Miracles of Science #2: Induced Pluripotent Stem Cells

A nano rare diagnosis does not just affect one person. It reshapes an entire family.  Realities of the Nano-rare takes you inside that experience. Hear directly from n-Lorem COO and nano rare mom Sarah Glass as she joins host Stan Crooke to share the day-to-day realities of raising her son Ethan and the ripple effects on their whole family, including his older sister.  Discover how they have adapted, what they have learned, and how they have built a new normal.  Tune in to this powerful two-part series featuring both Sarah and Oliver Glass.    On This Episode We Discuss:  5:08 – Introduction to Sarah Glass and her family's Nano-rare experience  9:00 – Initial challenges of accurately identifying Sarah's son Ethan had a Nano-rare disease  11:30 – Symptoms and telltale signs that there was something else going on with Ethan  14:45 – Identifying that Ethan has DYRK1A  17:20 – Challenges of finding solutions post-diagnosis  20:20 – Navigating Sarah's son's DYRK1A with her unaffected daughter  23: 04 – What's a good day with DYRK1A?  27:01 – What's a bad day with DYRK1A?  34:30 – Managing frustrations of taking care of someone with a Nano-rare disease  37:05 – Compromises in professional life  41:00 – Planning for contingencies   43:10 – Unexpected tender moments of raising someone with DYRK1A  47:30 – Advice for managing expectations, future plans, and working with others    Make sure to check out our sponsor Chemgenes!

Welcome to part two of our series that pulls back the curtain on the scientific and medical breakthroughs that make n-Lorem possible. We call them miracles and without them, n-Lorem would not exist today. Every day, our patients benefit from what can only be described as miracles of science. Not the fairytale kind. Not wishes granted overnight. These miracles are earned by humans. Built over centuries by scientists whose discoveries stacked, evolved, and refined over time. They are the result of human curiosity, persistence, and an ever-deepening understanding of biology, and they’ve fundamentally changed how we see the world and treat disease. Today’s miracle: Induced Pluripotent Stem Cells or iPSCs iPSCs are a truly transformative scientific breakthrough that are changing what’s possible for nano-rare patients! They allow us to study, test, and personalize treatments in ways that were unimaginable not long ago. For example, they enable n-Lorem to take easily obtained cells, such as skin cells, grow them and then reprogram them into entirely different cell types of interest, like neurons, cells that exist within the brain and would otherwise be inaccessible without invasive procedures. Incredible!   On This Episode We Discuss: 0:53 – What are induced pluripotent stem cells (iPSCs) why they are essential to n-Lorem and personalized medicine  4:25 – The origins of iPSCs began with curiosity and the discovery and study of cells  10:19 – Cell differentiation: How a single cell (fertilized egg) can create such a diverse universe of other types of cells  18:45 – Terminal differentiation: The final stage of a cell's development reaching its final form  21:45 – The iPSC breakthrough and the doors they’ve opened for treatments     

On This episode We Discuss: 2:30 – Andrew realized that finance plays a very large role in drug development and is an obstacle in for rare disease patients getting the treatments they need 4:30 – The triple whammy of drug development 6:22 – Economic decisions when lives are at stake; Balancing the ethical considerations of medicine and finance 12:17 – Small biotech companies are better for innovation, and a distributed investment process is more productive; Innovation comes from orthogonal thinking 22:40 – Very small prevalence disease drug programs tend to get terminated at commercial organizations; Movement toward finding a way to create a commercial path for extremely rare disease drugs 25:45 – FDA draft guidance for potential commercialization of nano-rare medicines; Where the guidance may lead; Value of a statistical life 30:32 – The cost of a nano-rare patient’s care; Courageous moves of the FDA 35:00 – Regulatory processes merging with the advances of science while still protecting patients   Andrew W. Lo is the 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. His healthcare-related research interests include: new financial engineering tools and business models for drug and device development and healthcare delivery, especially for rare and ultra-rare diseases; statistical methods for incorporating patient preferences into the drug approval process; predicting clinical trial outcomes via machine learning techniques; and novel reimbursement models for creating a robust gene and cell therapy ecosystem. He is a co-founder of BridgeBio Pharma, QLS Advisors, Quantile Health, and Uncommon Cures; a director of AbCellera, Atomwise, BridgeBio, Uncommon Cures, and Vesalius; and a member of the advisory board to the American Cancer Society’s BrightEdge Impact Fund. Lo received his B.A. in economics from Yale University and his A.M. and Ph.D. in economics from Harvard University.   Patient Empowerment Program host: Stan Crooke, n-Lorem Founder and CEO

