Novartis acquires Kate Therapeutics focused on developing adeno-associated virus based gene diseases
Novartis has acquired Kate Therapeutics, a San Diego-based, preclinical stage biotechnology company, focused on developing adeno-associated virus (AAV)-based gene therapies to treat genetically defined neuromuscular diseases. Kate Therapeutics’ primary programs include preclinical candidates for Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD), and myotonic dystrophy type 1 (DM1)
Kate’s Directed Evolution of AAV Capsid Leveraging In Vivo Expression of Transgene RNA (DELIVER) platform combines diverse capsid library generation with transcript-based in vivo selection and machine learning to evolve functional capsid variants. DELIVER has been used to evolve a novel class of liver de-targeted muscle-tropic capsids, which transduce both skeletal muscle and cardiac tissue with potency and selectivity in vivo, while avoiding the liver. Kate Therapeutics’ technology platforms integrate capsid and cargo technologies to deliver payloads to desired tissues, while potentially mitigating off-target effects to tissues such as the liver. This approach aims to improve both the efficacy and safety of gene therapies, opening potential possibilities for treating complex diseases previously difficult to address with current technologies, including inherited neuromuscular diseases. Under the terms of the agreement, Kate Therapeutics’ shareholders are entitled to receive up to USD 1.1bn, comprising a cash payment that was paid at closing of the transaction and additional amounts payable on achievement of specified milestones.
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