Patient Empowerment Program: A Rare Disease Podcast

Miracles of Science #2: Induced Pluripotent Stem Cells

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 
 
 

Where Emerging FDA Draft Guidance may Lead for Nano-rare Patients

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

Reflections from the n-Lorem Chairman | 2026

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

Miracles of Science: Genomics

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.
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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: 2026 Q&A

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 podcast@nlorem.org.
 
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

Best of the 2025 Nano-rare Patient Colloquium

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 

Natacha Gassenbach of Biogen: 2025 Hero of n-Lorem

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

Seeing with Grace: A Teen’s Journey with a Rare Vision-Affecting Disease

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.

Sneak Peak of the 2025 Nano-rare Patient Colloquium

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 

A Conversation with the 2025 Heroes of n-Lorem

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. On this episode of the Patient Empowerment Program podcast, we welcome three of our five 2025 Heroes for a conversation about their inspiration to take action:
- Wendy Erler, Senior Vice President, Patient Affairs, Sarepta Therapeutics
- Richard S. Geary, Ph.D., Chief Development Officer, Executive Vice President of Development, Ionis Pharmaceuticals
- Scott Henry, Ph.D., DABT, SVP, Nonclinical Development, Ionis Pharmaceuticals
 
 
Sponsors:
ChemGenes
Hongene Biotech
 
NRPC25: https://www.nlorem.org/nano-rare-patient-colloquium-2025/