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Novartis, assessing peers’ muscular dystrophy failures, pens $1.1B deal for gene therapy biotech

Novartis had one eye on the struggles its peers have encountered in the muscular dystrophy space when it decided to acquire Kate Therapeutics, according to the Big Pharma’s CEO. The Swiss drugmaker signed off on $1.1 billion in a combination of upfront cash and potential milestone payments to buy the gene therapy biotech, it announced today. San Diego-based Kate is focused on preclinical programs aimed at Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD) and myotonic dystrophy type 1 (DM1). These programs have been developed using the biotech’s Directed Evolution of AAV Capsid Leveraging In Vivo Expression of Transgene RNA (DELIVER) platform to produce “liver de-targeted muscle-tropic capsids, which transduce both skeletal muscle and cardiac tissue with potency and selectivity in vivo, while avoiding the liver,” according to a Nov. 21 release. This year has been a stark reminder of what tricky indications muscular dystrophies can be. Pfizer suffered a $230 million financial hit over the summer when its DMD gene therapy flunked a phase 3 trial while Fulcrum Therapeutics’ losmapimod encountered its own late-stage failure just four months after Sanofi bet $80 million on the FSHD candidate.

Full report : Novartis, assessing peers’ muscular dystrophy failures, pens $1.1B deal for gene therapy biotech.