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.
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.
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 ...
EpilepsyGTx, a biotechnology company focused on research and development of cutting-edge gene therapies to treat refractory epilepsy, today announced it has raised $33 million in a Series A financing ...
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 ...
Scientists at UCL and GOSH have used groundbreaking base-edited CAR‑T cell therapy — BE‑CAR7 — to treat aggressive T‑cell ...
The first-of-its kind gene therapy uses a patient’s skin cells to create sheets to treat the open wounds caused by the rare, ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback