Tag Archives: gene

The uncertainty around the future of Elevidys hasn’t destroyed Roche’s belief in the potential of gene therapies, the Swiss company’s head of pharma has told Fierce Biotech, as the pharma continues to expand its research reach outside of its traditional oncology focus. Roche and its partner Sarepta Therapeutics both paused trials of Elevidys back in April following the death of a 16-year-old …

CRISPR gene-editing therapy has shown great potential to treat and even cure diseases, but scientists are now discovering how it can be used to prevent them as well. A team of researchers found a way to edit a single gene in a mosquito that prevented it from transmitting malaria, according to a paper published in Nature. These genetically modified mosquitos …

Andrew Joseph covers health, medicine, and the biopharma industry in Europe. You can reach Andrew on Signal at drewqjoseph.45. European regulators on Friday said that a Duchenne muscular dystrophy gene therapy should not be approved, further imperiling the future of the treatment that is facing increasing scrutiny in the U.S. and globally.  The decision in Europe comes as Japan and …

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54. This is the online version of Adam’s Biotech Scorecard, a …

Thomas Fuller | SOPA Images | Lightrocket | Getty Images Sarepta Therapeutics said on Monday that it will pause all shipments of its Elevidys gene therapy in the United States after a muscular dystrophy patient who received a different, experimental treatment died. U.S. regulators on Friday asked Sarepta Therapeutics to voluntarily halt shipments of its gene therapy. However, the company …

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54. Sarepta Therapeutics said Monday evening it will pause all shipments …

Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy  Fierce Biotech We are mothers of Duchenne patients. Recent setbacks with Sarepta must not stop progress  statnews.com Sarepta Therapeutics stock sinks after FDA tells company to stop shipping its bestselling drug  Yahoo Finance Drugmaker Refuses F.D.A. Request to Pull Treatment Linked to Patient Deaths  The New York Times …

Sarepta says it won’t comply with FDA request to stop shipping gene therapy Elevidys  Reuters Sarepta Refuses FDA Request to Stop Shipping Muscular Dystrophy Drug Elevidys  The New York Times Sarepta shares plunge 40% as future of its gene therapy appears at risk  CNBC Third death from a Sarepta gene therapy  biocentury.com We are mothers of Duchenne patients. Recent setbacks with Sarepta must not …

Vitamin D is not only an essential nutrient, but also the precursor of the hormone calcitriol, indispensable for health: it regulates the uptake of phosphate and calcium necessary for bones by the intestines, as well as cell growth and the proper function of muscles, nerve cells, and the immune system. Now, researchers have shown for the first time in Frontiers …

The Food and Drug Administration will ask Sarepta Therapeutics to halt all shipments of its marketed gene therapy for Duchenne muscular dystrophy, a source familiar with the matter confirmed to BioPharma Dive. Sarepta last month paused shipments for certain older Duchenne patients following the death from acute liver failure of a second teenager treated with the therapy, called Elevidys. The …