Wnt7a-Analog Protein Therapeutic for the Treatment of Muscular Dystrophy

Peter Flynn
Fate Therapeutics Inc

21st June 2013

Fate Therapeutics presented a proposal to develop a Wnt-based protein therapeutic as a potential treatment for a broad range of muscular dystrophies and neuromuscular diseases, including Duchenne muscular dystrophy (DMD). The academic work of Dr Michael Rudnicki has characterized the role of Wnt7a signaling in satellite stem cell proliferation and skeletal muscle hypertrophy.Fate therapeutics presented data demonstrating the successful engineering of the Wnt7a protein to analogs with preferred product development characteristics. In support of potential beneficial effects for muscular dystrophy, the Wnt7a analogs were seen to induce a significant increase in the satellite stem cell population, reduced inflammation and fiber damage and significantly improve muscle strength in preclinical model systems. The company proposed a development plan including an initial human proof of concept study with targeted delivery of the Wnt7a-analog protein to specific muscle groups followed by a program expansion to systemic delivery. The TACT committee suggested that while an intramuscular administration may be useful for certain muscular dystrophies or stages of disease, true therapeutic benefit for DMD would require a systemically delivered product.

12 Apr 2017