PharmaTelevision - Luc Dochez on why Prosensa’s exon skipping therapy is personalised medicine in its ultimate form for Duchenne’s

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Video title: Luc Dochez on why Prosensa’s exon skipping therapy is personalised medicine in its ultimate form for Duchenne’s

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In this episode of PharmaTelevision News Review, Fintan Walton talks to Luc Dochez, CBO and Senior VP BD, Prosensa.

Full video transcripts are available with PharmaTelevision Premium Content. Click here to buy a subscription or sign up for a 14 day free trial.

Prosensa:Origin and Technology

Fintan Walton:

Hello and welcome to PharmaTelevision News Review here in Munich at BIO-Europe 2010. On this show I have Luc Dochez , who is the Chief Business Officer at a company called Prosensa, which is based in AL Leiden in The Netherlands, welcome to the show.

Luc Dochez:

Thank you.

Fintan Walton:

Luc Dochez , Prosensa is a company is a very specialist company has its own unique underlying technology that can be applied to specific types of diseases, can you tell us little bit about the underlying technology?

Luc Dochez:

Sure, we are focused on RNA modulations so it's a company using antisense oligonucleotides to work at the RNA level so it is not gene therapy it is genetic therapy, but we tried to correct something that went wrong at the DNA level at the RNA level to then have the positive effects that we want to have downstream at the protein level. One example of that is for example of exon skipping in which we apply duchenne muscular dystrophy which is our lead therapeutic indication and in the case of exon skipping apply skipping an exon we tried to convert them out-of-frame mutation into an in-frame mutation which then leads to a shorter but still functional protein.

Fintan Walton:

So this particular technology particularly lends itself to duchenne muscular dystrophy because of the nature of that particular disease?

Full video transcripts are available with PharmaTelevision Premium Content. Click here to buy a subscription or sign up for a 14 day free trial.

Fintan Walton

Dr Fintan Walton is the Founder and CEO of PharmaVentures . After completing his doctoral research on the genetics of cell proliferation at the University of Michigan(US)and Trinity College (Dublin, Ireland), Dr Walton gained broad commercial experience in biotechnology in management positions at Bass and Celltech plc (1982-1992).

Luc Dochez

Chief Business Officer

Luc Dochez , Chief Business Officer and Senior Vice-President Business Development joined Prosensa in November 2008 and is responsible for all business and corporate development activities at the company. He has over 10 years experience in the biotech industry and was directly involved in multiple financing rounds and partnering deals with various biotech companies. Before joining Prosensa, he was a consultant within Arthur D. Little's biotechnology practice, Director of Business Development at Methexis Genomics NV, VP Business Development at TiGenix Inc. and President of TiGenix Inc. Luc Dochez holds a PharmD degree from the University of Leuven (Belgium), a postgraduate degree in Business Economics from the same university and an MBA from Vlerick Management School.

PharmaVentures

PharmaVentures is a corporate finance and transactions advisory firm that has served hundreds of clients worldwide in relation to their strategic deal making in the pharmaceutical, life science and healthcare sectors. Our key offerings include: Transactions / deal negotiations; Product / technology valuations; Deal term advice; Due diligence & expert reports; Strategy formulation; Alliance management; and Expert opinion for litigation/arbitration cases. PharmaVentures provides the global expertise to ensure our clients generate the highest possible return on investment from all their deal making activities. We have experience of all therapeutic areas and can offer advice on both product and technology commercialisation.

Prosensa

Prosensa is a biopharmaceutical company focused on the discovery, development and commercialization of RNA modulating therapeutics. The company targets genetic disorders with a large unmet medical need, with a primary focus on neuromuscular and neurodegenerative disorders such as duchenne muscular dystrophy (DMD), Myotonic Dystrophy(DM1), Huntington's Disease (HD) and Spinal Muscular Atrophy (SMA). The company has developed a portfolio of clinical and pre-clinical RNA-based drug candidates. Prosensa's current clinical portfolio is focused on the treatment of duchenne muscular dystrophy . Its lead product, PRO051-GSK2402968, aims at restoring dystrophin expression and improving muscle condition and function in a relatively large subpopulation of Duchene patients and has successfully completed a Phase I/II study. Preparations are ongoing to start enrolling patients in a Phase III study in 2010. PRO044, the company's second product candidate, addresses another subpopulation of Duchenepatients and has entered clinical trials in the last quarter of 2009. Prosensa was founded in 2002 and is located in Leiden, The Netherlands. The company works closely together with Leiden University Medical Center (LUMC) and is supported by a consortium of leading biotech investors, including Abingworth, Life Sciences Partners, GIMV, AGF and MedSciences Capital. In October 2009, Prosensa entered into a strategic alliance with GlaxoSmithKline (GSK) to accelerate and broaden the development of its DMD product candidates.