Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy.

Lentiviral vectors effectively deliver the gene that encodes coagulation factor IX (FIX) to the liver in dogs with naturally occurring hemophilia B (factor IX deficiency), researchers report in an ...

AGC Biologics, your friendly CDMO expert, announced an agreement to provide comprehensive development and manufacturing for Rarity PBC's gene therapy.

Lentiviral vectors have emerged as indispensable tools in gene therapy, offering the ability to integrate therapeutic genes into both dividing and non-dividing cells. Their unique capacity to achieve ...

Viral vectors are indispensable tools for advancing gene therapy discovery and development. As researchers progress towards clinically relevant processes, scaling up production and clarification of ...

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

Rocket Pharmaceuticals (NASDAQ:RCKT) added ~11% in the premarket on Tuesday after the company announced that the FDA had ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

Two recent studies identified gene knockouts that may make CAR T-cell therapy more effective, but the next step is applying the hypotheses in humans.

Rocket Pharmaceuticals ( ($RCKT) ) has shared an announcement. On October 14, 2025, Rocket Pharmaceuticals announced that the FDA accepted the ...