Prosensa closely collaborates with key academic centers and patient advocacy groups to achieve its objective of bringing innovative treatments to patients as quickly as possible.
Interested in a partnership?
Contact Luc Dochez, Chief Business Officer & SVP Business Development, for more information about partnerships.
Leiden University Medical Center (LUMC)
In 2003, Prosensa obtained an exclusive world-wide license from LUMC (Leiden, the Netherlands) for the application of its proprietary RNA modulation technology in the field of neuromuscular diseases such as muscular dystrophies. The technology was developed by the DMD genetic therapy group (Dr. Judith van Deutekom and Dr. Annemieke Aartsma-Rus) at the Human Genetics department of Prof. Dr. Gert-Jan van Ommen. In 2009, Prosensa strengthened this strategic alliance with LUMC through the extension of this license agreement. This recent extension gives Prosensa now the rights to apply the RNA modulation technology for the development of treatments for indications outside the field of neuromuscular disorders as well. In addition, Prosensa closely collaborates with LUMC in a variety of research project which explore such applications.
Nijmegen Centre for Molecular Life Sciences (NCMLS)
The Nijmegen Centre for Molecular Life Sciences (NCMLS) concentrates expertise in molecular life science research from the University Medical Centre and the Science Faculty of the Radboud University. One of the main focuses of its Department of Cell Biology is signalling cascades in human disease, with emphasis on Myotonic Dystrophy. The department was one of the groups who discovered that Myotonic Dystrophy type 1 (DM1 or Steinert’s disease) is caused by an expanding trinucleotide (CTG∙CAG)n repeat in the 3’ noncoding region of the DM protein kinase gene, and published a study on our lead compound for DM1 (Mulders et al., PNAS 2009, Mulders et al., 2010). Based on these first results additional studies were initiated to investigate specific properties that (CUG)n-directed AONs require for effective knockdown of expanded DMPK RNA. AON length and nucleotide chemistry appeared to be major parameters for therapeutic efficacy (Gonzáles-Barriga et al., Molecular Therapy-Nucleic Acids, 2013)
TREAT-NMD is a Network of Excellence facilitating collaborative research in neuromuscular disease that aims to create the infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. One of the key TREAT-NMD infrastructures built up since its inception in 2007 is a global patient registry for DMD and SMA comprising more than 30 national patient registries worldwide. In 2009, Prosensa successfully completed a feasibility enquiry using the TREAT-NMD Global Database for DMD and the TREAT-NMD Care and Trial Registry for the planning of the Phase III study for its lead compound drisapersen (previously PRO051/GSK2402968).
Patient Organization Partnerships
Prosensa has strong relationships with various patient organizations. We work together to achieve our common goal: to make treatments available to patients as quickly as possible.
Created in 1958 by a group of patients and their families, and recognized as being of public utility in 1976, AFM (French Muscular Dystrophy Association) has a single objective: to defeat neuromuscular diseases which are devastating muscle-wasting diseases. It has set itself two missions: curing neuromuscular diseases and reducing the disabilities they cause.
Aktion Benni & Co e.v.
After Conny and Claus Over (Kreis Neuwied, Rheinland-Pfalz, Germany), parents of Benni, had been confronted with the diagnosis of their son´s disease, they decided to take action against this desperate situation. They founded "Aktion Benni & Co" in June 1996, in order to intensify investigation to cure Duchenne Muscular Dystrophy and to provide financial support. Many affected families from the whole of Germany have joined them from that moment.
Charley’s Fund directs resources into the hands of researchers who have the best shot at developing a treatment or cure for Duchenne Muscular Dystrophy. Their goal is to cure DMD in time to save Charley’s life and the lives of thousands of boys like him.
CureDuchenne is a non-profit organization founded in 2003 by Debra and Paul Miller, parents of a Duchenne boy. CureDuchenne’s vision is to cure Duchenne Muscular Dystrophy. CureDuchenne aggressively seeks the most promising, leading edge research breakthroughs and expedites them to the clinical trial process. The ultimate goal is give the boys that have Duchenne now a chance to live a normal life by expediting the cure and/or the availability of therapies that can give quality of life to Duchenne boys, much like insulin does for diabetics.
Duchenne Children’s Trust
The Duchenne Children’s Trust was set up by Emily and Nick Crossley, after their son, Eli was diagnosed with Duchenne Muscular Dystrophy. The mission quite simply is to raise money to give to the best global research effort – to fund a treatment or cure in time to save Eli’s life and the hundreds of thousands of other boys like him.
Duchenne Parent Project
A Dutch foundation founded by parents of children that suffer from Duchenne Muscular Dystrophy in the Netherlands, aiming at research acceleration in order to cure or to find a treatment for Duchenne Muscular Dystrophy and to provide information to everybody involved with Duchenne Muscular Dystrophy patients.
Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne. We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community. Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives.