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Gene Therapy & GDNF

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Gene therapy firm uniQure signed a collaborative agreement with researchers at the University of California, San Francisco (UCSF) to further develop an early clinical-stage gene therapy for Parkinson disease (PD) based on the glial cell derived neurotrophic factor (GDNF) gene. uniQure was established in February to take over Amsterdam Molecular Therapeutics (AMT) and progress its pipeline of gene therapy products for hemophilia B, acute intermittent porphyria, Parkinson disease, and Sanfilippo B. uniQure holds an exclusive license to the GDNF gene from Amgen.

The gene therapy candidate comprises the GDNF gene carried by an adeno-associated virus-2 (AAV-2) vector, and has been developed by UCSF’s Krystof Bankiewicz, Ph.D., and Howard Federoff, M.D., at Georgetown University. FDA approval has been granted to enable the start of clinical trials, and UCSF has inked a collaboration with National Institute of Neurological Disorders and Stroke (NINDS) researchers to conduct the study in PD patients.

Under terms of the uniQure-UCSF deal the Amsterdam-based firm retains exclusive commercial rights to all UCSF preclinical data and IND-enabling Phase I clinical data provided to UCSF by NINDS. If the Phase I study demonstrates proof-of-concept, then uniQure will carry out manufacture of the AAAV construct and shoulder future development of the gene therapy product. The firm says it will look for a partner for the more advanced development stages.

“GDNF has been shown to be involved in several other CNS disorders,” comments Jorn Aldag, uniQure CEO. “If we reach the proof-of-concept stage in PD, we can potentially expand product development quickly and efficiently into clinical for other indications, such as Huntington disease and multiple system atrophy.”

uniQure is developing a pipeline of AAV-based gene therapy products for hemophilia B, acute intermittent porphyria, PD, and the lysosomal storage disorder Sanfilippo. Acquisition of AMT this year included the latter’s commercial-scale AAV manufacturing capabilities, backed by experience in the regulatory processes for gene therapies.

The firm’s existing pipeline includes Factor IX gene therapy program for hemophilia B program (AMT-060), which is currently undergoing Phase I/II evaluation. A clinical study with a porphobilinogen deaminase (PGBD) gene therapy for treating acute intermittent porphyria is due to start this year. uniQure’s Sanfilippo B gene therapy program (AMT-110) is in development in partnership with the Institut Pasteur in Paris, and an exploratory Phase I/II clinical study is also expected to start during 2012. uniQure has an option to license rights to the Sanfilippo product after successful completion of Phase I/II trial.

AMT had to admit financial defeat and sell itself off as a result of the EU’s Committee for Medicinal Products for Human Use (CHMP) repeated recommendation against approving lead lipoprotein lipase deficiency (LPLD) gene therapy candidate Glybera, despite the more positive view of both the regulator’s Committee for Advanced Therapies and the program’s Scientific Advisory Group. A final decision by the EC on whether to go with the CHMP’s recommendation, or approve the product, has still to be made. 

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