Prosensa Named a Fierce 15 Biotech Company ‘One of the most promis­ing pri­vate biotech­nol­o­gy com­pa­nies in the indus­try’

Leiden, The Netherlands – 19 September 2012 – Prosensa, the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA-mod­u­lat­ing ther­a­peu­tics for rare dis­eases with high unmet need, has been named by FierceBiotech as one of 2012’s Fierce 15, des­ig­nat­ing it as one of the most promis­ing pri­vate biotech­nol­o­gy com­pa­nies in the indus­try.
Prosensa’s main focus is the treat­ment of rare neu­ro­mus­cu­lar dis­eases. Drisapersen (PRO051/GSK2402968), its lead­ing ther­a­py for the treat­ment of Duchenne mus­cu­lar dys­tro­phy (DMD), is cur­rent­ly in Phase III tri­als in part­ner­ship with GlaxoSmithKline (GSK).
“When New Enterprise Associates went look­ing for its first European biotech invest­ment, they found an ide­al can­di­date in Prosensa,” says FierceBiotech Editor John Carroll. “Its Big Pharma part­ner GlaxoSmithKline is exe­cut­ing a piv­otal study on its lead drug for Duchenne mus­cu­lar dys­tro­phy, mak­ing Prosensa − which has five oth­er DMD pro­grams in the pipeline − a leader in rare dis­eases worth watch­ing close­ly. A strong invest­ment syn­di­cate gives Prosensa some real options to pon­der about its future. And we’ll be watch­ing every step of the way.”
Hans Schikan, Prosensa CEO said: “Prosensa is very pleased to have been recog­nised as a ‘fierce’ bio­phar­ma­ceu­ti­cal com­pa­ny. Thanks to our part­ner­ships with patient organ­i­sa­tions, acad­e­mia and phar­ma­ceu­ti­cal com­pa­nies, along with the sup­port of our investors, we will pas­sion­ate­ly con­tin­ue on our path of growth to become a lead­ing rare dis­ease play­er with an ini­tial focus on Duchenne mus­cu­lar dys­tro­phy, myoton­ic dys­tro­phy and Huntington’s dis­ease. Prosensa’s plat­form of RNA mod­u­la­tion can be applied to a large num­ber of unmet med­ical needs.”
Prosensa has the most advanced port­fo­lio of drug can­di­dates for the treat­ment of DMD, with two com­pounds in clin­i­cal tri­als in part­ner­ship with GSK (dris­apersen and PRO044) and four addi­tion­al com­pounds in pre­clin­i­cal devel­op­ment, as well as pre­clin­i­cal com­pounds for myoton­ic dys­tro­phy and Huntington’s dis­ease. Prosensa’s DMD com­pounds are based on its pro­pri­etary exon-skip­ping tech­nol­o­gy that uses anti­sense oligonu­cleotides to restore expres­sion of a func­tion­al dys­trophin pro­tein and to pro­vide poten­tial treat­ment for patients affect­ed by this pro­gres­sive­ly debil­i­tat­ing neu­ro­mus­cu­lar dis­ease.
This is FierceBiotech’s tenth annu­al Fierce 15 selec­tion. The Fierce 15 cel­e­brates the spir­it of being “free” – cham­pi­oning inno­va­tion and cre­ativ­i­ty, even in the face of intense com­pe­ti­tion. A com­plete list of the “Fierce 15” com­pa­nies can be found at

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