NEW YORK – Novartis on Thursday announced it has acquired Kate Therapeutics to bolster its capabilities to develop gene therapies for inherited neuromuscular diseases.
In the transaction valued at $1.1 billion, Kate Therapeutics' shareholders received an undisclosed amount of cash from Novartis once the deal closed, and they are also eligible to receive additional milestone payments.
In exchange, Novartis is gaining Kate Therapeutics' preclinical adeno-associated virus-based gene therapy candidates for Duchenne muscular dystrophy, facioscapulohumeral dystrophy, and myotonic dystrophy type 1. Novartis will also obtain Kate Therapeutics' Directed Evolution of AAV Capsid Leveraging In Vivo Expression of Transgene RNA, or DELIVER, platform, which enables the creation of functional capsid variants via a diverse capsid library, transcript-based in vivo selection, and machine learning. San Diego-based Kate Therapeutics has used DELIVER to advance a novel class of liver de-targeted muscle-tropic capsids that can transduce skeletal muscle and cardiac tissue but avoid 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," Novartis said in a statement announcing the acquisition. "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."
Novartis already markets Zolgensma (onasemnogene abeparvovec), a gene therapy for spinal muscular atrophy, a nerve condition due to SMN1 gene mutations that leads to muscle wasting and weakness. By acquiring Kate Therapeutics, Novartis expects to further strengthen its presence in the gene therapy market for neuromuscular conditions.
"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," Robert Baloh, global head of neuroscience research at Novartis, said in a statement. "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."