Novartis has acquired Kate Therapeutics, a San Diego, California-based gene-therapy company for treating genetically defined neuromuscular diseases, in a $1.1-billion deal (undisclosed upfront payment and additional milestone payments) 

Kate Therapeutics’ primary programs include preclinical candidates for several rare neuromuscular diseases: Duchenne muscular dystrophy, a severe, inherited disease that causes progressive muscle weakness and degeneration; facioscapulohumeral dystrophy, a genetic muscle disorder;  and myotonic dystrophy Type I, a genetic disorder that affects multiple systems in the body, including the muscles, heart, brain, and eyes. 

The company has a proprietary technology platform (Directed Evolution of AAV Capsid Leveraging In Vivo Expression of Transgene RNA [DELIVER]) based on capsid variants of adeno-associated viruses (AAVs) that enable delivery to desired tissue while potentially mitigating off-target effects to tissues such as the liver. The platform has been used to evolve a class of liver de-targeted muscle-tropic capsids, which transduce both skeletal muscle and cardiac tissue in vivo while avoiding the liver. 

Under the deal, Kate Therapeutics’ shareholders are entitled to receive up to $1.1 billion, comprising a cash payment that was paid at closing of the transaction and additional amounts payable on achievement of specified milestones. 

Source: Novartis