Research & Development
uniQure research and development is focused on rational and efficient progression of projects from non-clinical proof-of-concept to clinical phase I - first in man studies - and potentially clinical phase II/III studies, for gene therapies targeting diseases with an unmet medical need. The main focus is in developing such therapies in the areas of metabolic disorders and disorders of the central and peripheral nervous system.
uniQure is experienced in gene therapy development targeting muscle, liver, and the CNS, and will exceptionally in collaborations also target other organs or systems. Through Glybera for Lipoprotein Lipase Deficiency (LPLD) the company has gained extensive expertise in muscle-targeted gene therapy using recombinant adeno-associated virus (AAV) as the delivery vehicle. In the course of the marketing authorization process for Glybera, uniQure’s technology platform was validated and accepted without any material safety concerns. The company also passed all the standard GMP and GCP audits.
Presently uniQure’s clinical development focus is on four projects; Hemophilia, Parkinson’s disease, Acute intermittent porphyria and Sanfilippo B. In research we are exploring additional therapeutic options especially for the use of GDNF (glial-derived neurotropic factor) in the area of neurodegenerative diseases Other activities in research are focused on future key areas in gene therapy: vector re-administration, controlled and regulated expression as well as targeted vector development.
We invite possible partners to contact us pro-actively, to discuss any projects they deem viable (firstname.lastname@example.org).