ROME, Italy, Dec. 10, 2025 /PRNewswire/ -- Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex ...
lt;pgt;Scientists have successfully reconstructed a virus thousands of years after it became extinct, a development they believe could herald a new step in treating genetic diseases such as cystic ...
Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...
Advancements in genetics have already begun changing the way scientists study, predict, diagnosis and treat diseases. But how does the public feel about gene therapy and gene editing? In general, the ...
Researchers at the University of Geneva, together with colleagues in Switzerland, France, the United States and Israel, ...
In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...
A pioneering genetic treatment is saving lives where chemotherapy failed, offering new hope for children and adults with aggressive leukemia. This world-first therapy has kept patients disease-free ...
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.
Biogen has discontinued all gene therapy programs using adeno-associated virus (AAV) capsids, instead shifting resources to modalities that have the highest likelihood of achieving better treatment ...
WASHINGTON — For Wedam Minyila, hospital rooms have always meant blinding pain. “Like someone is jamming a knife in me,” he said. But for a brief moment on a recent December morning, Wedam, 19, who ...
Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. The New York pharma is ending global development and commercialization of Beqvez ...
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