2nd Annual
Nano-rare
Patient Colloquium
2024
October 30-31, 2024
Boston Marriott Cambridge
Cambridge, Massachusetts
We are delighted to share that 750 people joined us, both in-person and virtually. Our heartfelt thanks go out to the nano-rare community for making this event a success!
About the Event
During the 2024 Nano-rare Patient Colloquium, we reflected on n-Lorem’s progress as our foundation continued to grow, shared new developments, and presented our vision for the future. We explored the knowledge we had gained and outlined our processes that allowed us to efficiently develop optimal personalized ASO medicines. Our patient families shared their journeys through diagnosis and treatment, and our partners discussed how supporting nano-rare initiatives had enriched their company cultures. We also heard from our supporters about the growing challenges as more nano-rare patients sought help from n-Lorem. Additionally, research physicians from across the country provided insights into the latest findings on neurological diseases—and much more!
The event was an extraordinary display of solidarity and inspiration. We learned, laughed, connected, and even shed a few tears, emerging as a stronger and more united nano-rare community.
2024 Nano-rare Patient Colloquium
Day 1 — October 30, 2024
Presentation
Providing hope for a better future, one nano-rare patient at a time
Stanley T. Crooke, M.D., Ph.D.
Founder and CEO, n-Lorem
Patient Panel:
Hope realized; The culmination of effort, perseverance and belief in a better outcome
Ryan’s journey and clinical experience
Our pioneering patients are paving a pathway for more nano-rare patients, providing valuable information on health and disease and experiencing, for the first time, targeted therapeutic intervention for their genetic conditions. Please join us as we hear their diagnostic stories from the patients and their families. We will follow these with case reports on these patient’s clinical experiences presented by the physicians who are so committed to caring for these patients.
Moderator
Brady Huggett
Features Editor at The Transmitter
Panelists
Ryan Strankowski
n-Lorem Patient
Nelson Leung, M.D.
David L. and Colleen B. Kessenich Professor of Multiple Myeloma, Consultant in the divisions of Nephrology and Hypertension, and Hematology in the Department of Internal Medicine at the Mayo Clinic
Patient Panel:
Hope realized; The culmination of effort, perseverance and belief in a better outcome
Connor’s journey and clinical experience
Our pioneering patients are paving a pathway for more nano-rare patients, providing valuable information on health and disease and experiencing, for the first time, targeted therapeutic intervention for their genetic conditions. Please join us as we hear their diagnostic stories from the patients and their families. We will follow these with case reports on these patient’s clinical experiences presented by the physicians who are so committed to caring for these patients.
Moderator
Brady Huggett
Features Editor at The Transmitter
Panelists
Kelley Dalby
n-Lorem Patient Mother, Director of Natural History and Diagnostics in Epilepsy at Praxis Precision Medicines
Olivia Kim-McManus, M.D.
Associate Clinical Professor, UC San Diego School of Medicine Dept of Neurosciences, Pediatric Neurologist and Epileptologist, Rady Children’s Hospital Precision Therapeutics Neuro-Interventional Program Director
Presentation
Discovering patterns: Learning from ‘n-of-few’ clinical trials in neurology
Jennifer Bain, M.D., Ph.D.
Associate Professor of Neurology and Pediatrics, Columbia University
Patient Panel:
Hope realized; The culmination of effort, perseverance and belief in a better outcome
Susannah’s and Sloane’s journeys and unique clinical experiences
Our pioneering patients are paving a pathway for more nano-rare patients, providing valuable information on health and disease and experiencing, for the first time, targeted therapeutic intervention for their genetic conditions. Please join us as we hear their diagnostic stories from the patients and their families. We will follow these with case reports on these patient’s clinical experiences presented by the physicians who are so committed to caring for these patients.
Moderator
Brady Huggett
Features Editor at The Transmitter
Panelists
Luke Rosen
n-Lorem Patient Father, Co-founder and Board Chair of KIF1A.ORG, Firefighter in the Sag Harbor Fire Department
Megan Hedstrom
n-Lorem Patient Mother
Jennifer Bain, M.D., Ph.D.
Associate Professor of Neurology and Pediatrics, Columbia University
Day 2 — October 31, 2024
Presentation:
From dream to reality: The establishment of a scalable solution for the treatment of the nano-rare
Sarah Glass, Ph.D.
Chief Operating Officer, n-Lorem
Presentation:
ASO design, discovery and research innovation in ASO technology
Konstantina Skourti-Stathaki, Ph.D.
Director, ASO Design and Discovery, n-Lorem
Presentation:
Efficient nonclinical development processes leading to successful treatment of nano-rare patients
Julie Douville, Ph.D.
