Lead candidates are in clinical development for Duchenne muscular dystrophy.

Prosensa raised €23 million in equity financing led by new investor New Enterprise Associates. Existing investors in the firm also participated. Prosensa says it aims to use the funds to progress its pipeline of RNA-modulating drugs for neuromuscular diseases including Duchenne muscular dystrophy (DMD), myotonic dystrophy (DM1), and Huntington disease (HD). Lead DMD candidate PRO051/GSK2402968 is undergoing Phase III evaluation in partnership with GlaxoSmithKline.

“This financing will help us to further strengthen our position in rare diseases and will allow us to deliver on our promise of accelerated development of treatment for patients in need,” remarks Hans Schikan, Prosensa CEO. “We have advanced the development of five additional compounds in DMD and have announced preclinical testing for a compound for DM1.”

Prosensa is focused on the development and commercialization of RNA-modulating therapeutics for genetic disorders.The firm is exploiting a technology platform based on the use of single-stranded RNA-based antisense oligonucleotides (AONs) to correct mutated mRNAs. It claims that in contrast with double-stranded RNA molecules such as RNAi, the AON technology, offers allele-specific (re)engineering of RNA processing. Resulting AON compounds can thus be designed to interfere with splicing to induce exon skipping, enhance exon inclusion or correct splicing mutaitons, remove mutant RNA or protein domains, or block RNA expression.  

Lead DMD candidate PRO051/GSK2402968 is designed to induces exon 51 skipping in the DMD gene, which could in principle correct the gene’s reading frame in about 13% of all DMD patients including patients with deletions of exon 50, 52, 45–50, 48–50, and 49–50. A second DMD candidate, PRO044, is being evaluated in a Phase I/IIa dose-escalating trial. The candidate has been designed to specifically induce exon 44 skipping in the DMD gene, which Prosensa says could be applicable to about 6% of all DMD patients including those with deletions in exons 43, 45, 38–43, 40–43, 42–43, and 45–54. Last month the firm received $1.5 million in financing from the charitable organization Charley’s Fund, to support development of exon 52-skipping DMD candidates.

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