Genzyme enters into partnership with Ceregene for CERE-120 gene therap y for PD

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Genzyme bets on gene therapy as others steer clearReuters
Thu Jun 21, 2007 2:24PM EDT
By Toni Clarke 
BOSTON, June 21 (Reuters) - Genzyme Corp. is expanding its research into gene 
therapy, a controversial and cutting-edge approach to treating disease which 
most companies avoid. 
The company, which built itself into one of the world's biggest biotechnology 
companies by developing drugs for rare genetic disorders, said on Thursday it 
has entered a partnership with Ceregene Inc. to develop a treatment for 
Parkinson's disease. 
Gene therapy is a controversial method of injecting genes into cells to make 
proteins that can fight disease, typically using a genetically engineered virus 
to carry the gene to the cell. 
So far, no gene therapy product has been approved, and research has shriveled 
following the death in 1999 of Jesse Gelsinger, an 18-year-old participant in a 
gene therapy clinical trial. 
"Gene therapy makes everyone nervous and there are only a limited number of 
players," said Dr. Harry Tracy, publisher of the monthly journal 
NeuroInvestment. "This is a daring area of research, and Ceregene's program is 
one of the most sophisticated out there." 
Genzyme began work on gene therapy in the early 1990s as a potential treatment 
for cystic fibrosis. That program didn't work, but the company continued its 
research and is now in mid-stage clinical trials with a treatment which 
stimulates blood vessel growth and could be valuable for patients with 
conditions characterized by poor circulation, such as peripheral artery 
disease. 
Ceregene's program, CERE-120, is composed of an adeno-associated virus vector 
carrying the gene for neurturin, a protein known to protect or repair nerve 
cells which secrete a brain chemical known as dopamine that is involved in 
movement. Parkinson's disease is caused by a reduction in dopamine-containing 
nerve cells. 
Gene therapy, in theory, represents an attractive technology for treating 
neurological disorders because the treatment circumvents the blood-brain 
barrier, a cell structure that protects the brain from foreign substances in 
the blood -- including large molecules such as therapeutic proteins. 
"Parkinson's is very attractive because you can deliver the gene to a localized 
area, the cells will make the protein and hopefully do the job," said David 
Meeker, head of Genzyme's rare genetic diseases unit. 
Meeker said its Parkinson's research is the cornerstone for what the company 
expects will be a growing central nervous system disorders program, including 
work on treatments for ALS, also known as Lou Gehrig's disease, and 
Huntington's disease, a condition in which the degeneration of brain cells 
causes uncontrolled movement and loss of intellectual function. 
"This is an area of high interest for us," Meeker said. 
Cambridge, Massachusetts-based Genzyme said it will pay Ceregene $25 million up 
front, make as much as $125 million in payments as the companies reach 
development milestones, and provide 50 percent reimbursement for late-stage 
development costs. 
Genzyme will gain marketing rights in all markets outside the United States and 
Canada. 
Ceregene, which was launched in January 2001 and is a former subsidiary of Cell 
Genesys Inc. , is currently conducting a Phase II, or mid-stage, clinical trial 
which is enrolling 51 patients. 
In an early-stage trial, CERE-120 showed a 36 percent reduction in Parkinson's 
symptoms at 12 months following administration. 
"This is early data which is very encouraging," Meeker said, "but it is too 
early to draw meaningful conclusions about the relative efficacy to other 
treatments." 
((Reporting by Toni Clarke, editing by Gerald E. McCormick; Reuters Messaging, 
toni.clarke.reuters.com@xxxxxxxxxxx, 617-367-4165)) Keywords: 
GENZYME/GENETHERAPY 
For more on this therapy, see PD Pipeline Database
www.pdpipeline.org__._,_.___  



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