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.
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