Pfizer has ended the global commercialisation of its haemophilia B (factor IX deficiency) gene therapy Beqvez, citing soft ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Fuse Vectors has raised $5.2 million of funding as it moves closer to commercialising its innovative new approach to ...

Danish start-up Fuse Vectors has received $5.2 million in preseed financing to further develop their gene therapy technology.

COPA syndrome causes lung hemorrhaging starting in childhood. But one in three people with the disease mutation are spared.

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored ...

An Italian foundation that helped a young boy overcome Wiskott-Aldrich syndrome becomes the first non-profit in the world to ...

A single injection of a healthy gene allowed blind children to recognize objects, shapes, and even read — offering hope for ...

AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...

and who do not have neutralizing antibodies to the adeno-associated virus serotype Rh74var capsid. The gene therapy had a list price of $3.5 million per dose at launch. Support for the approval ...

Gene therapy, the idea of fixing faulty genes ... activity and is small enough to be packaged within a non-pathogenic virus that can deliver ...

An experimental trial of gene therapy has helped four toddlers - born with one of the most severe forms of childhood ...