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Leiden, Netherlands

Prosensa is a biotechnology company engaged in the discovery, development and commercialization of RNA-modulating therapeutics. The company targets genetic disorders with a large unmet medical need, with a primary focus on neuromuscular and neurodegenerative disorders such as Duchenne Muscular Dystrophy , Myotonic Dystrophy and Huntington's disease.Prosensa's portfolio of clinical and pre-clinical RNA-based drug candidates is currently focused on the treatment of DMD. Prosensa’s lead product, drisapersen, has completed Phase III clinical trials and the company intents to submit a New Drug Application to the Food & Drug Administration later this year. Three additional products, PRO044, PRO045 and PRO053 are in clinical development, and PRO052 and PRO055 are in advanced preclinical development. In parallel, Prosensa has also advanced program called PROSPECT into pre-clinical testing, which includes a new and innovative application of its exon-skipping technology platform, applying multiple exon skipping, to specifically target rarer mutations in the dystrophin gene. This approach could have applicability between 5-13% of the DMD population.In addition to its clinical trials, Prosensa is running a Natural History Study, which has completed enrollment with 269 patients across the US, Europe and Latin America. The purpose of this study is to characterize the natural history and progression of DMD, to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.Prosensa commenced operations in 2002 and is located in Leiden, The Netherlands. The company works closely together with academia, patient groups and experts worldwide. Prosensa entered into an exclusive licensing agreement in 2003 with Leiden University Medical Center for their proprietary RNA modulation exon-skipping technology to develop treatments for DMD, other neuromuscular disorders and indications outside the field of neuromuscular disorders.In November 2014, BioMarin Pharmaceutical agreed to pay up to $840 million to acquire Prosensa. Wikipedia.


The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.


The current invention provides for methods and medicaments that apply oligonucleotide molecules complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to modified oligonucleotides which can be applied in method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.


The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing an isolate muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in the method.


The invention relates to an oligonucleotide and to a pharmaceutical composition comprising said oligonucleotide. This oligonucleotide is able to bind to a region of a first exon from a dystrophin pre-mRNA and to a region of a second exon within the same pre-mRNA, wherein said region of said second exon has at least 50% identity with said region of said first exon, wherein said oligonucleotide is suitable for the skipping of said first and second exons of said pre-mRNA, and preferably the entire stretch of exons in between.


The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing and/or delaying DMD or BMD.

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