• 29 Jan 2021
• Admin
• News Article

Dynacure Receives US Patent For Investigational Antisense Product Candidate, DYN101 To Treat Myotubular And Centronuclear Myopathies

Dynacure, a clinical-stage company, announced that the US Patent and Trademark Office (USPTO) have granted US Patent No. US 10, 865, 414 related to DYN101, an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein (DNM2), for the treatment of Myotubular and Centronuclear Myopathies (CNM).The US patent is part of a patent family, exclusively licensed from Ionis Pharmaceuticals, which covers modulators of DNM2 expression. This patent family includes one additional US pending application and one European application and 18 other foreign pending applications (Australia, Brazil, Canada, Chili, China, Colombia, Israel, India, Japan, Korea, Mexico, Malaysia, New-Zealand, Peru, Russia, Singapore, South Africa and Taiwan). Patents issuing from the pending applications in this family, if any, are expected to expire in 2039, exclusive of possible patent term adjustments or extensions or other forms of exclusivity.“Today, there are no FDA- or EMA-approved medicines to treat the underlying cause of CNM. We believe that using an antisense oligonucleotide to modulate the disease-driving protein, DNM2, is the preferred modality to provide therapeutic benefit to patients,” said Frederic Legros, chief operating officer of Dynacure. “Designing and developing the right therapy with the potential to reverse the progression of disease has been an important challenge, which required the expertise of our strategic partner, Ionis Pharmaceuticals. We are pleased to expand our IP footprint with today's grant for DYN101 in the United States, which if approved, could provide the first disease-modifying treatment for a majority of patients living with CNM.”The US patent announced today expands Dynacure's existing intellectual property estate covering claims to modulate the DNM2 protein for the treatment of CNM, which includes four issued patents in the US, China, Europe and Japan, one US pending application, and five foreign pending applications in Australia, Brazil, Canada, India, and Japan.Myotubular and Centronuclear Myopathies (CNM) are serious, rare, life-threatening disorders that affect skeletal muscles from birth. CNM derives its name based on the central location of the muscle fiber nucleus, which is an abnormal finding observed in muscle biopsies. People with CNM begin experiencing muscle weakness at any time from birth to early adulthood and many patients die within the first 18 months of life. Patients who survive longer require intense medical management and nearly uninterrupted support, including permanent ventilation, brace with head support and feeding tubes.DYN101, an investigational antisense oligonucleotide product candidate using Ionis Pharmaceuticals' proprietary antisense technology, is designed to reduce the expression of dynamin 2 protein (DNM2) for the treatment of Myotubular and Centronuclear Myopathies (CNM). Preclinical studies have shown that DYN101 has the potential to be disease modifying in CNM, with preclinical activity observed in animal models of XLCNM and ADCNM.