According to a story from CureDuchenne, the biotechnology company Dyne Therapeutics recently announced that CureDuchenne Ventures has made an equity investment that will support the development of all new precision therapies to treat Duchenne muscular dystrophy (DMD), a rare neuromuscular disorder. Dyne is focused on the development of treatments for serious muscle diseases. Part of the impetus for this move is Dyne’s proprietary FORCE™ platform, which has the potential to boost the effectiveness and efficiency of an approach called exon-skipping, which is already being used to treat this disease.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy is a neuromuscular disease, and it is one of the more severe types of muscular dystrophy. It is characterized by progressive muscle weakness that usually begins around age four and worsens quickly. As an X-linked genetic disease, boys are mostly affected, with girls only occasionally displaying mild symptoms. The disease is caused by mutations of the dystrophin gene. 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. Treatment includes a variety of medications and therapies that can help alleviate symptoms and slow disease progression. 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.
Exon-Skipping and the FORCE Platform
The FORCE platform is currently the most eye-catching innovation coming out of Dyne Therapeutics. It has been designed from the ground up to improve the delivery of exon-skipping medicines to the muscles, including skeletal muscles, smooth muscle, and cardiac muscle. This could improve the effectiveness of exon-skipping and reduce the frequency of dosing. It is designed to function with a therapeutic molecule developed by Dyne which is comprised of an exon-skipping oligonucleotide linked to an antibody. Ultimately, the developers hope that it will be effective enough to halt the progression of muscle atrophy.
A treatment that could halt the progression of Duchenne muscular dystrophy would be a major breaktrough. The approach is expected to be tested in clinical studies soon.
“CureDuchenne Ventures is delighted to support Dyne as part of our comprehensive approach to finding a cure for Duchenne.” – Debra Miller, founder and CEO of CureDuchenne
“We appreciate the collaboration with and investment from CureDuchenne Ventures, which will help us advance our novel approach to targeting the genetic basis of DMD.” – Joshua Brumm, president and CEO of Dyne Therapeutics
To learn more about CureDuchenne, click here.