Novartis has acquired San Diego-based biotech Kate Therapeutics, in a bid to strengthen its position in gene therapies targeting neuromuscular diseases.
Per the deal, Kate shareholders will receive up to $1.1 billion, comprising a cash payment that was paid at closing of the transaction and additional milestone payments.
Kate’s primary AAV-based gene therapy programs include preclinical candidates for Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD), and myotonic dystrophy type 1 (DM1).
The biotech’s 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. Kate believes this approach can 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.
Earlier this week in the DMD space, Regenxbio advanced its gene therapy to pivotal stage and dosed its first patient, eying a BLA submission in 2026. The treatment, RGX-202, is designed to support the delivery and targeted expression of genes throughout skeletal and heart muscle using the proprietary NAV AAV8 vector and a well-characterized muscle-specific promoter. Incidentally, Novartis’ spinal muscular atrophy drug, Zolgensma, was the first gene therapy based on Regenxbio’s NAV technology platform to get a regulatory go-ahead when it was approved in 2021.
Subscribe to our e-Newsletters
Stay up to date with news, articles and insights relevant to cell and gene therapy development and manufacturing. Plus, get special offers from Cell & Gene Therapy Review delivered right to your inbox!
Sign up now!