Prosensa to Receive $1.5m from Charley’s Fund for Skipping of Exon 52 in Duchenne Muscular Dystrophy Patients

Leiden, The Netherlands, 1 December 2011 – Prosensa, the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA mod­u­lat­ing ther­a­peu­tics for rare dis­eases with unmet med­ical needs, announced today that it has received financ­ing of $1.5m for three years from the char­i­ta­ble organ­i­sa­tion Charley’s Fund, to sup­port devel­op­ment of com­pounds for the skip­ping of exon 52 in patients with Duchenne Muscular Dystrophy (DMD).
Under the terms of the agree­ment, Prosensa will iden­ti­fy anti­sense oligonu­cleotides for the skip­ping of exon 52 in spe­cif­ic sub­pop­u­la­tions of DMD patients. Prosensa will assess the safe­ty and effi­ca­cy of the com­pounds in suit­able pre­clin­i­cal mod­els with the aim to move a select­ed can­di­date into clin­i­cal tri­als.
“Working close­ly with DMD patient advo­ca­cy groups has been cen­tral to Prosensa’s mis­sion in iden­ti­fy­ing and devel­op­ing effec­tive treat­ments for DMD” com­ment­ed Luc Dochez, Prosensa’s Chief Business Officer. “The com­pa­ny has already received finan­cial sup­port from Charley’s Fund and oth­er groups dur­ing its ear­ly years. This addi­tion­al sup­port for the exon 52 pro­gram will enable us to accel­er­ate our efforts towards clin­i­cal devel­op­ment.”
“Since our first invest­ment in Prosensa six years ago, we have been impressed by their stead­fast progress in drug devel­op­ment for DMD,” said Benjamin D. Seckler M.D., founder and President of Charley’s Fund. “We are excit­ed to part­ner once again to help more chil­dren with DMD ben­e­fit from this promis­ing ther­a­peu­tic strat­e­gy.”

Leave a Reply

Your email address will not be published. Required fields are marked *