Commentary
Video
Mechanism of Action of SAT-3247 in Muscular Dystrophy: Frank Gleeson
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The president and chief executive officer of Satellos Bioscience provided insight on a new small molecule therapy, SAT-3247, in development for patients with muscular dystrophies. [WATCH TIME: 3 minutes]
WATCH TIME: 3 minutes
"There are stem cells, we discovered, do not properly divide the way they are intended to divide to create new muscle cells. As a result, we believe that the progressive damage to the muscle that is seen in muscular dystrophy is a result of their bodies not being able to keep up with the normal wear and tear plus the combined damage done by the nature of the genetic disease."
Duchenne muscular dystrophy (DMD) is a severe X-linked recessive disorder caused by mutations in the dystrophin gene and consequent complete loss of dystrophin protein expression. Due to its massive size, the mutation rate for the dystrophin gene is quite high, with about one-third of mutations occurring de novo and the remaining caused by germline mosaicism or inheritance from a carrier mother. In recent years, the advances of an understanding of the molecule pathways affected in DMD have led to the development of many therapeutic strategies that tackle different aspects of the disease etiopathogenesis, leading to the first successful approved orphan drugs for this condition.
Through the use of an in-situ muscle stem cell screening platform, Satellos Bioscience has identified a highlight druggable protein kinase target called adapter associated kinase 1 (AAK1). When inhibited, this target promotes functional rescue of asymmetric stem cell division, resulting in the robust production of progenitors in vitro and in vivo. At the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, the company presented a summary of key preclinical data on SAT-3247, its investigational small molecule inhibitor targeting AAK1.
Frank Gleeson, president and chief executive officer of Satellos Bioscience, sat down with NeurologyLive® at the meeting to discuss the mechanism of action of this agent and why it can be successful. He spoke on the molecular structure of muscular dystrophy, the previous literature on AAK1 inhibitors, and why this may be an avenue to explore.
