According to a story from CRWE World, the genetic medicines company Audentes Therapeutics has recently announced its intent to develop new therapies for myotonic dystrophy and Duchenne muscular dystrophy. These therapies will be based on the advanced vectorized antisense mechanism which uses AAV vectors to deliver antisense oligonucleotides. Audentes is focused on developing treatments for rare neuromuscular illnesses and disorders.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy is a neuromuscular disease and it is one of the more severe types of muscular dystrophy. Symptoms of Duchenne muscular dystrophy include falling, abnormal walking posture, eventual loss of walking ability, muscle fiber deformities, intellectual disability (not in all cases), enlargement of the tongue and calf muscles, skeletal deformities, muscle atrophy, heart abnormalities, and difficulty with breathing. Lifespan is usually into the thirties with good care. Better treatments for this disease are urgently needed. To learn more about Duchenne muscular dystrophy, click here.
About Myotonic Dystrophy
Myotonic dystrophy is a long term disorder characterized by progressive loss of muscle function. Symptoms usually appear in early adulthood and may include myotonia, muscle loss and weakness, heart problems, cataracts, and intellectual disability. There is no cure; management may include a pacemaker to normalize heart function, mobility aids, and an exercise routine to help maintain muscle strength. There are two forms of myotonic dystrophy which are distinguished by the mutations that cause them. To learn more about myotonic dystrophy, click here.
A New Path for Treatment
Both of these illnesses urgently need new therapies that can improve the lives of the patients that live with them. Antisense therapies are a unique type of treatment that, when delivered, allow cells to skip over abnormal or mutated fragments of genetic code. This mechanism is also known as exon-skipping. In Duchenne muscular dystrophy, for example, this allows the cells affected by the treatment to express a healthy level of the dystropin protein, which is deficient in patients. Therapies using this mechanism could offer greater impact than other approaches such as gene replacement with microdystrophin.
Audentes is currently working on the development of several Duchenne muscular dystrophy therapies that will use this mechanism. Its development for myotonic dystrophy type 1 is in the earlier stages. The company is researching and comparing both vectorized exon-skipping and vectorized RNA knockdown as the potential mechanism for a future myotonic dystrophy therapy.
