Category Archives: Biomedicine

Craig Venter: many battles won, one left unfinished

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Credit: Brett Shipe / J. Craig Venter Institute

Craig Venter was the first person to read his own genome (his full sequence was published in 2007). He was able to study his genetic predispositions and undergo the most advanced tests to verify their real-world relevance. In 2014, he launched a company called Human Longevity with the goal of building bridges between genetic sequences and diagnosis. In this way, by 2016 he had identified and defeated prostate cancer, but science and luck were not enough to save him a second time in 2026. When he passed away on April 29, he had not accomplished everything he had hoped to, but more than enough to secure a place in history, and perhaps even earn the respect of many of his former rivals.

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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.

Gene editing’s new bet on PKU

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Jennifer Doudna and Fyodor Urnov have founded a company that will take on the challenge of editing rare diseases, starting with phenylketonuria.

The problem is well known: many diseases are theoretically treatable by correcting the corresponding genetic defects with the help of CRISPR tools, but doing so risks being difficult or even impossible because of regulatory rigidity and economic unsustainability. The case of the first newborn treated with a bespoke therapy developed in record time (KJ Muldoon) was a splendid proof of principle. But it left many parents of children with rare diseases asking: when will it be our turn? At the same time, specialists have long been asking: will investors return to viewing gene editing as a profitable approach worth investing in?

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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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Can technology replace animal testing?

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New Approach Methodologies (NAMs) have a bright future ahead, but they should be seen as complementary rather than alternative to classical experimentation.

Regulatory and funding agencies in the U.S. and Europe are promoting ambitious initiatives to foster the development and adoption of advanced systems capable of testing the effects of drugs and other substances without using animal models. The hope is that biomedical research can become more ethical, safer, and cheaper. But the challenge is complex, and the requirements vary depending on the application. As a result, some voices urge a faster “transition,” while others warn that rushing the process could be risky. Recently published articles in leading scientific journals capture this polarized debate, but they also hint at a possible middle ground.

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