NorthSea Therapeutics bags FDA rare paediatric disease tag for NASH drug
Pharmaceutical Technology
OCTOBER 17, 2023
The Netherlands-based company’s treatment has been awarded the designation by the US FDA following a successful Phase I trial.
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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies
Pharmaceutical Technology
SEPTEMBER 30, 2022
Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.
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First gene therapy trial for Hunter syndrome opens in Manchester, UK
Drug Discovery World
OCTOBER 31, 2023
The clinical trial will be led by Professor Rob Wynn, Consultant Paediatric Haematologist at Royal Manchester Children’s Hospital (RMCH), together with Professor Simon Jones, Consultant in Paediatric Inherited Metabolic Disease at Saint Mary’s Hospital, and Professor Bigger.
What to expect from PEGS Europe 2023: Day 2
Drug Discovery World
NOVEMBER 9, 2023
Thereafter, there will be two more presentations: John Anderson, PhD, GOSHCC Professor, Honorary Consultant Oncologist, Experimental Paediatric Oncology, University College London, on: ‘New T cell engineering approaches for mitigation of exhaustion and targeting of low antigen density’.
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