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AAV2-GDNF

uniQure BV

Drug Names(s): GDNF Gene Therapy

Description: uniQure is investigating GDNF (glial-cell line-derived neurotrophic factor) gene therapy.

Using the ClearPoint Neuro Interventional System, GDNF gene packaged in an AAV vector is administered to the brain. The ClearPoint system has been developed to allow real-time observation of brain surgery. The hypothesis is that GDNF’s neuro-regenerative and protective properties may protect and strengthen brain cells that produce dopamine. Dopamine is a chemical that affects brain function. People with Parkinson's disease have problems producing dopamine in a specific area in the brain (substantia nigra) responsible for the control of body movements.

Deal Structure: In June 2012, uniQure announced the signing of a collaborative agreement to develop further a gene therapy incorporating uniQure's GDNF (glial cell derived neutrophic factor) gene for the treatment of Parksinson's disease. Professor Krystof Bankiewicz at the University of California, San Francisco (UCSF) and Professor Howard Federoff of Georgetown University have developed a product approved to start clinical trials in the U.S. using uniQures GDNF gene incorporated into an adeno-associated virus-2 (AAV-2) delivery vector. Under the terms of uniQures agreement with UCSF, uniQure holds the exclusive commercial rights to all UCSF preclinical data and to IND enabling Phase I clinical data provided to UCSF by NINDS. In the event that the Phase I study shows proof of concept, uniQure will use its proprietary manufacturing system for future production of the AAV construct and take responsibility for future development of the gene therapy product. uniQure holds the exclusive license to the...See full deal structure in Biomedtracker

Partners: University of California, San Francisco Amgen, Inc.


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