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2014

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

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    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.

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