Walt Whitman’s 1855 poem, I Sing the Body Electric, is a celebration of the human body and its intrinsic connection to the universe. During that era, scientists were just beginning to understand that humans are, indeed, electrical beings. Whitman’s deliberate use of the word "electric" in his tribute to the human body was quite remarkable, as the term was not widely used at the time and electricity for human use was still in the experimental stage. With that in mind, the poem’s title serves as both a poetic and scientific statement.

Many have compared great science to art, but we believe it is more akin to poetry. A poet distills intricate concepts, stories, and emotions into the most concise, impactful expression—cutting through the noise to reach the heart and soul of the matter. Similarly, great scientists achieve this with their work. Together, scientists and patients contribute their own verses to the epic poem of understanding known as science.

This episode of the Patient Empowerment Program podcast is proudly sponsored by Hongene Biotech. With more than 26 years of experience in the nucleic acid industry, Hongene is a specialized CDMO and raw materials supplier committed to providing high-quality and high value products and services with the best in class lead times.

Make Hope Possible and donate, today www.nlorem.org/donate

Episode sponsor: Hongene Biotech https://www.hongene.com/

Join Stan Crooke, n-Lorem founder, CEO, and host of the Patient Empowerment Program Podcast, alongside Amy Williford, Sr. Director of Communications and Donor Relations, for a special episode.

In this post-colloquium Q&A, Stan recaps the 2024 Nano-rare Patient Colloquium and the progress n-Lorem has made as the organization nears its 5-year anniversary, shares his candid reflections on the event, and dives into additional thoughts and questions that weren’t covered.

Do you have additional questions? Email [email protected].

Question Bank:

Make hope possible with a donation.

This year’s Colloquium spanned two perfect autumn days in beautiful Cambridge, Massachusetts where hundreds of nano-rare community members from around the world gathered under a single roof to learn, connect, and support one another. We’re pleased to announce that the event welcomed over 750 attendees, both in-person and virtually. It’s each and every one of you who made the event such a success! For this special episode, we gathered the ‘Best’ clips from the event for you to learn from and enjoy.

To submit questions for our upcoming Q&A episode, email [email protected]. We can’t wait to hear from you!

Two years of treatment—two years of strength, courage, and blazing a trail for other nano-rare patients! 🎉 Susannah's story was the first shared on the Patient Empowerment Program podcast. Now, two years later, her father, Luke Rosen, and her physician, Dr. Jennifer Bain, detail Susannah’s remarkable progress since beginning regular treatments—highlighting improvements in her motor skills, cognition, energy, and more!

Susannah's Story (May 25, 2022)

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2024 Nano-rare Patient Colloquium

On This Episode We Discuss:

• 3:10 Susannah's KIF1A mutation

Take a glimpse behind the curtain as we offer you a sneak peek of the 2024 Nano-rare Patient Colloquium. We’re thrilled to welcome back longtime biotech journalist and current Features Editor of The Transmitter, Brady Huggett, to the podcast. In an interview with n-Lorem founder and CEO Stan Crooke, they preview the upcoming Colloquium, diving into key topics that will shape the conversation at the event and around nano-rare diseases in the year ahead.

2:21 How many Investigational New Drugs (INDs) has n-Lorem filed? 

5:22 How many patients treated with an n-Lorem ASO have been on therapy long enough to observe a benefit? 

6:50 What does it mean for a patient like Susannah who has experienced a significant decrease in behavioral arrest incidents since receiving treatment 

9:39 Providing ASOs to patients earlier 

13:25 INDs for the ‘n-of-few' 

23:15 Introducing Whole Genome Sequencing (WGS) for all newborns 

29:05 Stan’s perspective on leadership 

33:50 n-Lorem's goals for the 2024 Nano-rare Patient Colloquium

Biogen.com

Hongene Biotech is a producer of RNA building blocks – the first step for what’s possible in RNA drug-discovery and development. David Butler, Ph.D., Chief Technology Officer of Hongene, joins the Patient Empowerment Program to discuss why Hongene aims to help make RNA medicines accessible and affordable for patients everywhere, regardless of prevalence.

On this episode we discuss:

2:40 Hongene Biotech and what do they do

8:09 The early days of nucleoside building blocks and maturation of Hongene

12:00 Hongene is a strong supporter of n-Lorem. Why?

15:30 David’s origins in the chemistry field

17:38 Incremental progress that has been made in science and manufacturing

Links:

2024 Colloquium - https://www.nlorem.org/nano-rare-patient-colloquium-2024/

Make Hope Possible with a Donation - https://www.nlorem.org/donate/

Hongene - https://www.hongene.com/

On This Episode We Discuss:

• Questions we can answer and teach us about health and disease and opportunities to treat more common diseases

The knowledge we are gaining at n-Lorem has even more value than benefiting nano-rare patients and their families. These insights will enable scientists to discover new therapeutic targets for both common and rare diseases, fundamentally changing the way we approach health and disease.

On This Episode We Discuss:

• Two most common words in medicine: Health and Disease

Paul Compton and his wife Andrea chose to start a family through adoption. Unknown to anyone, one of their adopted boys, Taeson, was the inheritor of DRPLA, a progressive brain disorder caused by a mutation in the ATN1 gene. Determined to find a treatment and advocate for their son and others with the disease, the Comptons established CureDRPLA. In early 2024, Taeson received his initial treatment with an ASO discovered and developed by n-Lorem. Paul discusses his family’s journey and shares his observations of Taeson's progress since beginning treatment.

In This Episode We Discuss:

3:05 – Paul's origins in business and finance

4:32 - Investment banking is different than traditional banking

7:27 – Opposites attract – Meeting his wife, Andrea, and starting a family through adoption

10:06 – Paul’s son, Tayson, has a progressive brain disorder known as DRPLA, which is caused by a mutation in the ATN1 gene; Discovering the idea of n-Lorem

16:03 – Tayson’s journey to a diagnosis

23:00 – The deterioration of his abilities over the years

25:00 – The progress Paul has observed in his son since initial treatment

Links:

Take our survey and receive an n-Lorem Store coupon code

Register for the 2024 Nano-rare Patient Colloquium / October 30-31, 2024

Make Hope Possible with a donation

Mutations affecting ion channels are the most common cause of 'applications for treatment' submitted to n-Lorem. These channels regulate the passage of essential electrically charged ions, like sodium, potassium, calcium, and chloride, into cells. Alas, ion channels pose a significant challenge in the ASO discovery process, as there is little room for error due to the need for highly allele-selective ASOs to achieve success. Let’s 'dive into the channel' and explore the complexities of treating patients with ion channel mutations.

Recently a report on one of our patients with an ion channel mutation was published in Endpoints News highlighting the power of our technology for these disorders. Check it out by clicking the link below.

Endpoints News (endpts.com) A teenager faced constant seizures. Could a drug developed just for him stop them?

Podcast Awards - The People's Choice

Survey – Patient Empowerment Program Podcast

Register for the 2024 Nano-rare Patient Colloquium

On This Episode We Discuss:

• Defining Ion Channels and Ions