The transaction includes an upfront payment of $1.55 billion, along with up to $1.1 billion in regulatory and commercial milestone payments
It's a development that marks a potential breakthrough for people living with the rare and debilitating disease Duchenne muscular dystrophy (DMD)
KER-065 is a novel ligand trap comprised of a modified ligand-binding domain derived from activin receptor type IIA and activin receptor type IIB
Elevidys is currently the only approved gene therapy that targets the root cause of Duchenne
Capricor’s BLA for Deramiocel received Priority Review in March 2025
The primary endpoint in the final analysis was assessed by change in the North Star Ambulatory Assessment at one year after treatment
This positions its platform as a rapidly evolving engine for next-generation genetic medicine
Key FDA decisions and pivotal late-stage obesity and breast cancer data could reshape competitive positioning
Salanersen was generally well-tolerated, slowed neurodegeneration, and enabled functional gains
PBGENE-DMD is designed to permanently correct mutations in the dystrophin gene between exons 45 and 55, the “hot-spot” region affecting roughly 60% of DMD patients
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