Weekly Rundown: The first personalized CRISPR therapy

Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.

The first personalized CRISPR therapy

While CRISPR-based therapies exist for people with genetic disorders like sickle cell disease, researchers recently reported the first personalized “N-of-1” CRISPR therapy for KJ Muldoon, a baby with the rare genetic disease, carbamoyl-phosphate synthetase 1 deficiency (CPSID) (1). CPSID makes it difficult for the body to break down proteins, leading to high levels of ammonia in the blood, which can cause brain damage or death. The condition has a 50 percent mortality rate in early infancy. Muldoon’s doctors, however, got permission from his parents to develop a personalized base editing therapy to correct his mutation causing CPSID. He received three doses of the in vivo base editing therapy, and according to a news report in Nature, his doctors are slowly reducing his ammonia-reducing medication. While they are hesitant to call him cured, Muldoon is doing well. – Stephanie DeMarco

UCLA patient receives the first human bladder transplant

A pair of surgeons at the University of California, Los Angeles and the University of Southern California announced that they performed the first bladder transplant in a 41-year-old man with a rare form of bladder cancer. Currently, most patients who have their bladder removed undergo a procedure that makes part of their intestine into a new pathway to excrete urine from the body. However, this leads to complications in the majority of patients. After developing the new transplant technique for four years — which involved transplanting a bladder and a kidney from a donor — urologic surgeon Inderbir Gill told The New York Times, “There is no question: A potential door has been opened for these people that did not exist earlier.” – Allison Whitten

FDA restricts access to updated COVID-19 vaccines

On Tuesday, the FDA released a stricter regulatory framework for updated COVID-19 vaccines (2). They plan to approve updated vaccines for adults 65 and older or those older than six months of age who are at high risk of severe COVID-19 outcomes if those vaccines lead to an antibody response in these populations — how previous COVID-19 booster vaccines and the annual flu shot are assessed. However, if vaccine developers wish to receive approval for their updated COVID-19 vaccines in healthy people aged six months to 64, the FDA will now require that they conduct randomized, placebo-controlled trials and show that the vaccines prevent symptomatic COVID-19 as the primary endpoint — which is not what the COVID-19 vaccines were originally intended to do. They were always meant to protect from severe disease. As noted in DDN’s prior coverage of the FDA’s earlier announcement of requiring randomized, placebo-controlled studies for updated COVID-19 vaccines, these trials would require enrolling thousands of participants and be incredibly expensive to conduct. The FDA will also require six months of follow up data in these trials, which could mean that the vaccine is no longer effective against the circulating strain of the virus by the time it is approved. All of these factors will likely disincentivize companies from running these kinds of trials.

As justification for their new framework, FDA Commissioner Martin Makary and director of the Center for Biologics Evaluation and Research Vinay Prasad wrote, “While all other high-income nations confine vaccine recommendations to older adults (typically those older than 65 years of age), or those at high risk for severe Covid-19, the United States has adopted a one-size-fits-all regulatory framework and has granted broad marketing authorization to all Americans over the age of 6 months,” but this statement is misleading. Other countries do reserve their recommendations for a COVID-19 vaccine to these populations, but the vaccine is still largely available to people over 6 months of age who want one. This is the case in Canada, and in the UK, COVID-19 vaccines are available for people to purchase out of pocket if they are not in the recommended groups.

This framework was also announced on Tuesday without consulting any experts on the FDA’s Vaccines and Related Biological Products Advisory Committee, who were set to meet to discuss future COVID-19 vaccine plans just two days later on Thursday. This action also preempts a CDC advisory committee meeting that is scheduled to discuss COVID-19 vaccine recommendations in June. Public Citizen Health Research Group Director Robert Steinbrook said in a statement, “The FDA announcement does not make a convincing public health case for why COVID-19 vaccines should become unavailable for healthy persons under 65. People should be able to look at the information, discuss it with their clinicians, and be vaccinated if that’s what they decide.” – Stephanie DeMarco

Microplastics weaken the lung’s macrophages

Work presented at the American Thoracic Society 2025 conference this week showed that inhaled microplastics can suppress the activity of white blood cells in the lungs called macrophages (3). These cells are crucial to a process called phagocytosis, in which a cell ingests bacteria and other material. Led by immunologist Adam Soloff at the University of Pittsburgh, the researchers measured macrophage function after culturing macrophages with microplastics and exposing mice to inhaled microplastics. After just 24 hours of microplastics exposure, the team revealed that macrophages were worse at the process of phagocytosis, and they found microplastics in the lung, brain, kidney, heart, and colon. By using the adenosine regulating agent Acadesine, the researchers could improve the function of the macrophages. In a press release, Soloff said, “Given the poor air quality in so many places around the world, you could imagine that developing a low-cost, low-side-effect therapeutic to restore pulmonary macrophage function may be an important tool to combat increasing rates of lung disease.” – Allison Whitten

A faster, less painful way to diagnose multiple sclerosis

A study published in Neurology Open Access showed that researchers from the University of Nottingham were able to diagnose multiple sclerosis in eight minutes using magnetic resonance imaging (MRI) scans (4). The discovery means that patients may no longer have to undergo painful spinal tap procedures to receive a diagnosis. The team used a T2*-weighted MRI scan and compared the diagnostic results to that of invasive spinal taps and confirmed that use of the central vein sign on the scan was just as sensitive. They observed side effects in nine percent of participants following the MRI scan compared to 75 percent with the spinal tap. Nikos Evangelou, lead neurologist on the study, said in a press release, “With the MRI scan, it takes eight minutes, is completely safe, and then you can go home. On average, we think we will reach the diagnosis three months earlier.” – Allison Whitten

FDA approved the first blood test for Alzheimer’s disease

Last Friday, the FDA approved the first blood test for Alzheimer’s disease (AD). The test, called the Lumipulse G pTau217/ß-Amyloid 1-42 Plasma Ratio, is manufactured by the Japanese company Fujirebio Diagnostics. In people with symptoms of cognitive decline, the test measures the ratio of the proteins pTau217 and β-amyloid 1-42 in a blood sample, and based on that ratio, it determines whether amyloid plaques are present or absent in the brain, helping doctors diagnose AD. Because it only requires a blood draw, this test makes AD testing much more accessible compared to other methods which require a lumbar puncture or a positron emission tomography scan. Maria Carrillo, the Chief Science Officer and Medical Affairs Lead of the Alzheimer’s Association said in a statement, “For too long Americans have struggled to get a simple and accurate diagnosis; with today’s action by the FDA we are hopeful it will be easier for more individuals to receive an accurate diagnosis earlier.” – Stephanie DeMarco

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