UK's NHS to roll out life-saving gene therapy for rare disease affecting babies

5 February 2022

UK and USA-based biotech firm Orchard Therapeutics’ (Nasdaq: ORTX) shares closed up 7.6% and rose a further 4.4% to $1.18 in after-hours trading on Friday, after the company gained clearance for Nation Health Service use in England and Wales for its stem cell therapy Libmeldy (atidarsagene autotemcel).

The NHS has struck a deal for the life-saving treatment, reportedly the most expensive drug in the world, that will offer babies and young children with a rare and fatal genetic disease the prospect of a normal life, the health service chief executive has announced.

Libmeldy, a revolutionary gene therapy treatment is used to treat metachromatic leukodystrophy (MLD), which causes severe damage to the child’s nervous system and organs, and results in a life expectancy of between just five and eight years.

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