Girl born with MLD cured after receiving gene therapy treatment developed by Orchard Therapeutics

A girl born with metachromatic leukodystrophy (MLD) is the first person to be cured on the NHS with help from a revolutionary gene therapy, Libmeldy, developed by UCLB spinout, Orchard Therapeutics.

MLD is a rare hereditary disease that causes the destruction of the protective fatty layer surrounding the nerves in the central nervous system and the peripheral nervous system. The life expectancy of those born with MLD is usually short.

Orchard Therapeutics develop potential cures for genetic and severe diseases. In 2018, Orchard announced the closing of a $225m IPO to help transform the lives of patients with serious and life-threatening rare diseases.

This treatment has been 20 years in the making, and Orchard – which was established in 2015 – has been supported by UCLB throughout its commercialisation journey, helping to bring these life-changing treatments to the end user.

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