Serepta, Prosensa to Present on DMD Research at Conference on RNA Therapies

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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shutterstock_134734202Biopharmaceutical company Prosensa announced that they will present at the 6th annual conference on RNA Therapeutics, which will take place between February 16 and 17, 2015 in London, and will discuss the latest developments of its investigational drug for the treatment of Duchenne muscular dystrophy. Prosensa, which earlier last week was acquired by U.S. drug manufacturer BioMarin, will be joining another promising DMD drug developer, Serepta Therapeutics, at the conference.

Prosensa is currently working on therapies for DMD, and their company’s research has been particularly important in advancing RNA-based medications that are able to target an abnormality in part of the dystrophin gene. Similarly, both Prosensa’s and Sarepta Therapeutics’ research is focused on targeting abnormalities in exon 51, and if they succeed in their research and development, it may impact the lives of about 13 percent of the patients suffering from Duchenne — a subject that both companies will address at the conference.

The presentation, entitled Exon Skipping in Duchenne Muscular Dystrophy, will be hosted by Prosensa’s Chief Medical Officer and SVP of R&D, Giles Campion, who will be discussing the company’s clinical programs and providing guidance on accelerating regulatory paths to approval. Campion will also be leading a round table discussion on the development of RNA-based therapies for the treatment of rare diseases.

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Sarepta will present a panel entitled From RNA splicing to Eterplirsen trials — a drug for Duchenne muscular dystrophy, which will be hosted by prominent scientist Ryszard Kole. The investigator will be presenting the company’s findings from a recent trial, as well as addressing questions such as the invention of RNA splicing and the assessment of Eterplirsen induced exon skipping of 51 in dystrophin pre-mRNA in DMD patients.

BioMarin acquired Prosensa in a $680 million deal, which includes two additional payments of approximately $80 million, pending regulatory approvals of the medication being currently developed by Prosensa for the treatment of Duchenne. Prosensa is currently one of the few biopharmaceutical companies in the world dedicated to creating therapies targeting the progressive muscle-wasting disorder.

In addition, the company remains dedicated to raising awareness about the disease, and has partnered with the national nonprofit organization CureDuchenne to present their collaborative project at the 6th annual Partnering for Cures event. Together with 1,000 medical research leaders, investors, and decision-makers, the event was organized to enhance partnerships to accelerate and improve outcomes from research on the disease.