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. 

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:

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 

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:

Hongene Biotech

NRPC25: https://www.nlorem.org/nano-rare-patient-colloquium-2025/ 

Rescue 7 is dedicated to serving the most vulnerable patient communities with care, compassion, and critical support. Inspired by the traditions of Fire Departments, Police Departments, and Military service worldwide, their programs go beyond emergency response. From patient transportation to sibling support initiatives, as well as emergency disaster relief efforts — Rescue 7 is there. Always ready and there when you need them.

Rescue 7 is founded by n-Lorem families. They will host the Family Club at the 2025 Nano-rare Patient Colloquium. Families are invited to stop by, hang out, or drop off their children for fun, firefighting-themed activities while caregivers attend the event. Get to meet the first responder volunteers, Luke Rosen, and Raena and Frank Vrtochnick in this episode of the Patient Empowerment Program Podcast!

Sarah and Ryan Marta, proud parents of three girls from Central Texas, began noticing that their middle daughter, Kinsley, was slow to reach mobility milestones in her early years. During a visit to a friend’s house, concerns about her vision were raised, prompting a check-up. What began as a single hospital visit quickly turned into many. An MRI revealed abnormal levels of white matter in Kinsley’s brain—leading to the devastating and confusing diagnosis of a nano-rare neurodegenerative disease called TUBB4A.

Looking high and low for help, they eventually discovered n-Lorem, a non-profit that discovers and develops personalized medicines for extremely rare individuals like Kinsley.

Kinsley is lively and cheerful with infectious laughter and a bright smile that lights up every room. She loves singing at the top of her lungs, swinging in the fresh air, and spending endless hours playing with her sisters and cousins. Kinsley uses a mobility walker to get around, but one of her biggest dreams is to run. When asked what she’d do after running, she simply says, “I’d run some more.”

On This Episode We Discuss:

2:35 - Early signs of Kinsley’s disease and her TUBB4A diagnosis

4:00 - Fight or flight – Sarah and Ryan did all they could to seek help

7:30 - Defining TUBB4A

9:40 - Kinsley's challenges today

11:00 - The impact rare diseases have on families

14:40 – Kinsley wants to do more, and an n-Lorem medicine provides optimism

Links: 2025 Nano-rare Patient Colloquium Agenda and Registration - https://www.nlorem.org/nano-rare-patient-colloquium-2025/ 

Donate to n-Lorem - https://www.nlorem.org/donate/

Hongene Biotech - https://hongene.com/ 

Emerging observations from our work at n-Lorem suggest that not all developmental delays are permanent. In the absence of structural damage—such as microcephaly, congenital deformities of the skull or bones, or organ malformations—there appears to be potential for improvement in movement disorders, cognition, autistic features, and more. These insights point to a level of resilience and plasticity in the central nervous system that may be greater than previously believed, and they are beginning to reshape how the scientific community understands the brain.

Links:

2025 Nano-rare Patient Colloquium Agenda and Registration - https://www.nlorem.org/nano-rare-patient-colloquium-2025/ 

Donate to n-Lorem - https://www.nlorem.org/donate/ 

Hongene Biotech - https://hongene.com/ 

At n-Lorem, every investment decision carries real weight by directly affecting the nano-rare patients we serve. We think very deeply about each decision of how to invest the limited and precious dollars that we have. But what about investing in basic research to advance antisense oligonucleotide (ASO) technology for the future? Certainly, most of the money should and is being put towards creating ASOs to help patients in need today. Meanwhile, it is also our belief that we must invest to continue innovating and, in this episode, we explore the research occurring at n-Lorem today that will lead to better treatments for more nano-rare patients tomorrow.

- Nano-rare patients need more from ASO technology

- Reasons n-Lorem are unable to help more patients

- Solutions to these challenges are possible and we know how to do it

Links:

Colloquium - https://www.nlorem.org/nano-rare-patient-colloquium-2025/

Donate - https://www.nlorem.org/donate/