Category: Gene therapy
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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+: FDA rejects Regenxbio’s rare-disease gene therapy
STAT+: FDA rejects Regenxbio’s rare-disease gene therapy The Food and Drug Administration has rejected a rare-disease gene therapy from Regenxbio, the company said Monday. The one-time treatment, called RGX-121, is designed to replace a malfunctioning gene that causes mucopolysaccharidosis type II, also known as Hunter syndrome, an ultra-rare disorder that causes physical and cognitive impairments. …
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The high stakes for Huntington’s patients
The high stakes for Huntington’s patients Biotech company UniQure is set to meet with the Food and Drug Administration to discuss the path forward for its gene therapy for Huntington’s disease, and the outcome could be potentially devastating for patients. That’s what patient advocate Lauren Holder says on the latest episode of “The Readout LOUD,”…
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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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STAT+: FDA’s stronger warning on Sarepta gene therapy raises new questions about heart risk
STAT+: FDA’s stronger warning on Sarepta gene therapy raises new questions about heart risk New warnings and restrictions placed on Sarepta Therapeutics’ gene therapy last week by the Food and Drug Administration have cast a spotlight on an important but overlooked heart-safety risk. “Acute, serious, and life-threatening” cases of heart inflammation called myocarditis and elevations…
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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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STAT+: Sarepta to seek approval for gene therapy in rare form of muscular dystrophy
STAT+: Sarepta to seek approval for gene therapy in rare form of muscular dystrophy An experimental gene therapy from Sarepta Therapeutics increased levels of the gene missing in an ultra-rare form of muscular dystrophy, according to data the company presented Friday. The company has said it plans to file for approval in the disease, known…
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A mother’s perspective on Sarepta’s gene therapy halt
A mother’s perspective on Sarepta’s gene therapy halt How are families of patients reacting to the removal of Sarepta’s gene therapy from the market? Why are investors excited about narcolpesy drugs? And why has there been less financing in oncology startups? We chat about all that and more on this on this week’s episode of…
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STAT+: For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak
STAT+: For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak Jennifer Hill Blair recognized the Cincinnati number on her buzzing phone and slipped out of the school meeting, excited to finally learn when her son Cayse would receive a therapy she believed would change his life. Blair, a second-grade teacher in southern…
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Opinion: We are mothers of Duchenne patients. Recent setbacks with Sarepta must not stop progress
Opinion: We are mothers of Duchenne patients. Recent setbacks with Sarepta must not stop progress They say death is one of life’s few certainties. For a boy or young man living with Duchenne muscular dystrophy, that certainty has a cruel twist: the anticipation of dying young. As mothers of children with this disease, we have…
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STAT+: What’s next for Sarepta Therapeutics with gene therapy under fire?
STAT+: What’s next for Sarepta Therapeutics with gene therapy under fire? This story is an excerpt from Adam’s Biotech Scorecard, a subscriber-only newsletter. STAT+ subscribers can sign up here to get it delivered to their inbox. The Duchenne muscular dystrophy community is reeling following the second death of a patient from liver failure caused by Sarepta Therapeutics’…
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FDA’s gene therapy turmoil, and an alternative model for funding research
FDA’s gene therapy turmoil, and an alternative model for funding research Why did the Food and Drug Administration suddenly oust one of its top gene therapy regulators? Is the agency growing too political? And should private firms fund academic research? We chat about all that and more on this on this week’s episode of “The…
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Opinion: Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys
Opinion: Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys On Sunday at 1 a.m. Eastern time, an announcement went out from the first-generation gene therapy company for Duchenne muscular dystrophy: A second patient who had been treated with Elevidys has died. This second family said yes to hope, yes to science,…
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STAT+: Top gene therapy regulator forced out at FDA
STAT+: Top gene therapy regulator forced out at FDA Nicole Verdun, director of the office that reviews cell and gene therapies at the Food and Drug Administration, and her deputy Rachael Anatol have been placed on administrative leave and escorted out of the agency, according to a recording of a meeting obtained by STAT. Verdun…
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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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Opinion: STAT+: RFK Jr. acknowledges receiving unproven stem cell treatment from an Antigua clinic
Opinion: STAT+: RFK Jr. acknowledges receiving unproven stem cell treatment from an Antigua clinic Welcome to Lab Dish, a First Opinion column on regenerative medicine from Paul Knoepfler. Health and Human Services Secretary Robert F. Kennedy Jr. recently revealed on a health influencer podcast that he received unproven stem cells at a clinic in Antigua…
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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 3 at ASGCT: Vinay Prasad speculation, screening newborns, promising data
STAT+: Day 3 at ASGCT: Vinay Prasad speculation, screening newborns, promising data NEW ORLEANS — Greetings from the home stretch of American Society of Gene and Cell Therapy conference, where everyone is still talking about Baby KJ and no one wants to talk about Vinay Prasad. The Vinay Prasad in the room Last year, hundreds…
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Prenatal genetic therapy for SMA shows promise in a mouse study
Prenatal genetic therapy for SMA shows promise in a mouse study Researchers have taken preliminary steps toward treating a devastating neurodevelopmental disorder, spinal muscular atrophy, with a genetic therapy delivered in the womb, before the worst damage to motor neurons can occur. The scientists at the University of California, San Francisco, and Johns Hopkins University…
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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…
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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…