Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to ...
VV169 saw complete clearance of disseminated myeloma tumors within 28 daysAnother dataset highlights Vyriad’s work in improved vector retargeting strategies in a comparative study of three methods for ...
Authors explore complexities in scaling gene-therapy production with special attention is to monitoring and enhancing ...
PERSEUS trial findings suggest that modern myeloma therapies could lead to survival rates similar to age-matched controls. KLN-1010, an off-the-shelf gene therapy, demonstrated promising MRD-negative ...
Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex vivo gene therapy for patients with Wiskott ...
The Structural Science Society (ACA) today announced the appointment of Mark Wilson, Professor and Associate Department Head of the Department of Biochemistry at the University of Nebraska?Lincoln, as ...
Minaris will move its center for viral vector innovation to occupy space within CGT Catapult’s collaborator laboratories in London and will work with CGT Catapult on the further development of ...
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 ...
CytoAgents, Inc. a clinical-stage biotechnology company developing CTO1681, a novel, steroid-sparing inhibitor of prostaglandin-mediated inflammation, announced today the appointment of Dr. Johannes ...
MedPage Today on MSN
Gene therapy wins FDA approval for life-threatening immunodeficiency
The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and older with Wiskott-Aldrich syndrome, a rare primary immunodeficiency.
Takeaway Gene corrected stem cell therapy represents a major scientific breakthrough for ADA SCID. When children are diagnosed early and treated quickly they can develop their own strong immunity and ...
The use of viral vectors to deliver genes to the nervous system shows great promise for both basic research and therapeutic applications. However, the brain presents a challenging target for gene ...
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