We have written extensively about this baby, who has become the mascot of tailor-made editing, thanks to a treatment developed in record time specifically for him. We are delighted to see him in excellent shape, dressed for the occasion at the STAT summit!
The coordinated effort that last spring saved the life of little KJ Muldoon earned widespread and enthusiastic media coverage. But between the invention of the treatment and its delivery to the patient lay a lesser-told story: an unprecedented manufacturing sprint. Genetic Engineering & Biotechnology News organized an online roundtable led by its deputy editor in chief, Julianna LeMieux, to discuss how therapeutic components were produced quickly, cost-effectively, and to clinical-grade standards.
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Last spring, we reported on the possible fall from grace of messenger RNA technology within the US administration. This was despite the Nobel Prize awarded to Katalin KarikĂł and Drew Weissman, and despite the millions of lives saved by RNA vaccines during the COVID-19 pandemic. So, how did it end?Â
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Last May, the case of baby KJ made headlines: the child, suffering from a severe metabolic disorder, received a therapy developed specifically for him in just six months. The rapid improvement in his condition and his discharge from the hospital left the rare disease community with a pressing question: was this an unrepeatable one-off, or a replicable model of intervention? The right answer might be the latter, as demonstrated by the launch of the Center for Pediatric CRISPR Cures in California. This new center, to be led by Fyodor Urnov, begins with the mission of developing customized genome-editing treatments for eight young patients with congenital metabolic and immune system disorders.
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Italy has a strong tradition in malaria research, and it’s noteworthy that one of the most innovative tools to fight this disease in the future was developed with the crucial contribution of an Italian scientist. Andrea Crisanti has become a familiar face in the country due to his expertise in COVID-19 epidemiology and numerous TV appearances, first during the pandemic and later in his role as a senator. Yet his international reputation is more firmly rooted in a different field: the development of CRISPR-based strategies to eliminate malaria-transmitting mosquitoes.
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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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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 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.
Continue readingThe 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.
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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CRISPeR FRENZY 