A UK hospital is taking an unprecedented step to seek approval for an ADA-SCID gene therapy, a rare disease, after many biopharma companies have abandoned similar efforts. This initiative, led by Dr. Booth, marks a significant shift from traditional pharmaceutical company applications to a hospital-led approach. The move has sparked discussions about the NHS's systems for approving new medicines, with criticism from Tanya Curry, CEO of the Motor Neurone Disease Association, who stated, 'It’s appalling that people with a particular genetic subtype of motor neurone disease face being denied what could be a life-changing treatment because of the NHS’s outdated systems for approving new medicine.'
Dr Booth is leading the way for all genetic medicines in “rare disease” with her approach to have her hospital gain approval for the gene therapy she trialed. 👏🏻👏🏻👏🏻 https://t.co/yHAmJS4Swp
Many biopharma companies have abandoned promising gene therapies for ultra-rare diseases. Now a British hospital is trying to get one approved. https://t.co/adpUOXokGt
Why a British hospital, and not a drugmaker, is trying to get a rare disease therapy approved https://t.co/StCMN5BxMO via @statnews
"It’s appalling that people with a particular genetic subtype of motor neurone disease face being denied what could be a life-changing treatment because of the NHS’s outdated systems for approving new medicine, " said @Tanyacurry2, CEO of @mndassoc. “I would like to see an… https://t.co/BFULMWyfFi
Also interesting. Curious to see if we get more of this, including in the US @PharmaPinkSheet @Citeline ... #Pinksheet #pharma: First Of Its Kind: UK Hospital To Apply For ADA-SCID Gene Therapy Approval https://t.co/ec2tpx4D3G