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 neuromuscular diseases, in a transaction valued at $1.1 billion, comprising an upfront payment and potential additional milestone payments.
 
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 the potential to treat complex diseases previously difficult to address with current technologies, including inherited neuromuscular diseases. 
 
“We are delighted to bring Kate Therapeutics’ platform technologies and programs together with Novartis scientific expertise and leadership in gene therapy and neuroscience innovation,” said Fiona Marshall, President of Biomedical Research at Novartis. “We have been highly impressed with the rigor and potential of Kate’s science, and we are confident this acquisition will further enhance our ability to bring forward new therapeutic options for patients living with neuromuscular diseases.”
 
“This acquisition builds on our expertise and leadership in neuroscience drug discovery and brings to Novartis talent, expertise and capabilities that are highly complementary to our ongoing internal efforts,” said Robert Baloh, Global Head of Neuroscience Research at Novartis. “It reflects our commitment to addressing unmet medical needs in neuroscience and tackling the limitations of existing gene therapies for patients with inherited neuromuscular conditions.”