Executive Director, ASO Discovery and Development, n-Lorem
Presentation:
n-Lorem’s clinical experience paves the way for precision medicine using antisense oligonucleotides
Laurence Mignon, Ph.D.
Executive Director, Clinical Development, n-Lorem
Panel:
Building a nano-rare network and managing institutional challenges
Creating, developing and providing an individualized/personalized ASO medicine for an individual patient is only part of the challenge that faces n-Lorem and our patients today. Our physicians and institutional partners are key to ensure that not only are our patients cared for, but that they can access the treatments we offer. We are continually expanding our network to bring more physician and institutional partners together to work with us in getting our nano-rare patients the treatments that could change their lives.
Moderator
Elizabeth Berry-Kravis, M.D., Ph.D.
Professor of Pediatrics and Neurological Sciences at Rush University Medical Center in Chicago
Panelists
Margot A. Cousin, Ph.D.
Assistant Professor of Medical Genetics in the Dept. of Molecular Medicine, Director of the N-of-1 Therapeutics Program in the Center for Individualized Medicine, and a KL2 Scholar through the Center for Clinical and Translational Science at Mayo Clinic
Luke Hamilton, MS
Professor of Pediatrics and Research at the TCU Burnett School of Medicine, Research Data Science and Analytics team leader at Cook Children’s Medical Center
Horacio Kaufmann, M.D.
Axelrod Professor of Neurology, Professor of Medicine, and Professor of Pediatrics at New York University Grossman School of Medicine
Julie Ziobro, M.D., Ph.D.
Assistant Professor of Pediatrics in the Division of Pediatric Neurology at the University of Michigan
Panel:
Innovative ways to support nano-rare
As n-Lorem scales to meet an ever-growing demand, new challenges arise. Many of these challenges will take innovative solutions and support from leaders and experts in a variety of different fields. Together as a community, we are creating a path forward for nano-rare patients and changing the way that genetic diseases are treated.
Moderator
Natacha Gassenbach
Head of Corporate Affairs, Chief Communications Officer, Biogen
Panelists
Paul Compton
Chairman of Investment Banking, Barclays
Andrew Lo, Ph.D.
Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management, director of MIT’s Laboratory for Financial Engineering, and principal investigator at MIT’s Computer Science and Artificial Intelligence Laboratory
Alan Lotvin, M.D.
Chief Executive Officer, Sequel MedTech LLC.
John Maraganore, Ph.D.
CEO and Principal, JMM Innovations LLC.
David A Pearce, PhD
Chair, International Rare Disease Research Consortium
Presentation
New business models and financing strategies for rare diseases
Andrew Lo, Ph.D.
Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management, director of MIT’s Laboratory for Financial Engineering, and principal investigator at MIT’s Computer Science and Artificial Intelligence Laboratory
Panel:
What it means to be a partner with n-Lorem for nano-rare
The challenges presented by nano-rare patients are far too complex for n-Lorem to solve even a fraction of the issues without support. Thus, our goal from inception has been to build as broad and effective a network of contributing stakeholders. In this panel, our partners in our community of care discuss what it means to be a partner with n-Lorem, including how partnering with n-Lorem provides their employees with a feeling of purpose and what more their organization can do to support nano-rare.
Moderator
Sarah Glass, Ph.D.
Chief Operating Officer, n-Lorem Foundation
Panelists
Kurt Andersson
OligoProduct Specialist Genomic Medicine, Cytiva (Retired)
David Butler, Ph.D.
Chief Technology Officer, Hongene
Tammy Cooper
President, Nitto Avecia
Kate Dawson, M.D.
Senior Vice President, Therapeutics Development Unit, Biogen
Stephen Wilson
General Manager, Charles River Senneville
Heroes of n-Lorem Awards
The ‘Hero of n-Lorem’ Award is a recognition given to an individual volunteer or organization that has made substantial contributions to n-Lorem and our nano-rare patients. This honor recognizes our ‘unsung heroes’, those who have gone the extra mile, taken time away from their schedules and families and leveraged their expertise and contacts to support n-Lorem’s mission to provide hope and potential help to nano-rare patients.
Awardees
Kurt Andersson
OligoProduct Specialist Genomic Medicine, Cytiva (Retired)
Eugene Schneider, M.D.
Executive vice president and chief clinical development and operations officer, Ionis Pharmaceuticals
Neil Shneider, M.D., Ph.D.
Claire Tow Associate Professor of Motor Neuron Disorders and the Director of the Eleanor and Lou Gehrig ALS Center at Columbia University