Author: Willem van Lawick

Leiden, The Netherlands, Oct. 10, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that it has commenced the submission process for a New Drug Application (NDA) regulatory filing to the United States Food and Drug Administration (FDA) for its lead exon-skipping drug candidate, drisapersen, for treating Duchenne muscular dystrophy (DMD). The drug received “Fast Track status” from the FDA, making it eligible for a rolling review of an NDA and was also granted “Breakthrough Therapy Designation” in June 2013. (more…)

Groningen, the Netherlands, September 30, 2014 – Mucosis B.V., a clinical stage biotechnology company developing novel vaccines for infectious diseases, will present updates on its research and development program at the Biotech in Europe Forum taking place in Basel, Switzerland 30 September through 01 October 2014.
Mucosis, which has recently announced several strategic partnerships including one with the Netherlands Enterprise Agency, an agency of the Dutch Ministry of Economic Affairs, and another with China-based Changchun BCHT Biotechnology Co (BCHT), is advancing its lead product, SynGEM®, as a vaccine candidate for Respiratory Syncytial Virus (RSV). (more…)

Leiden, The Netherlands, Sept. 26, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that it has extended its comprehensive program of re-dosing of drisapersen in patients with Duchenne muscular dystrophy (DMD) into Europe, with the PRO051-02/DMD114673 sites re-opening, beginning with Belgium. This follows quickly on the re-dosing program that began in the United States (US) last week. (more…)

Leiden, The Netherlands, Sept. 24, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that the results of its research into developing an accurate and reproducible method for the measurement of dystrophin in patients with Duchenne muscular dystrophy (DMD) and Becker’s muscular dystrophy (BMD) have been published in the online peer reviewed journal PLOS ONE (http://bit.ly/ZJ8ShM).
The publication, by Chantal Beekman et al from Prosensa, describes the Company’s semi-automated image analysis method, which was shown to be objective (operator independent), reproducible (in multiple samples and experiments) and sensitive for assessing dystrophin levels by immunofluorescence in muscle biopsies from BMD and DMD patients in natural history studies or clinical studies with compounds aiming to restore dystrophin expression. (more…)

Leiden, The Netherlands, Sept. 17, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that a comprehensive program of re-dosing has commenced, with the first patients now re-dosed in the United States. All dosing in the drisapersen clinical program had been placed on hold by GSK on September 20, 2013, upon announcement of the DEMAND III study results.
The re-dosing program in North America will include up to 72 patients across 14 sites who had participated in the drisapersen DEMAND V (Phase II) & DEMAND III (Phase III) studies. Prosensa regained the rights to drisapersen from its previous partner GSK in January 2014, and soon thereafter committed to re-dose patients as quickly as possible, accepting that the transfer of data and clinical materials, and the re-engagement with clinical sites would be a complex process. (more…)