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.

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 ...

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 ...

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

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

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

Opportunities in viral vector manufacturing arise from rising demand for GMP-grade materials and increasing gene therapy ...

FDA accepts Rocket Pharmaceuticals' resubmission for Kresladi gene therapy in severe LAD-I, with a decision expected by March ...

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