For men with hemophilia B, receipt of gene therapy comprising an infusion of etranacogene dezaparvovec results in sustained endogenous factor IX expression and low annualized bleeding rates over five ...
The rate of severe infections decreased from 2 infections to 0.2 infections per PYO after treatment with etuvetidigene autotemcel.
Fondazione Telethon announced that FDA has approved its Biologics License Application (BLA) for Waskyra (etuvetidigene ...
The Food and Drug Administration this week granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...
Based on initial positive results from HOPE-B, etranacogene dezaparvovec became the first gene therapy approved for adults ...
A breakthrough therapy is driving aggressive leukaemia into remission. Here’s what the headlines don’t tell you about life after treatment.
Investment from XGEN Venture, British Business Bank, and a global biopharmaceutical company will support first-in-human Phase ...
The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...
FDA approves 1st gene therapy for Wiskott-Aldrich syndrome, offering a new treatment for patients lacking matched stem cell donors.
Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...
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 ...
Later-stage financing deals for cell and gene therapy companies reflects a broader derisking trend in the pharma industry.
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