RARE Daily

CureDuchenne Ventures Invests in Dyne Therapeutics to Advance Its DMD Therapies

March 31, 2020

Rare Daily Staff

CureDuchenne Ventures has made an equity investment in Dyne Therapeutics to support Dyne’s development of precision therapeutics designed to restore muscle health in people living with Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle deterioration and weakness that primarily affects boys with symptoms beginning as early as three years of age. It is the most common type of muscular dystrophy, and is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas of the body. The condition is universally fatal, and death usually occurs before the age of 30 generally due to respiratory or cardiac failure. DMD occurs in about one out of every 3,600 male infants worldwide.

Dyne’s FORCE platform enhances the delivery of exon skipping therapeutics to skeletal, cardiac and smooth muscle with the potential to improve efficacy and reduce dosing frequency. The company’s DMD therapeutic molecule consists of an antibody linked to an exon skipping oligonucleotide. The antibody binds to specific receptors that are highly expressed on the surface of muscle cells. Once a receptor is engaged, the antibody delivers the exon skipping oligonucleotide inside the cell, enhancing mRNA modification in the nucleus and increasing dystrophin production. This approach is designed to promote muscle health and stop the progressive loss of muscle function that characterizes DMD.

CureDuchenne uses a venture philanthropy model to fund research, early diagnosis, and community education aimed at improving and extending the lives of patients with DMD.

“We know that progress comes from approaching research from multiple angles, and believe that Dyne’s FORCE platform holds substantial promise for advancing precision exon skipping therapies that are targeted to reach affected muscles throughout the body,” said Debra Miller, founder and CEO of CureDuchenne.

Photo: Debra Miller, founder and CEO of CureDuchenne


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