BONUS The Path with Becky Quick: Head of the Class 3/20/26

Fri Mar 20 2026

Baby KJ was a pioneer before he was even a year old. Born in 2024 with a rare, life-threatening genetic mutation, Nicole and Kyle Muldoon’s son is the world’s first patient to receive successful personalized gene editing therapy. Doctors at the Children’s Hospital of Philadelphia (CHOP) and Penn Medicine collaborated to create a unique drug just for KJ, to treat his disorder known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. After spending the first months of his life in the hospital, KJ’s medical breakthrough came with his first dose in February 2025 when he was 6 months old. Now, a busy toddler and youngest brother, KJ is a piece of medical history – and he just started to walk. Join us in advancing awareness and understanding of rare diseases. Visit CNBC.com/Cures to access clips, resources, or to sign up for our weekly newsletter. Learn more about rare disease – and what to do in a diagnosis – at the National Organization for Rare Disorders: https://ra...

Summary

Squawk Pod – BONUS The Path with Becky Quick: Head of the Class – March 20, 2026

Episode Overview

This deeply moving episode of "The Path" series, hosted by Becky Quick for CNBC Cures, chronicles the extraordinary journey of the Muldoon family and their son, KJ – the first person in the world to receive a personalized CRISPR-based gene editing therapy for a rare genetic disorder called CPS1 deficiency. Through the voices of Nicole and Kyle Muldoon, listeners experience the harrowing uncertainties of rare disease diagnosis, the pioneering medical intervention at Children’s Hospital of Philadelphia (CHOP), and the emotional complexities of hope, risk, and resilience. The episode offers rare insight into the lived realities of rare disease families and trailblazing medical science.

Key Discussion Points & Insights

The Unexpected Birth and Diagnosis

Life-threatening Pregnancy & Emergency Delivery
- Nicole Muldoon describes a high-risk pregnancy culminating in a life-saving emergency C-section.
- “I told them, like, my kids need me, my husband needs me. Save us both… I wrote letters in case, but thankfully I woke up and was able to delete all of them.” (Nicole, 04:31)
KJ’s Immediate Health Crisis
- Within hours of birth, KJ’s severe symptoms led doctors to suspect a grave issue: his ammonia levels were astoundingly high.
- "Typical ammonia levels... are between 9 and 33. KJ's was up in the 1600s. So that's why they acted so quickly with the dialysis." (Nicole, 08:39)
Rare CPS1 Deficiency Revealed
- Official diagnosis: complete deficiency in CPS1, essential to the urea cycle.
- “We just hope it's not CPS1 deficiency... we're so sorry, it's a CPS1 deficiency.” (Kyle, 10:17)

Confronting Difficult Choices

Palliative Care vs. Hope
- Doctors initially recommend palliative, end-of-life care due to the grim prognosis.
- “We had to give him an opportunity to fight.” (Kyle, 11:28)
- “We fought to get him here because we love him, and out of love, we want to give him that opportunity to fight for himself.” (Nicole, 11:47)
Waiting for a Liver Transplant
- The only established cure was liver transplant, but KJ wasn’t immediately eligible – he needed to grow, and a donor match was required. (Nicole, 13:27)

The Gene Therapy Breakthrough

Approached for Pioneering Therapy
- The CHOP team (Dr. Rebecca Ahrens Nicholas and Dr. Kiran Musunuru) present a personalized gene editing solution.
- “Kind of far-fetched kind of stuff you hear about on, like, podcasts." (Kyle, 17:49)
How Base Editing Works
- Doctors use base editing to change the faulty DNA letter, delivered via lipid nanoparticles, aiming to restore enzyme function in KJ’s liver.
- “This could revolutionize the way we treat rare disorders for patients who have limited or no other options.” (Nicole, 16:53)
- Nicole likens it to fixing a typo: “The gene editing basically went into that sentence and input… the missing gene into his sentence or into his liver in the urea cycle.” (Nicole, 21:50)
Family Deliberation and Decision
- The Muldoons gather every specialist for a full-team Q&A session, balancing hope against unknown long-term risks.
- “When we made this decision, I was very gut. But thank God that we have Nicole on our team, dotting every I and crossing every T." (Kyle, 20:50)

The Infusion and Recovery

Infusion Day & Family Dynamics
- The Muldoons prepared their three other children as best as possible and created a hospital environment reminiscent of home.
- “Our oldest had an understanding of what that was… [the younger ones] were just kind of like, oh, KJ is getting a little procedure done today.” (Nicole, 23:45)
Watching for Signs of Success
- Early promising indicators were lab numbers (ammonia, labs), not just clinical appearance.
- “When those tests would come up on the app, we'd be like, oh my God, did you see like this number was this or this number was that.” (Kyle, 26:38)

