In late January 2021, Dynacure, a clinical-stage drug development company focused on rare diseases, announced that it was granted a patent (U.S. Patent No. US 10, 865, 414) for its investigational drug candidate DYN101. This potential therapy is designed for patients with centronuclear myopathy, a rare condition affecting skeletal muscles. While there are multiple forms of centronuclear myopathy, this drug is designed to treat all of them, including the most common and severe form known as myotubular myopathy.
The Patent
In particular, this patent centers around the dynamic 2 protein (DNM2). DNM2 gene mutations are linked to the development of centronuclear myopathies. Thus, DYN101 works to inhibit DNM2 expression. Dynacure believes that through this, DYN101 could help modify disease progression and improve patient outcomes. DYN101 is an investigational antisense oligonucleotide product designed using Ionis Pharmaceuticals’ (“Ionis”) proprietary technology. Additionally, this patent provided to Dynacure is part of a patent family licensed by Ionis. In preclinical studies, DYN101 showed benefits for strength and overall survival rate. The investigational drug has received Orphan Drug and Rare Pediatric Disease designations within the United States, and Orphan Drug designation in Europe.
Centronuclear Myopathy
TTN, BIN1, and DNM2 gene mutations can all cause centronuclear myopathy, a rare condition characterized by muscle weakness and wasting. According to the Muscular Dystrophy Association, centronuclear myopathy is named:
for the mislocation of cell nuclei in the muscle fibers. Normally, these nuclei are arranged around the periphery of the fiber.
However, in these cases, the nucleus is located centrally instead. The Muscular Dystrophy Association also describes myotubular myopathy as one of the most common, but severe, of the centronuclear myopathies. It is congenital (present at birth) and caused by myotubularin protein deficiencies. Unfortunately, many patients do not live past childhood. Those who do often require a high level of additional support, including feeding tubes and ventilation. Currently, no approved treatments exist.
Symptoms include:
- Progressive muscle weakness
- Scoliosis
- Developmental and intellectual delays
- Difficulty breathing and other respiratory issues
- Muscle pain which worsens during exercise
- Issues with walking or other movements
- Weakness, numbness, or pain in the hands and feet
- Cardiomyopathy
- A high palate
- Droopy eyelids
Learn more about centronuclear myopathy.
