About uniQure BV
uniQure is a world leader in the research and early development of human gene based therapies. Its lead product, Glybera, for the treatment of lipoprotein lipase deficiency was approved by the European Commission in November, 2012. Glybera is the first gene therapy approved in the Western world. uniQure has a product pipeline with several gene therapies: for hemophilia B, acute intermittent porphyria, Parkinson’s disease and Sanfilippo B. uniQure is based in Amsterdam, the Netherlands. The company employs a staff with extensive scientific and industry experience, and is the world leader in manufacturing of AAV-based products on a commercial scale, meeting cGMP requirements, and has extensive experience in the regulatory process for gene therapies.
Gene Therapy Opportunity
For many serious diseases today there is no lasting solution or cure available that addresses the cause of the problem. Existing therapeutic strategies offer symptomatic relief at best.Gene therapy promises long term, potentially life long treatment for seriously debilitating diseases based on a single administration of the therapeutic product. With Glybera approved as the first gene therapy in the Western world, gene-based therapies – much as antibody markets in the past decade - are destined to witness dramatic growth and capture multi-billion dollar markets.
Strategy
uniQure’s strategy is predicated on 3 pillars:
(i) Portfolio strategy
Based on extensive experience and expertise in the development of our lead product Glybera® and our unique manufacturing capabilities we are building a strong gene therapy pipeline for diseases originating in the liver (Hemophilia, Porphyria) and the central nervous system (GDNF applications for Parkinson’s, Huntington’s, MSA, hearing loss, and lysosomal storage diseases such as Sanfilippo B); many academic institutions are accessing our platform and provide development and commercial rights to first in class projects to uniQure.
(ii) Technology platform
Our AAV technology platform has been extensively reviewed and validated by regulatory agencies. It is second to none for high-yield, and high quality production of gene therapy products. We are striving to develop critical technologies to maintain our technology leadership position, including repeat administration, gene expression control, and optimization of our proprietary delivery technologies.
(iii) Commercial and financial strategy
We are seeking partnerships for non-dilutive funding and expect to bring products for orphan indication to market to drive revenues and avoid as much as possible dilutive funding from capital markets.
UniQure is a world leader in the development of human gene based therapies