Category: CRISPR
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STAT+: Prime Medicine to seek approval for gene-editing treatment after two-patient trial
STAT+: Prime Medicine to seek approval for gene-editing treatment after two-patient trial Prime Medicine said Tuesday it will ask the Food and Drug Administration to approve a gene-editing treatment that has been given to only two patients. The application will test an agency that has promised to speed new gene-editing treatments to patients but has…
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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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Opinion: Celebrating a new, faster path to gene-editing medicines on demand
Opinion: Celebrating a new, faster path to gene-editing medicines on demand In May, news broke of a biomedical first: the on-demand design and clinical use of a personalized gene editor for a baby boy born with a rare, severe genetic condition. At the STAT Summit in October, the child’s treating physician from the Children’s Hospital…
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STAT+: FDA chiefs offer roadmap to expand custom gene-editing treatments like Baby KJ’s
STAT+: FDA chiefs offer roadmap to expand custom gene-editing treatments like Baby KJ’s Top Food and Drug Administration officials on Wednesday detailed a roadmap for approving the world’s first personalized gene-editing treatments. The perspective, published in the New England Journal of Medicine, comes six months after researchers announced they had crafted a custom gene-editing treatment…
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STAT+: CRISPR-based treatment cuts cholesterol and triglycerides in early study
STAT+: CRISPR-based treatment cuts cholesterol and triglycerides in early study A CRISPR-based gene editing therapy dramatically reduced levels of low-density lipoprotein, often called “bad cholesterol,” and triglycerides in patients with heart disease, a new study found. Some researchers said that the treatment, developed by the biotech CRISPR Therapeutics and so far tested in just 15…
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Gene-edited pig lung transplanted into a brain-dead patient for first time
Gene-edited pig lung transplanted into a brain-dead patient for first time In a milestone for the newly resurgent field of xenotransplantation, a 39-year-old brain-dead person in China has become the first human to receive a lung from a pig. With consent from the person’s family, researchers took the organ from a CRISPR’d pig, trimmed it,…
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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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STAT+: At gene therapy meeting, no one wants to talk about the Vinay Prasad (not) in the room
STAT+: At gene therapy meeting, no one wants to talk about the Vinay Prasad (not) in the room NEW ORLEANS — Last year, hundreds of people poured in to see Peter Marks, the Food and Drug Administration’s head of biologics, speak at the American Society of Gene and Cell Therapy conference. The regulator was seen by…
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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+: Scientific societies call for a moratorium on using CRISPR to create genetically modified children
STAT+: Scientific societies call for a moratorium on using CRISPR to create genetically modified children Leading trade organizations representing the makers of cell and gene therapies are calling for a 10-year international moratorium on the use of CRISPR and other DNA-editing tools to create genetically modified children, according to a draft of the declaration provided…