By: Siri Chundru

24 December 2025

In May of 2025, doctors successfully cured the rare genetic disorder carbamoyl phosphate synthetase 1, or CPS1 deficiency, for the first time in a 9-and-a-half-month-old baby named K.J. Muldoon. I, along with many others, saw the breakthrough as a beacon of hope for the millions of people worldwide born with devastating genetic conditions. It felt like proof that science could finally offer more than management or survival.

CPS1 deficiency is a rare and life-threatening disease that prevents the body from breaking down proteins, causing toxic levels of ammonia to build up in the blood. For infants born with the disorder, the risk is extreme. Nearly half do not survive early childhood.

Using CRISPR, a gene-editing tool, scientists developed targeted proteins that altered K.J.’s DNA by correcting the defect in specific liver cells responsible for the condition. Through consistent dosing, doctors were able to rewrite the faulty genetic code and cure him in six months. K.J.’s story shook the medical world. It showed the potential of personalized gene therapy and made curing genetic diseases feel closer than ever.

But the story also revealed a darker reality.

Personalized gene therapies can cost up to 4.5 million dollars per patient. For every family like K.J.’s that gains access to a cure, there are millions who never will. A treatment that exists only for those who can afford it is not a solution. It is a privilege.

Medical breakthroughs are often celebrated as victories for humanity, but we rarely stop to ask who actually benefits from them. When life-saving treatments are priced so high that most people cannot access them, innovation alone does not improve public health. Instead, it risks deepening the divide between those who can afford care and those who cannot.

This problem is not limited to gene therapy. From insulin to cancer drugs to vaccines, effective treatments have existed long before they reached the people who needed them most. According to the World Health Organization, at least half of the world’s population still lacks access to essential health services, even when treatments are available. Science cannot change lives if it never reaches the people it was meant to help.

As students growing up in a world of rapid scientific progress, we are taught to celebrate innovation. And we should. But we should also be taught to question it. If medicine continues to focus more on what is possible than on who can access it, progress becomes something to admire from a distance rather than something that actually improves lives.

A medical breakthrough should not be measured by whether it can cure one patient under ideal conditions. It should be measured by whether it can reach the people who need it most. Until access becomes just as important as discovery, these miracles will remain out of reach for much of the world.

We cannot treat access as an afterthought. If scientific progress is truly meant to serve humanity, then affordability, distribution, and equity must be part of the conversation from the very beginning, not something addressed years later. We should be asking harder questions about who benefits from innovation, pushing institutions to value accessibility alongside discovery, and using our voices to demand a future where cures are not reserved for the few who can pay for them. 

K.J.’s recovery gives us hope, but real progress will only come when stories like his are no longer the exception.

Cornetta, Kenneth, et al. "Gene therapy access: Global challenges, opportunities, and views from Brazil, South Africa, and India." Molecular Therapy, vol. 30, no. 6, 1 June 2022, pp. 2122-29. PubMed Central, pmc.ncbi.nlm.nih.gov/articles/PMC9171243/. Accessed 24 Dec. 2025.

"Decoding the high costs and potential impact of emerging gene therapies." Kaiser Permanente, 27 Jan. 2025, business.kaiserpermanente.org/georgia/healthy-employees/pharmacy/gene-therapy. Accessed 24 Dec. 2025.

"Essential medicines." World Health Organization, 25 Sept. 2024, www.who.int/news-room/fact-sheets/detail/essential-medicines. Accessed 24 Dec. 2025.

Murdock, Andy. "First Patient Treated with Personalized CRISPR Therapy, Developed in Just Six Months." Innovative Genomics Institute, 15 May 2025, innovativegenomics.org/news/first-patient-treated-with-on-demand-crispr-therapy/. Accessed 24 Dec. 2025.

"Universal health coverage (UHC)." World Health Organization, 5 Dec. 2025, www.who.int/news-room/fact-sheets/detail/universal-health-coverage-%28uhc%29. Accessed 24 Dec. 2025.