Phase 1 Clinical Trial of CERE-120 Gene Therapy begins

["ljherman1@xxxxxxxx" <ljherman1@xxxxxxxx>]

FROM PR Newswire:

Ceregene Reports Initiation of Phase 1 Clinical Trial of Novel Gene Therapy for 
Patients With Parkinson's Disease

SAN DIEGO, Sept. 21 /PRNewswire-FirstCall/ -- Ceregene announced today that 
patient treatment is underway in its Phase I study of CERE-120 to treat 
Parkinson's disease. The study is being conducted at the University of 
California, San Francisco Medical Center and Rush University Medical Center in 
Chicago. CERE-120 is a novel gene therapy product that delivers the neurturin 
(NTN) gene via an adeno-associated virus (AAV) type2 vector delivery system. 
The naturally occurring NTN gene encodes the NTN protein that maintains 
survival of dopamine-producing nerve cells that are required for normal bodily 
movement and are the nerves that degenerate in Parkinson's disease patients. 
Neurturin is a member of the same protein family as GDNF and they have similar 
pharmacological properties. GDNF has previously been tested in Parkinson's 
disease patients. Ceregene owns exclusive technology and product rights to 
CERE-120.

"CERE-120 has the potential to significantly slow or even halt the progression 
of symptoms of this devastating disease," said Jeffrey M. Ostrove, Ph.D., 
president and chief executive officer of Ceregene. "Numerous studies in animal 
models, including the most widely accepted animal models of Parkinson's 
disease, consistently demonstrated that this NTN gene therapy product may be 
able to improve symptoms as well as retard the progression of the disease. 
These studies also demonstrate that CERE-120 is also safe and well tolerated in 
animals at doses hundreds of times higher than the equivalent doses being 
tested in humans. We are pleased that human clinical studies of our CERE-120 
product are now under way."

This study is being led by William J. Marks, Jr., M.D., a neurologist at the 
University of California, San Francisco (UCSF). Dr. Philip Starr, a 
neurosurgeon at UCSF, performs the surgery. UCSF investigators also include 
neurologist Jill Ostrem, M.D. and neurosurgeon Paul Larson, M.D. In addition, 
neurologist Leo Verhagen, M.D, Ph.D. and neurosurgeon Roy Bakay, M.D. are 
conducting the study at Rush University Medical Center in Chicago.

This Phase I study involves in vivo (inside the body) gene therapy delivery of 
NTN. Ceregene's in vivo gene therapies utilize a non-replicating viral vector 
to deliver therapeutic genes to the nervous system, where they express proteins 
with the ability to prevent neurodegeneration and restore normal nerve 
function. The adeno-associated viral (AAV) vector, which has never been 
causally associated with disease in humans, is an important gene delivery tool 
that has demonstrated ability to efficiently and stably express genetic 
information in non-dividing target cells, such as the neurons degenerating in 
Parkinson's disease, and to potentially achieve long-term, therapeutic benefit. 
The goal of this study is to determine the safety and efficacy of this 
treatment. Efficacy will be measured by standardized tests for Parkinson's 
patients, as well as brain imaging studies.

"Parkinson's disease represents a major unmet medical need, in that current 
therapies are only useful for a limited time, for eventually their side effects 
become debilitating," stated Raymond T. Bartus, Ph.D., Ceregene's senior vice 
president and chief operating officer. "Moreover, no currently available 
treatment is able to slow the progression of the disease, as we hope will be 
possible with CERE-120, based on our animal studies." Dr. Bartus further 
stated, "We are pleased that we have been able to develop two different 
clinical products and have now initiated clinical programs for two serious 
neurodegenerative diseases within a one-year timeframe." Ceregene also has an 
active clinical program in Alzheimer's disease (CERE-110 or AAV-NGF) and has 
recently completed enrollment in a Phase I clinical study at Rush University 
Medical Center in Chicago.

About Ceregene Ceregene, Inc. is a San Diego-based biotechnology company 
focused on the development of gene therapies for neurodegenerative disorders. 
Ceregene is in the clinic with CERE-110, an AAV2 based vector expressing nerve 
growth factor that is being tested as a treatment for Alzheimer's disease, and 
CERE-120 for Parkinson's disease. CERE-130 is in late preclinical development 
for ALS. Ceregene was launched in January 2001 and is a former subsidiary of 
Cell Genesys, Inc. (Nasdaq: CEGE), which is headquartered in South San 
Francisco, CA. Ceregene closed a $32M Series B financing in August 2004 that 
was co-led by Alta Partners and MPM Capital.

Contact: Jeffrey M. Ostrove, Ph.D. President and
Chief Executive Officer
858-458-8808


SOURCE Ceregene, Inc.
Web Site: http://www.ceregene.com

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