Dynacure, a clinical stage drug development company focused on improving the lives of patients with rare and orphan disorders, today announced that Belinda Cowling, Ph.D., Chief Scientific Officer and co-founder of Dynacure, has received the 2019 Irène Joliot-Curie Prize for Women in Business and Technology. Dr. Cowling was honored for her work in the field of neuromuscular disease research, her leadership role in the development and advancement of research performed at the Institute of Genetics and Molecular and Cellular Biology (IGBMC) in France, and the creation of Dynacure.
The Irène Joliot-Curie Prize for Women in Business and Technology is given to one researcher each year in France who devotes herself to developing scientific and technological innovations that are useful to society by working within a company or contributing to the creation of a company. The prize, is named after famous French Nobel Prize winning Scientist Irène Joliot-Curie and is awarded by the French Ministry of Higher Education Research and Innovation and Airbus, with the support of the Academies of Science and Technology. The annual prize is intended to promote women in research and technology, whose careers combine excellence and dynamism.
Dr. Cowling completed her PhD in Australia at Monash University in 2008. She then moved to the IGBMC to complete a postdoc as a member of the team of Dr. Jocelyn Laporte. There, she studied the normal biological function of dynamin 2, which is mutated in centronuclear myopathy, as well as the pathological mechanisms leading to disease. She identified down regulation of this gene as a novel therapeutic target for centronuclear myopathies1,2. Belinda’s research at the IGBMC led to the creation of Dynacure in 2016. Today, Dr. Cowling is responsible for Dynacure’s R&D strategy and leading the company’s research programs in centronuclear myopathy and other disease domains. In addition, Dr. Cowling focuses on translational research, drug-candidate development and the expansion of Dynacure’s research network with academic and industrial organizations.
Dr. Cowling has published more than 32 publications in peer-reviewed journals and has several filed and published patents. Dr. Cowling was also awarded the Scientific Prize from Strasbourg University in 2019 for scientists that show originality and vitality in the implementation of their research, and whose future developments are promising.
Dynacure’s lead program, DYN101, is an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2) for the treatment of Myotubular and Centronuclear Myopathies (CNM). CNMs are serious, rare, life-threatening disorders that affect skeletal muscles from birth. Patients become progressively limited in their abilities to run, walk, even breath and eat. Centronuclear Myopathies affect between 4,000 and 5,000 patients in the EU, US, Japan and Australia3. DYN101 is being developed in collaboration with Ionis Pharmaceuticals, the leader in RNA-targeted drug discovery. Preclinical efficacy observed in multiple mouse models of the disease gives great confidence in the ability to demonstrate potential disease-modifying results in humans. The development plan for DYN101 was designed to be very broad and it is the only known program being investigated for most CNM populations, XLCNM and ADCNM. In addition to investigating DYN101 for CNM, Dynacure aims to expand its use and explores additional indications where the overexpression of DNM2 is a disease-driving factor.
DYN101 has been granted Orphan Drug designations by the US FDA and EMA. More information can be found at https://clinicaltrials.gov/ct2/show/NCT04033159?term=dynacure&rank=1.
About Centronuclear Myopathies
Myotubular and Centronuclear Myopathies (CNM) are serious, rare, life-threatening disorders that affect skeletal muscles from birth. CNMs derive their name based on the central location of the muscle fiber nucleus, which is an abnormal finding observed in muscle biopsies. The disease is driven by mutations in multiple genes including MTM1, DNM2 and BIN1 and Dynacure scientists have discovered the link between an increase in DNM2 and the direct cause of the disease (Cowling et al 2014 JCI). There are many genetic forms of CNM including X-linked recessive (XLCNM/ Myotubular Myopathy), autosomal dominant (ADCNM), and autosomal recessive (ARCNM), which are all associated with poor prognosis. Centronuclear Myopathies affect between 4,000 and 5, 000 patients in the EU, US, Japan and Australia3.
Dynacure is a clinical-stage drug development company focused on improving the lives of patients with rare and orphan diseases. The Dynacure team leverages its proven track record in rare disease drug development to build a pipeline of novel drugs. Dynacure is developing DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 for the treatment of Myotubular and Centronuclear Myopathies, with Ionis Pharmaceuticals. Dynacure is also building a complementary research portfolio targeting other orphan disorders. The company maintains its headquarters in Strasbourg, France. Dynacure’s investors are Andera Partners, Bpifrance, IdInvest, Ionis Pharmaceuticals, Kurma Partners and Pontifax.
For more information, please visit www.dynacure.com.
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1. Nat Commun. 2017 Jun 7;8:15661. doi: 10.1038/ncomms15661.
2. Proc Natl Acad Sci U S A. 2018 Oct 23;115(43):11066-11071. doi: 10.1073/pnas.1808170115. Epub 2018 Oct 5.
3. Neuromuscul Disord. 2018 Sep;28(9):766-777. doi: 10.1016/j.nmd.2018.06.012. Epub 2018 Jul