The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Oxford, UK – 5 February 2025: OXB (LSE: OXB), a quality and innovation-led cell and gene therapy CDMO, today announces the appointment of Jefferies International Limited ("Jefferies") as its Joint ...

researchers used a lentiviral vector to deliver gene therapy to five patients with hemophilia A. After treatment, all five had stable levels of factor VIII in their blood, with no serious safety ...

VectorBuilder's partner, Ucello, announced that its CD19-targeting umbilical cord blood-derived allogeneic CAR-T cell therapy ...

In the original lentivirus vectors described by Naldini et al (Naldini et al., 1996a) (Naldini et al., 1996b) , the vector pCMVΔR8.2 supplies all but the HIV envelope in trans, the vector pMD.G is ...

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

There were no serious adverse events or treatment-related mortalities. Evidence Rating Level: 2 (Good) Study Rundown: Transfusion-dependent β-thalassemia (TDT) is a condition that results in iron ...

Thirty patients have received the gene therapy within a clinical trial and early-access program, and the foundation also hopes to seek approval in the US.

X-linked sideroblastic anemia (XLSA) is a rare genetic disorder caused by mutations in the ALAS2 gene, which plays a critical role in heme biosynthesis within red blood cells.

senior vice president of process and analytical development at viral vector manufacturer iVexSol, which uses its lentiviral vector technology to reduce the time and expense involved in cell and ...

Its multi-platform development approach applies the lentiviral vector and adeno-associated viral vector gene therapy platforms. Its clinical program is an LVV-based gene therapy for the treatment ...