Swiss pharmaceutical company Novartis (NYSE: NVS) has announced the acquisition of US-based Kate Therapeutics, Inc., a pre-clinical biotech firm specializing in adeno-associated virus (AAV)-based gene therapies for muscle and heart diseases, for a substantial USD 1.1 billion. This strategic move allows Novartis to gain access to Kate’s pre-clinical gene therapy pipelines, which include treatments for Duchenne muscular dystrophy (DMD), facioscapulo-humeral muscular dystrophy (FSHD), and myotonic dystrophy type 1 (DM1), among others.
Kate’s Innovative DELIVER Platform
Kate Therapeutics prides itself on its Directed Evolution of AAV Capsid Leveraging In Vivo Expression of Transgene RNA (DELIVER) platform, a cutting-edge technology that combines diverse capsid library generation with transcript-based in vivo selection and machine learning. This advanced platform is designed to evolve functional capsid variants, leading to the development of a novel class of liver de-targeted muscle-tropic capsids. These capsids are capable of transducing both skeletal muscle and cardiac tissue with high potency and selectivity in vivo, while deliberately avoiding the liver.
Implications for Novartis and the Future of Gene Therapy
The acquisition of Kate Therapeutics and its DELIVER platform significantly strengthens Novartis’s position in the gene therapy space. It not only expands the company’s pipeline of potential treatments for debilitating muscle and heart diseases but also adds a powerful technology platform to its arsenal. This move is expected to accelerate the development of new therapies and further establish Novartis as a leader in the field of gene therapy.-Fineline Info & Tech
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