Category Archives: medicine

The lesson of little Adam: gene therapy and cancer risk

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Four years after an experimental treatment for a rare disease, the child underwent surgery for cancer, the first case caused by a virus widely used as a vector, but he is now doing well.

The question of whether gene therapy using adeno-associated viruses (AAVs) can cause tumors has been the subject of intense debate over recent decades. Until now, our understanding of their mechanisms and the data collected over the years from thousands of patients had appeared reassuring. In fact, AAVs are the most commonly used vectors for “in vivo” therapies precisely because they usually deliver their package, containing the therapeutic gene, near the patient’s DNA without integrating into it, thereby avoiding disruptions. Will the risk-benefit balance now change, after the first documented case of a tumor linked to their use in a clinical trial?

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CRISPR creates cancer-fighting cells inside the body

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Don’t miss the video where reserachers from the Innovative Genomics Institute and UC San Francisco explain their cancer immunotherapy work, published in Nature on March 18, 2026. A breakthrough that could make next-generation CAR-T therapies faster, cheaper, and accessible to more patients worldwide.

CRISPR medicine: a 2026 Snapshot

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Amid research funding cuts under the Trump administration, a slowdown in venture capital investment, and uncertainty driven by shifting policies in adjacent fields such as RNA vaccines, the past year has been anything but smooth for genome-editing therapies. Even so, clinical trials have continued to expand, and a couple of treatments are nearing commercialization. Moreover, the success of the first personalized therapy developed for a newborn with a rare disease (Baby KJ) has sparked a regulatory rethink that could open up new opportunities. This is the latest snapshot from the Innovative Genomics Institute, which regularly tracks the progress and prospects of CRISPR-based medicine. Post scriptum: In addition to the main article, be sure to explore the dedicated sections on specific disease areas, including cardiovascular disease, autoimmune disorders, cancers, rare genetic diseases, red blood cell disorders, diabetes, and infections.

A first for Prime Editing

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It had never happened before that a company decided to submit a commercial authorization request for a therapy tested on only two people.

We do not know the name of the teenager from Vancouver who, a year ago, became the first person in the world to receive a treatment based on a genetic correction approach similar to Word’s “find and replace.” What we do know is that before becoming a pioneer patient, even a common cold represented a serious threat to him. The father of the technique known as prime editing, David Liu, now describes him as “healthy, stable, and living with a functioning immune system.” Seeing him on skis in the the snow in the photo published by the Canadian Institutes of Health, is worth more than many words. The American National Institutes of Health, for their part, confirm that the second patient treated is also doing well.

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CRISPR N=1 in 101 seconds

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Petros Giannikopoulos, director of the clinical laboratory at the Innovative Genomics Institute at the University of California, Berkeley, explains in 101 seconds the bespoke treatment developed in record time for a very special patient, whom we have mentioned many times here on CRISPeR Frenzy: Baby KJ. The video was shot by Sean Patrick Farrell.

The first CRISPR therapy works, but adoption is slow

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Two years after Casgevy received commercial approval, only around sixty people with sickle cell disease or thalassemia have been able to benefit from it, due to a technical hurdle that the next generation of treatments will attempt to overcome

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CRISPR: hitting the genetic bullseye

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Imagine throwing a trillion darts and having every single one hit the bullseye. Achieving this level of precision in gene editing would require highly intelligent delivery of the molecular machinery for DNA repair (CRISPR or one of its variants) into patients’ bodies, reaching only defective cells while bypassing healthy tissues. The benefits would be substantial: maximum therapeutic efficiency, zero waste, and reduced risks in terms of toxicity, immunogenicity, and unwanted mutations. How can such precise targeting be achieved? By acting on multiple levels, explain Jennifer Doudna and three researchers from her Innovative Genomics Institute. See their review article, Targeted delivery of genome editors in vivo in Nature Biotechnology.

Baby KJ in Nature’s Top Ten

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Every December, Nature selects the 10 people of the year, those who have most shaped the year that is coming to an end. For 2025, the little KJ Muldoon, about whom we have written many times, could not be left out. The first newborn to receive a CRISPR treatment developed specifically for him, the inspiration for new rules on the testing of advanced therapies for rare diseases, the mascot that patients, families, doctors, and scientists needed to look to the future of medical editing with renewed confidence.

The FDA charts a new route for bespoke therapies

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Inspired by the Baby KJ case, the agency proposes a flexible framework allowing personalized treatments for individual patients to contribute to shared, platform-based approvals.

The announcement appeared on November 12 in the New England Journal of Medicine under a seemingly cautious title: “The FDA’s New Plausible Pathway.” Yet the article, written by two senior figures at the Food and Drug Administration, reveals vision and leadership. For once, it is worth starting from the end, which reads like a strong statement of intent: “Nearly 30 years after the sequencing of the human genome, bespoke therapies are close to reality. The FDA will work as a partner and guide in ushering these therapies to market, and our regulatory strategies will evolve to match the pace of scientific advances.”

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Baby KJ Effect: the new horizon of bespoke CRISPR therapies

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The FDA is set to authorize “umbrella” clinical trials for rare diseases; the new approach will make the process faster and more sustainable by combining data from similar protocols, cutting redundant procedures, and reducing animal testing.

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