Partners
Prosensa closely collaborates with a group of strong key academic and industrial / corporate partners in order to achieve its objective of bringing innovative treatments to patients as quickly as possible.
Interested in a partnership?
Contact Luc Dochez for more information about partnerships.
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Academic Partners
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Treat-NMD
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. At the end of August 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 PRO051/GSK2402968.
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Nijmegen Centre for Molecular Life Sciences (NCMLS)
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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 recently published a study on our lead compound for DM1, a fully 2’-O-methyl-phosphorothioate modified (CAG)7 AON that silences mutant DMPK RNA expression and reduces the number of ribonuclear aggregates in a selective and (CUG)n-length-dependent manner (Mulders et al., PNAS 2009).
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Leiden University Medical Center (LUMC)
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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.
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Corporate Partners
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GlaxoSmithKline (GSK)
Prosensa and GSK entered in an exclusive worldwide collaboration for the development and commercialization of RNA based therapeutics for Duchenne Muscular Dystorphy.
The alliance was established under GSK’s Centre of Excellence for External Drug Discovery (ceedd) which seeks to collaborate with companies at the leading edge of highly innovative and transformative science. The scope of the alliance includes four RNA-based products intended to treat specific, but different, subpopulations of patients suffering from DMD.
Under the terms of the agreement, GSK will obtain an exclusive worldwide license to develop and commercialize Prosensa’s lead compound, PRO051/GSK2402968, intended to treat DMD by skipping exon 51 of the dystrophin gene. Mutations in the dystrophin gene result in the absence of normal dystrophin protein, which is necessary for proper muscle cell function. GSK’s Neurosciences Medicines Development Centre will continue to progress the further development of PRO051/GSK2402968 in collaboration with Prosensa. Both parties have begun preparations for a Phase III study which is intended to start in early 2010. GSK will fund all costs associated with the further clinical development of PRO051/GSK2402968. In addition, GSK has exclusive options to license three more RNA-based compounds targeting additional DMD exons. One such option includes Prosensa’s second lead compound, PRO044, which targets the skipping of exon 44 and for which Prosensa has initiated a Phase I/II study at the end of 2009. In this case, GSK’s option rights will be triggered by a successful completion of this study.
The financial terms include a GBP 16 million (USD 25 million) upfront payment. Furthermore, Prosensa is eligible to receive up to GBP 412 million (USD 655 million) in milestones payments if all four compounds are successfully developed and is also entitled to double-digit royalties on product sales. Prosensa will retain commercial participatory rights, and has an option to expand its commercial rights, in certain European countries on products resulting from the collaboration.
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Patient Organisation Partnerships
Prosensa has strong relationships with various patient organizations. We work together to achieve our common goal: make a therapy available for patients as quickly as possible in a reasonable and qualitative manner.
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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 heal or to find a treatment for Duchenne Muscular Dystrophy and to provide information to everybody involved with Duchenne Muscular Dystrophy patients.
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Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy’s mission is to improve the treatment, quality of life, and long-term outlook for all individuals affected by Duchenne Muscular Dystrophy (Duchenne) through research, advocacy, education, and compassion.
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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.
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CureDuchenne
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.
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Charley's Fund
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.
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AFM
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.
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