Category: gene editing
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STAT+: FDA unveils rules for bespoke gene therapies, predicting flood of rare disease applications
STAT+: FDA unveils rules for bespoke gene therapies, predicting flood of rare disease applications The Trump administration on Monday released detailed guidance for approving the first bespoke medicines crafted to treat patients’ individual mutations. Food and Drug Administration Commissioner Marty Makary and biologics chief Vinay Prasad had already previewed the new approach, known as the plausible mechanism…
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STAT+: Key scientist from Baby KJ team launches startup to scale personalized CRISPR medicines
STAT+: Key scientist from Baby KJ team launches startup to scale personalized CRISPR medicines In between bites of thin, crispy slices of pizza at Berkeley’s Pizzeria da Laura, gene editing researcher Fyodor Urnov and venture capitalist Johnny Hu began hatching a plan for a different kind of CRISPR company. It was spring 2024 and Urnov,…
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The top medical advances of 2025
The top medical advances of 2025 It has been a tough year for medicine. The U.S. research enterprise has been scrambled by a series of political decisions with long-reaching consequences. But make no mistake: Science didn’t stop, and medicine has had its share of major advances. As I have written for years, this is biology’s…
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STAT+: FDA reassures rare disease advocates that ‘being flexible’ is plan for gene therapy
STAT+: FDA reassures rare disease advocates that ‘being flexible’ is plan for gene therapy What will the Trump administration mean for gene therapy? Researchers, advocates, and executives got a glimpse of the answer Thursday, as the Food and Drug Administration convened a “Cell and Gene Therapy Roundtable.” It was an unusual event. Multiple panelists told…
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STAT+: What Baby KJ means for the CRISPR gene editing industry
STAT+: What Baby KJ means for the CRISPR gene editing industry For the ailing gene editing industry, hope came earlier this month in the tiny, smiling, fuzzy-headed form of KJ Muldoon. At just 6 months old, KJ received a gene editing treatment custom-built to correct his unique mutation. He’s not cured, researchers explained at the…
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Uncertainty in CRISPR world and the start of more M&A
Uncertainty in CRISPR world and the start of more M&A What was the mood like at a big gene therapy conference last week? Why is Novo Nordisk’s CEO stepping down? And why has Adam been in such a good mood? We discuss all that and more on this week’s episode of “The Readout LOUD,” STAT’s…
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STAT+: Day 2 of ASGCT: Good news for Baby KJ and the dream of personalized gene editing
STAT+: Day 2 of ASGCT: Good news for Baby KJ and the dream of personalized gene editing NEW ORLEANS — Greetings from ASGCT, where the gene therapy field had some great news for once, about a highly photogenic infant. Baby KJ gets a custom CRISPR edit to his genome Kiran Musunuru, a researcher at the University of…
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STAT+: Day 1 at ASGCT: Missing scientists, a gene editing failure, and a gene editing first
STAT+: Day 1 at ASGCT: Missing scientists, a gene editing failure, and a gene editing first NEW ORLEANS — The mood at the annual meeting of the American Society of Cell and Gene Therapy isn’t amazing. The biotech market has been bad for years. The FDA and NIH are shrouded in uncertainty. But many researchers…