Cedars-Sinai Lands $2.5M Grant for a Gene-Therapy-Based Preclinical Trial

A multi-party ALS-research collaboration between Cedars-Sinai Regenerative Medicine Institute, the University of Wisconsin, Madison and Netherlands-based biotechnology company uniQure is now backed by a $2.5M grant from the Department of Defense (DOD). The funding will support preclinical studies that could be the basis for a gene-therapy based clinical trial in ALS. Previous work from Masatoshi Suzuki’s laboratory at University of Wisconsin, Madison (see story from June 2013) has shown that intramuscular injection of stem cells engineered to produced glial cell-line derived neurotrophic factor (GDNF) into a rat model of ALS prolongs survival and attenuates loss of motor function. These studies, to be conducted in Clive Svensen’s laboratory at Cedars-Sinai and Masatoshi Suzuki’s laboratory will use AAV5 viral vectors to deliver GDNF into muscles of the leg and diaphragm in order to target two major muscle groups affected in ALS. The DOD funding will support the preclinical studies from animal studies through the filing of an Investigational New Drug application with the Food and Drug Administration.

A multi-party ALS-research collaboration between Cedars-Sinai Regenerative Medicine Institute, the University of Wisconsin, Madison and Netherlands-based biotechnology company uniQure is now backed by a $2.5M grant from the Department of Defense (DOD). The funding will support preclinical studies that could be the basis for a gene-therapy based clinical trial in ALS. Previous work from Masatoshi Suzuki’s laboratory at University of Wisconsin, Madison (see story from June 2013) has shown that intramuscular injection of stem cells engineered to produced glial cell-line derived neurotrophic factor (GDNF) into a rat model of ALS prolongs survival and attenuates loss of motor function. These studies, to be conducted in Clive Svensen’s laboratory at Cedars-Sinai and Masatoshi Suzuki’s laboratory will use AAV5 viral vectors to deliver GDNF into muscles of the leg and diaphragm in order to target two major muscle groups affected in ALS. The DOD funding will support the preclinical studies from animal studies through the filing of an Investigational New Drug application with the Food and Drug Administration.

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