In this video message, Chairman Stan Crooke reflects on the close of 2025, six years after founding n-Lorem. He shares his gratitude for the community that made it the organization’s most successful year to date and offers perspective on how progress across every area of the foundation is carrying n-Lorem forward into 2026. Read the 2026 Chairman Letter

Today, we’re launching a NEW SERIES that pulls back the curtain on the scientific and medical breakthroughs that make n-Lorem possible. We call them miracles and without them, n-Lorem would not exist today. Every day, our patients benefit from what can only be described as miracles of science. Not the fairytale kind. Not wishes granted overnight. These miracles are earned by humans. Built over centuries by scientists whose discoveries stacked, evolved, and refined over time. They are the result of human curiosity, persistence, and an ever-deepening understanding of biology, and they’ve fundamentally changed how we see the world and treat disease. Today’s miracle: Genomics. Coming next: iPSCs, the RNA World, and Antisense Technology. The breakthroughs that pushed possibility even further. ------ The Patient Empowerment Program podcast is hosted by n-Lorem Founder and CEO, Stan Crooke. Dr. Crooke recently joined the advisory board of the CNBC Cures, an initiative bringing together families, doctors, investors and regulators with one goal in mind: helping to raise awareness of, and improve patient outcomes for, the 30 million Americans suffering from rare diseases. You can join the CNBC Cures Newsletter here: https://www.cnbc.com/cnbc-cures-newsletter/ Today's sponsor is ChemGenes: https://www.chemgenes.com/ 

Your questions, answered. n-Lorem CEO, Stan Crooke, answers questions directly from the nano-rare community in this special Q&A episode. How often is Drug safety monitored? When is an existing ASO made available to others? Not capping accepted applications and more. Do you have additional questions? Email [email protected].   On This Episode We Discuss: 5:40 - What is the frequency range of intrathecal and/or LP administrations? Do the treatments not typically cross the blood brain barrier? 7:06 - How do you administer the ASO to animals - is it according to how you plan to administer to the patient? 7:36 - How do you evaluate the potential on-target toxicity if the nonclinical species does not contain the targeted sequence? 9:35 - How often is safety monitored? Is it only monitored quarterly at the Data/Drug Safety Monitoring Board (DSMB) meetings? 11:03 - How long after an ASO is developed for one child, is it offered to other children? Of course, we know that they will have to submit an application. Does the ASO become potentially available for other children after the first child's 2-year trial is over? Or is it potentially available after just a few doses to confirm safety and efficacy? 14:18 - Are there plans to commercialize drugs for the nano-rare? 16:27 - What proportion of institutions elect to use external / centralized institutional review boards (IRBs)? 18:21 - Will there be a cap of accepted applicants from n-Lorem? The demand will only grow and there’s a fear for some families that there’s a chance of n-Lorem capping the number? 19:44 – Once we have our neurologist team working on getting our child accepted, what else do we need to do? What I guess is our next step? Is there anything we should do in the meantime? 20:31 – What should a parent expect once their application has been accepted to n-Lorem? 21:19 – There are widely varying timelines regarding this process. What as a parent should I expect regarding timing? 22:30 – There are companies and academic scientists that claim to provide ASOs for a fee. What do you think about these providers? 23:40 – Insights: What is n-Lorem learning that will be broadly applicable to the scientific community; Dreaming bigger and hoping for more

