We're addressing perhaps the most difficult decision n-Lorem has to make in our work, declining a patient application. Today, our acceptance rate is ~59%. These decisions are made with heavy hearts at n-Lorem, and we are aware of how heartbreaking the decision to decline an application may be for a patient and their family. In this episode, we'll discuss the main reasons a patient's application may be denied, including safety, geography, and other factors.

On This Episode We Discuss:

• 1:43 – Non-technical issues: Outside of USA, inability to find treating physician, patient population size, if the patient is being treated by another experimental medicine, including repurposed drugs

• 7:15 – Definitions to better understand the conversation: Heterozygous mutation, haploinsufficiency

• 9:40 – n-Lorem must understand the cause of the disease, the nature of the mutation, and n-Lorem must be confident that the ASO designed would deliver value to the patient

• 12:35 – n-Lorem must be confident that only a single gene is causing phenotypes, and that the target is not a null mutation

• 13:37 – n-Lorem may accept patients, but then discontinue a program if the patient cells are unable to be grown for lab use and testing

• 14:56 – For patients requiring an allele-selective ASO, n-Lorem must identify non-pathogenic mutations to attempt to create an ASO capable of helping the patient

Links:

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

Sponsors:

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

GondolaBio - https://gondolabio.com/

A lot can happen in a year, and at n-Lorem, this past year has brought important milestones, new developments, and plenty to discuss at the upcoming 2026 Nano-rare Patient Colloquium.

In this special Colloquium preview episode, Brady Huggett, editor-in-chief of Asimov Press and longtime moderator of the Colloquium’s patient experience panel, returns to sit down with n-Lorem CEO Stan Crooke. Together, they reflect on the past year, explore some of the topics likely to take center stage, and preview what attendees can expect at the 2026 Nano-rare Patient Colloquium.

On this episode:

2:28 – What is a day in the life of an n-Lorem research team member?

9:08 – How many ASOs are required to move into tolerability studies and what may cause a program to be terminated

12:05 – Improvements in efficiencies, bringing down costs, and growth have allowed n-Lorem to respond to the extraordinary demand

13:14 – Discussing the creation of individualized ASOs for two boys with SCN2A mutations and their potential to help others with the same mutation and single nucleotide variant

25:00 – Thoughts on the FDA’s Plausible Mechanism Framework

29:00 – Commercial opportunities will not alter the n-Lorem charitable arm but will provide sustainable revenues to charitably treat more patients

36:05 – What are the Limits of Hope and expanding those limits

37:54 – Empathy is an expanded sense of self, and a sphere of oneness is felt at the Colloquium

Links:

2026 Nano-rare Patient Colloquium

Donate to n-Lorem

Episode and NRPC Gold Sponsors:

ChemGenes

GondolaBio

Hongene Biotech

Sally Jackson is a former actress, cookbook co-author, and mother of Susannah. In this Realities of the Nano-rare episode, Sally lets us into her family’s bubble, speaking candidly about the deeply complex and often terrifying realities of navigating KIF1A-associated neurological disorder. From helping Susannah's sibling understand the harsh consequences of her disease to confronting distress, uncertainty, and the profound unfairness of it all, Sally shares the difficult truths her family has faced and continues to carry and fight through. On this episode: 4:00 – Sally and her husband's story first began in a college acting class 9:55 – Forming a career alongside celebrity chef Bobby Flay 13:30 – Family of four including Nat and Susannah 16:55 – Early abnormalities and a diagnosis initially withheld from the family 22:50 – The fight for Susannah and others with KIF1A post-diagnosis 29:24 – Dealing with the seriousness of a degenerative condition while trying to live a normal life 34:00 – Enduring relentless seizures 38:31 – The reality that not even doctors know what's best and often experiment to attempt to relieve rare disease symptoms, and the unfairness of rare disease 45:00 – Informing siblings about the severity and consequences of rare disease 57:00 – Changing the course of their life to advocate to the fullest extent 1:02:25 – After the long fight, there was an ASO 1:07:30 – After years of treatment, Susannah had to stop, a crushing blow that was followed by worsening symptoms before once again resuming treatments Links:

Nano-rare Patient Colloquium 2026

Support n-Lorem with a donation

Today's Sponsor - Hongene