Baby KJ is a symbol of hope — But is it replicable?

The personalized editing therapy developed at record speed for the American newborn required preliminary studies, favorable circumstances, and a heroic collective effort.

After 307 days, little KJ Muldoon was discharged from the Children’s Hospital of Philadelphia (CHOP), wearing a tiny graduation outfit complete with a blue gown and cap. Born ten months ago with a severe metabolic disorder, the baby received a genome editing therapy developed exclusively for him, and his remarkable progress has been hailed by many as the dawn of a new era in precision medicine. A month after the publication of his case in the New England Journal of Medicine, we take a closer look at how researchers managed to develop the treatment in just six months—and whether this breakthrough could be replicated for other rare disease patients in need of life-saving therapies.

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First CRISPR treatments in Italy

Italy has begun administering the first CRISPR-based treatment. The therapy for sickle cell disease and beta thalassemia (Casgevy) has already been delivered to four patients across three clinical centers within a month. The announcement was made during the conference “Italian Primacy in the Treatment of Hemoglobinopathies” held yesterday at the Senate.
For more information, we recommend Francesca Ceradini’s article in Osservatorio Terapie Avanzate.

Emma and Tumor-Infiltrating Lymphocytes

Oncologist Emil Lou and patient Emma Dimery

Emma Dimery was 23 years old when she was diagnosed with stage 4 colon cancer. Over the next decade, she tried everything in vain: surgery, chemotherapy, radiation, and conventional immunotherapy. That changed in March 2023, when she received an infusion of tumor-infiltrating lymphocytes (TILs) edited with CRISPR at a key gene checkpoint, CISH. Since then, she has been in complete remission, with no detectable traces of the tumor.

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Hi I’m JK, the first baby treated with a personalized CRISPR therapy

The announcement was made yesterday at the annual meeting of the American Society of Gene & Cell Therapy and simultaneously published in the New England Journal of Medicine. I wrote about it for the Italian edition of Scientific American, but this story is worth seeing and reading.
PS: Before KJ was treated for his metabolic disorder, there was the case of Terry Horgan, who had muscular dystrophy. Unfortunately, the personalized treatment for him came too late, and he did not survive an adverse reaction. So yes, we can say that KJ represents a first: the first time genome editing was used early, rapidly, and tailored enough to truly offer hope for a happy ending.

Breakthrough Prize 2025: Jodie Foster explaining gene editing and more

Gene editing pioneer David Liu received the Breakthrough Prize from the hands of Jodie Foster and Lily Collins — but the biggest applause went to young CAR-T patient Alyssa Tapley.

The movie stars in the audience in Santa Monica on April 12 were easy to spot: Brad Pitt, Margot Robbie, Sean Penn, and others. But it’s rare for a scientist to become a celebrity beyond academic circles. This role reversal happens just one day a year, when the Breakthrough Prize is celebrated in California. Richer than the Nobel (the prizes are worth three times as much) and steeped in glamour, the event honors the stars of science with the help of Hollywood and technofinance.

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CRISPR News – A Trio of Firsts

Many interesting papers have been published recently; here are our top three picks. They cover an innovative gene therapy trial, a new experimental approach for oncology, and the development of novel tools to map gene enhancers.

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The rare story of Sonia and Eric: pioneers by force and by love

A scientific adventure whose ingredients include the looming threat of a fatal disease, the decision to reinvent themselves as biologists, and the goal of silencing prions.

The clinical trial with antisense oligonucleotides, born of their efforts, is considered one of the most interesting trials of 2025, but this is only a part of the story. This married couple is also pursuing other avenues to halt the onset of prion diseases. In the summer of 2024, they published a study in Science using epigenetic editing in mice. Then, in January 2025, their experiments with base editing were published in Nature Medicine. Yet Sonia Vallabh was a newly graduated jurist, and her husband, Eric Minikel, was working in urban planning, when they discovered that she carried a mutation that would condemn her to die of fatal familial insomnia within two or three decades.

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The Huge Little Thing: NanoCas is Coming

3D structure of the NanoCas system [Mammoth Biosciences]

It is currently only a preprint on bioRxiv, but it has already attracted significant attention from the scientific community and the journal Science. Mammoth Biosciences, a company founded by CRISPR co-inventor Jennifer Doudna, has developed NanoCas, a mini-editor that is just one-third the size of traditional gene-editing scissors (Cas9).

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Why AI + CRISPR Will Define 2025

Image Credit Karen Arnott/EMBL-EBI

Have you read Jennifer Doudna’s article in Wired? She discusses how the combination of CRISPR and artificial intelligence could be transformational. In her lab, researchers recently used AI tools to help find “small gene-editing proteins that had been sitting undiscovered in public genome databases because we simply didn’t have the ability to crunch all of the data that we’ve collected”.

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Chemotherapy pretreatment claims a victim in a CRISPR trial

Busulfan 3D

Experimental patients often find themselves in a paradoxical situation: they must be sick enough to qualify for a clinical trial but healthy enough to endure its side effects. They also need the audacity to subject their bodies to protocols whose safety and efficacy remain unproven. For this reason, many describe them as pioneers or even warriors.

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