In this special Best Of episode, we revisit some of the powerful moments from the 2025 Nano-Rare Patient Colloquium—an extraordinary gathering that brought together more than 875 patients, family members, scientists, physicians, advocates, and industry leaders from around the world, both in person and virtually.  This episode highlights moving patient stories, thought-provoking scientific conversations, and community-led discussions that reflect the momentum building across the nano-rare space. Together, these voices showcase the growing impact of personalized experimental medicines and the shared determination to turn possibility into progress.  From inspiration to action, this Best Of captures the heart of the Colloquium—and the collective commitment to creating a brighter future for individuals and families living with nano-rare diseases.    Recap page:  https://www.nlorem.org/nano-rare-patient-colloquium-2025/    Colloquium YouTube Playlist (alternative to recap page):  https://youtube.com/playlist?list=PLrDVyc3t26FxvnqoiApY_Qw1_weTAQ4MS&si=EWBvQ0ZdDH-Rq1mJ 

A conversation with Natacha Gassenbach, 2025 Hero of n-Lorem and Biogen leader. She shares Biogen’s decision to become a founding donor of n-Lorem, the impact of the Nano-Rare Patient Colloquium. Natacha also explores “the movement for nano-rare” and a shared vision of tackling difficult challenges to drive meaningful change.   Holiday Ornament: https://www.nlorem.org/holiday-ornament-fundraiser/   On this episode: 1:18 – Natacha introduction 3:00 – Biogen’s path to becoming a founding donor of n-Lorem and giving back to the community 4:57 – Why Natacha and Biogen invested in n-Lorem and convincing new individuals to buy into the mission 7:19 – Biogen has hosted the n-Lorem Nano-rare Patient Colloquium since 2023 9:40 – Biogen and n-Lorem share the connection of tackling difficult tasks to make a difference in the world 15:05 – A movement for nano-rare the possibilities it may bring 17:45 – Taking advantage of nano-rare learnings and implementing them into drug discovery for larger patient populations

Meet Grace Hoyt — a bright, witty high schooler with a passion for singing, art, and romantasy audiobooks. Grace also happens to live with a rare disease (FLVCR1) that has left her legally blind, unable to feel pain, and unsteady on her feet. On her 13th birthday, Grace received a first-of-its-kind ASO medicine designed by n-Lorem to preserve her remaining vision. Today, she can still read snippets of her beloved “book children” — the physical versions of her favorite audiobooks — and, importantly, recognize and assess her sometimes life-threatening skin infections, a skill that could save her life.

As the 2025 Nano-rare Patient Colloquium approaches, we welcome back Transmitter Features Editor Brady Huggett to guest-host this episode of the Patient Empowerment Program podcast. Brady sits down with n-Lorem founder and CEO, Dr. Stan Crooke, to reflect onto reflect on the strides made over the past year—insights that will help shape the discussions at this year’s Colloquium. On This Episode We Discuss: 2:10 How n-Lorem decides to give a medicine specifically designed for one person to another individual 8:10 n-Lorem built systems to be able to treat more than one patient with a single drug 11:08 n-Lorem creates medicines for nano-rare genetically caused forms of ALS 15:14 New suggestions about the plasticity of the central nervous system and the future of medicine 19:40 Why science isn’t in a good place and will experience lasting repercussions 25:00 The Colloquium will focus on what we’ve learned at n-Lorem and what was once thought to be impossible is possible 27:34 An update on the quantity of INDs filed and patients treated with an n-Lorem ASO 28:80 The benefits of implementing whole genome sequencing (WGS) at birth 30:37 What surprises have emerged throughout the organizational journey of n-Lorem   NRPC25 registration: https://www.nlorem.org/nano-rare-patient-colloquium-2025/    Sponsors ChemGenes: https://www.chemgenes.com/  Honegene: https://www.hongene.com/en