Sarepta Therapeutics (SRPT) [stock_quote symbol=”SRPT”] reported promising results on Tuesday from an early-stage study testing its gene therapy in patients with Duchenne muscular dystrophy (DMD), driving the company’s shares up 60 percent. Sarepta said data from the three-patient study showed an 87 percent mean reduction in serum creatine kinase levels, which is used as a preliminary diagnosis tool for DMD.
If you recall we highlighted SRPT in our Morning Coffee segment a few days ago. This is exciting news for SRPT and patients suffering from DMD.[finviz ticker=SRPT]
About Duchenne’s muscular dystrophy
Duchenne’s muscular dystrophy is a devastating, progressive, X-linked muscle-wasting disease. It is the most common form of muscular dystrophy, affecting 1 in 3500 boys. With an onset in early childhood, the disease progresses to final stages that are characterized by cardiorespiratory failure and death, which usually occurs in the teen age years or early 20s. The disease affects striated muscles of the limbs, diaphragm, and heart and is associated with a progressive loss of muscle mass, leading to impaired ambulation and paralysis. Skeletal myofibers degenerate and undergo necrosis; muscles progressively accumulate calcium deposits and are replaced by connective tissue and fat. source
About Sarepta Therapeutics (SRPT)
Sarepta Therapeutics, Inc. focuses on the discovery and development of RNA-based therapeutics, gene therapy, and other genetic medicine approaches for the treatment of rare neuromuscular diseases. The company offers EXONDYS 51, a disease-modifying therapy for the treatment of duchenne muscular dystrophy (DMD), which is a rare genetic muscle-wasting disease caused by the absence of dystrophin. It also provides Golodirsen, a product candidate that binds to exon 53 of dystrophin pre-mRNA, which results in exclusion or skipping of exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping; and Casimersen, a product candidate that uses phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 45 of the DMD gene. In addition, the company provides SRP-5051, a peptide conjugated PMO that binds to exon 51 of dystrophin pre-mRNA resulting in exclusion of exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. The company has strategic alliances with Nationwide Children’s Hospital for the advancement of micro-dystrophin gene therapy program under the research and license option agreement, as well as Galgt2 gene therapy program under the license agreement; and Genethon for the advancement of micro-dystrophin gene therapy program under a research and exclusive license option agreement. It also has a research and license option agreement with Duke University for the advancement of gene editing CRISPR/Cas9 technology for muscular dystrophy; and a collaboration and license agreement with Summit (Oxford) Ltd. to commercialize products in Summit’s utrophin modulator pipeline. The company distributes its products through a network of specialty distributors and specialty pharmacies in the United States, as well as through distributors internationally. Sarepta Therapeutics, Inc. was founded in 1980 and is headquartered in Cambridge, Massachusetts. Source