Topic
gene-therapy
19 stories related to this topic, newest first.
Wiredscience9 hrs ago
U.S. biotech firm begins first human test of gene therapy for cellular ageing
A Boston-based biotechnology company has started the first clinical trial of a gene therapy intended to reverse cellular ageing. The treatment, called ER-100, targets optic nerve damage and has moved into Phase 1 safety testing.
foxnews.comscience12 days ago
Gene Therapies Advance to Human Trials for Heart Regeneration
Researchers have begun the first clinical trial of a gene therapy designed to grow new heart-muscle cells. At least four additional regenerative gene therapies for heart conditions are in development by companies.
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Statscience21 days ago
UCSF Team Submits Application for First In Utero Gene Therapy Trial
A University of California San Francisco team has submitted an investigational new drug application to the Food and Drug Administration for a small trial of in utero gene therapy. The proposed study would treat five fetal patients with a rare lysosomal storage disorder.
ncbi.nlm.nih.govworld23 days ago
Krystal Biotech Develops Gene Therapy for Rare Skin Disorder
Suma Krishnan, founder and president of Krystal Biotech, discussed a gene therapy treatment for epidermolysis bullosa at an America 250 event. The therapy targets the rare disease that leaves children born without fully formed skin.
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Statscience27 days ago
Regenxbio Gene Therapy for Duchenne Muscular Dystrophy Succeeds in Trial
Regenxbio announced on May 14, 2026 that its experimental treatment produced high levels of a miniaturized muscle protein, clearing the path for an FDA submission. The Rockville, Md.-based company aims to offer a safer and more effective option than Sarepta Therapeutics’ Elevidys…
Statscience27 days ago
Scientists Link Boy’s Brain Tumor to Gene Therapy Viruses
A 13-month-old boy with a rare disease received an experimental gene therapy after his stem cell transplant failed. A routine scan later revealed a golf-ball-sized tumor on his brain. Scientists have linked the tumor to viruses used in the gene therapy, according to a STAT report…
prnewswire.comscience28 days ago
Encoded Therapeutics Reports Initial Results from Gene Therapy Trial for Dravet Syndrome
A trial by Encoded Therapeutics involving 21 children with Dravet syndrome showed that sirolimus, used alongside steroids to prevent immune responses, could blunt the therapy's benefits. The findings, published May 13, 2026, come from a Boston-based report examining a key concern…
Statscience28 days ago
Scientists Link Boy’s Brain Tumor to Gene Therapy Viruses
Doctors identified a link between a gene therapy treatment and a brain tumor in a young patient with a rare disease. The case is described as rare, with medical professionals stating that treatment risks must be weighed against benefits. The patient had previously undergone a ste…
The Independentworld29 days ago
Small Study Shows Modified Immune Cells Suppress HIV in Two Patients
Researchers reported that a single dose of genetically engineered CAR-T cells strongly suppressed HIV in two people for nearly one and two years without daily medication. The early-stage experiment modified immune cells to better target HIV-infected cells while protecting them fr…
prnewswire.combusiness29 days ago
Capsida Biotherapeutics Updates Investigation Into Death of Child in CAP-002 Gene Therapy Trial
The company said Tuesday it remains unable to determine what caused the death of a child last September in a trial of its experimental brain-targeted therapy CAP-002. Scientists have been blocked from full investigation because the hospital has declined to provide autopsy tissue…
pharmaphorum.comscience30 days ago
Astellas to Restart Gene Therapy Trial for XLMTM Five Years After Patient Deaths Halted Program
Patients and families affected by the rare muscle disorder XLMTM are receiving a second chance at treatment as Astellas relaunches its gene therapy program. The move comes as fresh turmoil grips the top of the FDA with questions swirling over whether Commissioner Marty Makary wil…
thehindubusinessline.comscience30 days ago
Two Patients Achieve Undetectable HIV Levels After Experimental Engineered Immune Cell Treatment
Scientists report that a single infusion of modified immune cells enabled two people to suppress HIV to undetectable levels, one for nearly two years. The findings, presented as proof of concept, draw on a decade of success using similar techniques against blood cancers. Research…
Statscience30 days ago
Parents of Boy with Rare Disease Report Reduced Suctioning Needs After NIH Treatment
Jessica Gonzalez realized an hour had passed without suctioning her son Joshua Jacob Gonzalez’s mouth and he was breathing normally, weeks after treatment at the National Institutes of Health. For his first two years, Javier and Jessica Gonzalez had suctioned saliva from the back…
science34 days ago
Researchers Use Viral Vectors to Deliver HIV Drug in Macaque Study
A study published by Science Translational Medicine found that a single dose of adeno-associated vectors carrying the HIV drug leronlimab produced sustained expression of the drug. The approach partially suppressed SHIV infection in macaques for up to one year. The drug blocks th…
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finance.yahoo.comscience35 days ago
FDA Shifts Remain Focus for Cell and Gene Therapy Industry at European Meeting
Cell and gene therapy developers gathered in Rome last week to discuss the sector's outlook in Europe. Conversations centered on recent FDA actions including a doubled rejection rate for such therapies since 2024 and the departure of the agency's top regulator for gene and cell t…
marketwatch.comscience48 days ago
Regeneron Agrees to Lower Drug Prices and Offer Free Gene Therapy in U.S. Deal
Biotech company Regeneron has entered into an agreement with the U.S. government to reduce medication costs for American patients. The deal includes offering a new hearing-loss gene therapy at no charge to eligible individuals. This is part of broader efforts to align U.S. drug p…
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Substrate placeholder — needs reviewworld62 days ago
Recent Advances in Gene Therapy for Rare Diseases Reported
Developments in gene therapy and targeted medications have shown progress in treating rare diseases such as spinal muscular atrophy and certain forms of blindness. These conditions affect an estimated 30 million people in the United States, with over 7,000 identified rare disease…
Substrate placeholder — needs reviewscience62 days ago
Asia Summit on Global Health Scheduled for May 2026 in Hong Kong
The Asia Summit on Global Health (ASGH) 2026 will take place on May 11-12 in Hong Kong. The event will cover topics including gene and cell therapies, precision medicine, smart ageing, artificial intelligence, and digital health. Organizers invite participants to register for the…
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Substrate placeholder — needs reviewscience63 days ago
Elevidys Gene Therapy Shows Positive Results in Duchenne Muscular Dystrophy Trial
Sarepta Therapeutics reported positive topline results from a phase 3 trial of its gene therapy Elevidys for Duchenne muscular dystrophy. The trial met its primary endpoint of improved motor function in patients aged 4 to 7. The Duchenne muscular dystrophy field has experienced s…
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