KJ CRISPR Update: Prasad on ctDNA & Gene Editing
KJ Muldoon’s groundbreaking CRISPR gene-editing treatment marks a significant advancement, with the infant now discharged after initial therapy. This critical development, detailed in a recent report, highlights the potential of CRISPR therapy for treating rare diseases, moving beyond the initial three-dose treatment. Concurrently, experts are scrutinizing the utility of circulating tumor DNA (ctDNA) tests in oncology, as conclusive survival benefits remain unproven; the debate continues regarding the use of ctDNA testing.Vinay Prasad of the FDA is signaling a flexible approach to drug approvals for rare diseases, with a willingness to expedite biotech advancements. for more on these urgent medical advancements, continue reading updates on News Directory 3. Discover what’s next for these significant breakthroughs.
CRISPR Therapy, ctDNA Testing, and FDA Views on Rare Diseases
Updated June 05, 2025
KJ Muldoon, the first infant to receive personalized CRISPR gene-editing therapy, has been released from Children’s Hospital of Philadelphia after 307 days, marking a critically important milestone. The experimental treatment, aimed at correcting an ultra-rare disease preventing his liver from breaking down ammonia, involved three doses administered starting at six months old. Now 10 months old, KJ’s condition is expected to be far milder, though not fully cured. Researchers are exploring ways to scale this CRISPR therapy for wider use.
Meanwhile, circulating tumor DNA (ctDNA) blood tests are gaining traction in oncology, but their clinical impact remains uncertain. Data presented at the ASCO meeting suggest that while these tests show promise in detecting cancer recurrence and treatment resistance, conclusive survival benefits are lacking. University of Pennsylvania cancer researcher Angela DeMichelle emphasized the need for rigorous studies to validate the true value of ctDNA testing, cautioning against premature interventions that could harm patients.
In other news, Vinay Prasad, the new chief of CBER at the FDA, addressed the national Organization for Rare Disorders, signaling a possibly more flexible approach to drug approvals for ultra-rare diseases.while previously advocating for randomized controlled trials,Prasad acknowledged that not every drug requires one,notably in cases like Baby KJ. He stated the FDA’s commitment to expediting access to therapies, even with early signs of promise based on biomarker changes likely to predict clinical outcomes. This represents a shift in his stance on surrogate endpoints, indicating a willingness to act swiftly on biotech advancements for rare conditions.
Johns hopkins bioethicist Ruth Faden criticized HHS Secretary Robert F. Kennedy, Jr.’s move to drop COVID vaccine recommendations for pregnant individuals. Faden argues this decision disregards evidence of the vaccine’s benefits for both mothers and newborns, especially concerning heightened risks like preterm birth and stillbirth. She deems Kennedy’s call for randomized controlled trials scientifically unneeded and ethically indefensible.
What’s next
The medical community will be closely watching KJ’s progress and the long-term effects of the CRISPR treatment. Further research is needed to determine the true clinical value of ctDNA tests and refine their application in cancer care. The FDA’s evolving approach to rare disease drug approvals under Vinay Prasad’s leadership will also be a key area of focus.