Novartis said its RNA therapy del-brax met goals of a Phase 1/2 study in facioscapulohumeral muscular dystrophy (FSHD), an inherited muscle disorder that currently has no FDA-approved medicines. Del-brax is one of three RNA therapies from Novartis’s $12 billion acquisition of Avidity Biosciences.
Avidity Biosciences brings to Novartis three RNA therapies in pivotal testing for rare neuromuscular diseases along with the technology that produced them. This platform develops drugs in a new class of targeted medicines offering potential to expand the delivery of RNA treatments beyond the liver.
By reducing administrative burden and redesigning workflows around human needs, it creates space for what matters most: connection between clinicians and patients.
Avidity Biosciences’ RNA therapy for facioscapulohumeral muscular dystrophy has preliminary clinical data showing it knocked down expression of a gene that causes this rare disease. More details will be presented at a medical conference this week.
Sarah Boyce knows she is a rare breed - a female biotech CEO. In this episode, Boyce talks about the novel therapy she is trying to develop to tackle a rare genetic muscle condition, while also building a company that is committed to diversity and inclusion.