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

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

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

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

In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants ...

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine ...

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

Scientists compared five families and multiple generations to find a gene variant that prevents COPA Syndrome and opens the door to a new gene therapy for the condition.

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

Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx's investigational gene ...

A groundbreaking gene therapy has restored sight in four young children born with severe blindness due to a rare genetic ...