CRISPR base editing has delivered a landmark n-of-1 gene therapy for an infant with carbamoyl phosphate synthetase 1 (CPS1) deficiency, in a clinical milestone that may redefine how rare genetic diseases are treated. CPS1 deficiency is a mitochondrial disorder caused by a loss-of-function mutation in the CPS1 gene that impairs the urea cycle and leads to life-threatening hyperammonaemia.
GSK Plc is to expand its portfolio of products for the treatment of liver disease with the acquisition of an asset to treat and prevent the progression of steatotic liver disease (SLD), formerly known as fatty liver disease. Announced on 14 May, the deal features an upfront payment of $1.2 billion for the asset with potential for additional success-based milestones of $800 million. The liver disease affects a significant percentage of the global population and is mainly driven by obesity, type 2 diabetes, and alcohol intake, according to a 2024 review in The Lancet.
Lars Fruergaard Jørgensen, chief executive of Novo Nordisk A/S, who has led the Danish company for eight years including a period of strong growth with the launch of two obesity drugs, is to step down. The management change was apparently taken under pressure from the Novo Nordisk Foundation which controls a majority of the company’s shares. It follows a steep decline in the company’s shares after Phase 3 data for a new obesity compound failed to meet investor expectations. It did however meet the trial’s primary endpoint.
Targeted protein degradation
Scientists are looking inside the cell
for new drug targets
By Jean-Claude Muller
Research Strategy
The quest for a new RNA
vaccine for Nipah virus
Interview with CEPI
Public Policy
EU outlines strategy on AI
AI pathology tool qualified
Company News - Financing
Isomorphic Labs raises $600 million
Augustine Therapeutics gets Series A
Company News
GSK gets rights to brain shuttle
Regulation and Policy
Haemophilia drug approved
FDA to phase out animal testing
Stock Market Performance
Dismal Q1 for European biotechs
By Robin Davison
Editorial - Stand up for the FDA
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