Terasaki Institute for Biomedical Innovation. He was formerly the Director of the Woo Center for Big Data and Precision Health and Hawkins Family Professor at Duke University. He received his BS, MS, and PhD degrees from Stanford University and the NSF faculty career award at Cornell University. He was the steering committee chair of the NCI Patient-Derived Model of Cancer Consortium, co-chair of the NCI Tissue Engineering Consortium, cancer track chair of Biomedical Engineering Society 2019, and leader of the Chan-Zuckerberg Gut-Brain Atlas Program. He founded several biotech startups and raised more than $100M venture funding, translating several technologies into clinical and drug discovery pipelines. His lab studies novel cancer therapeutics from a systems biology perspective.

Natalie Silver, M.D., is Staff at the Cleveland Clinic Head and Neck Institute and ss the Director of Head and Neck Cancer Research. She is Principal Investigator (PI) in the Center for Immunotherapy and Precision Immuno-Oncology at the Cleveland Clinic Lerner Research Institute and an Assistant Professor at Case Western Reserve University. Dr. Silver is a head and neck ablative surgical oncologist and completed a research and clinical fellowship at UT MD Anderson Cancer Center. Dr. Silver’s translational research program at Cleveland Clinic is focused on developing therapeutic personalized mRNA nano-vaccines for head and neck cancer patients and exploring the role of intra-tumoral bacteria in immunotherapy response. Her goal is to translate discoveries from the lab to the clinic to improve patient outcomes. Dr. Silver’s research laboratory is funded by the National Institutes of Health (NIH), American Cancer Society and several foundation awards.

 

Blanton Tolbert, Ph.D.

           

Ken Yamada, Ph.D., is currently an Assistant Professor in RNA Therapeutics Institute at UMass Chan Medical School (2021-current) and is investigating transformative novel chemistry platforms for opening difficult extra-hepatic tissues to oligonucleotide-therapeutics intervention. His exploration with Nucleic Acid Chemistry started when he was a PhD student in Prof. Mitsuo Sekine’s lab, where he cultivated his discipline as a nucleic acid and oligonucleotide chemist. After his career path led to a postdoctoral position in Prof. Masad Damha’s lab at McGill University (2012-2014) and an Assistant Professor position in Prof. Fumi Nagatsugi’s lab at Tohoku University (2014-2017), he joined Prof. Anastasia Khvorova’s lab to explore his potential as a chemist to contribute to the siRNA therapeutics field. In a dynamic collaborative scientific culture in RTI, he established several innovative chemistry platforms that surpass current state-of-art siRNA technology. His inventions generated multiple patents, part of those licensed out to top-leading biotech companies.

 

Phillip Zamore, Ph.D.

 

Ran Zheng 

Accreditation Statement
In support of improving patient care, Mayo Clinic College of Medicine and Science is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC) to provide continuing education for the healthcare team.

Credit Statement(s):

AMA
Mayo Clinic College of Medicine and Science designates this live activity for a maximum of 12.50 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Other Healthcare Professionals:
A record of attendance will be provided to all registrants for requesting credits in accordance with state nursing boards, specialty societies or other professional associations.

For disclosure information regarding Mayo Clinic School of Continuous Professional Development accreditation review committee member(s) and staff, please go here to review disclosures.

Available Credit

  • 12.50 AMA PRA Category 1 Credit
  • 12.50 Attendance
 
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