Bringing KJ Home

Discharge After 307 Days
- KJ is released from CHOP with emotional fanfare, family, and community support (including a police escort home).
- “We spent almost a year in the hospital... we become so accustomed to juggling raising three children and having our fourth in the hospital.” (Kyle, 29:39)
The Weight of New Normals
- Both Nicole and Kyle share moments of profound gratitude, persistent anxiety, and the adjustment to life outside the hospital.
- “Every night before I would go to sleep, and the last thing I did before I went to sleep [was] to call and check in with the nurses... and that was the first time... he's here.” (Nicole, 30:41)

Reflections, Uncertainties, and Hopes

On the Future with Gene Therapy
- The therapy is life-changing, but not a cure—all eyes are on KJ’s evolving story, as he’s the first-ever case.
- “They don’t know. So he is the data… But the biggest tell right now is we take turns with lowering the medication dose that he’s on and upping the protein allowance.” (Nicole, 35:17)
- “None of that really matters to me because my son is doing all the things he should be doing... so cure or not, what the therapy has made his life significantly better.” (Kyle, 36:07)
Profound Impact & Broader Message
- Kyle and Nicole hope KJ’s case paves the way for further research and hope for other families.
- “Our biggest hope is that he represents the entirety of civilization and how people deserve something as simple as healthcare.” (Kyle, 37:22)
- “We agreed to the research because we wanted to help other families… so that we could help others.” (Nicole, 38:19)

Notable Quotes & Moments (with Timestamps)

On Parental Determination

“I fought my entire pregnancy to get him here… out of love, we want to give him that opportunity to fight for himself.” – Nicole (11:47)

On Being the First

“He is the data, which is good.” – Nicole (35:17)
“Some people will live their entire lives and maybe not make the impact that you made. That’s because you’re a fighter.” – Kyle (36:34)

On the Medical Team's Approach

“Dr. Aarons Nicholas was telling us these extraordinary things in the most humble way... you just keep doing what you’re doing and let us know, if this comes, we’ll be ready.” – Kyle (17:49)

On Returning Home

“That was his 307th day in the hospital, and he was finally set free.” – Nicole (29:25)
“Every night before I would go to sleep... was to call and check in with the nurses... and that was the first time I was like, oh, I don’t have to do this now. He’s here.” – Nicole (30:41)

On the Hope for Others

“We agreed to the research because we wanted to help other families… never knowing that it would work for kj.” – Nicole (38:19)

Key Timestamps

Intro & KJ’s Early Challenges: 01:03 – 08:39
Diagnosis and Life-or-Death Decisions: 09:51 – 13:27
Gene Therapy Introduction & Deliberation: 14:30 – 22:35
The Infusion Experience: 23:34 – 27:37
Discharge and Homecoming: 29:12 – 32:14
Long-term Prospects & Reflections: 35:00 – 38:19
Closing Reflections & Next Episode Preview: 39:05 – end

Episode Tone

Heartfelt, genuine, occasionally humorous amidst adversity, and thoroughly hopeful. The conversation blends clinical facts with powerful emotion, empowering listeners to witness both the scientific and personal sides of rare disease innovation.

For Further Support and Resources:
Becky invites listeners to reach out with thoughts or for resources, continuing the mission of building a community for rare disease families.

Next episode: Actress Selma Blair discusses her multiple sclerosis journey.

Transcript

Narrator/Announcer (0:00)

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Nicole Muldoon (1:03)

They wanted to initially start talking about comfort care, so end of life care.

Kyle Muldoon (1:07)

We had to give them an opportunity to fight.

Becky Quick (1:09)

What did you think when they brought up the idea of a gene therapy?

Kyle Muldoon (1:12)

Kind of far fetched kind of stuff you hear about on like podcasts.

Becky Quick (1:20)

I'm Becky Quick. This is the Path, a podcast series from cnbc. Cures about the people, the powerful communities and the pioneers of rare disease. Even the smallest pioneers.

Nicole Muldoon (1:36)

Hi. Hi cutie. Yay.

Becky Quick (1:41)

Who are all these people? KJ Muldoon or Baby KJ is the first person in the world to receive a personalized CRISPR based gene editing therapy. He is the whole data set. He's one of a kind and he is a success story of modern medicine. A hopeful sign for any of the 30 million Americans impacted by the thousands of rare diseases and conditions. Rare can mean a lot of things. These conditions can be misdirections in neuropathways, deficits of critical proteins or enzymes, sort of genetic typos, biological chances that impact small patient populations and 95% of rare diseases lack a cure. He looks like such a happy boy born with the Rare genetic deficiency CPS1. KJ was just hours old when it became clear that a severe enzyme deficiency was preventing his liver from working properly. This condition is so rare that it affects just one child out of every 1.3 million babies and most don't survive long enough for a liver transplant, which is the only known cure until KJ received a custom gene editing therapy from doctors at the Children's Hospital of Philadelphia before his first birthday he lost one shoe.