(August 2013 – Ceregene was acquired by Sangamo Biosciences)

Ceregene, Inc. is focused on the treatment of major neurodegen- erative disorders using the delivery of nervous system growth factors. Neurodegenerative diseases such as Parkinson’s disease, Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), and Huntington’s disease are the result of specific cell loss in the central nervous system. Substantial scientific evidence from numerous international laboratories, as well as in their labs, has shown that neurotroph- ic growth factors (NGFs) can have re- markable effects in numerous models of neurodegenerative diseases.

However, the inability to accurately and effectively target NGF delivery has limited their utility in the clinic. Ceregene’s proprietary adeno-associ- ated virus (AAV)-based gene delivery approach solves this problem, provid- ing targeted delivery of the NGFs in a sustained fashion for the lifetime of the patient following a single dosing procedure. The proprietary use of neurotrophic factor genes, delivered by gene therapy, to treat neurode- generative diseases provides the company with a broad platform of therapeutic opportunities.

 
How Ceregene’s Gene Delivery Technology Works
Gene transfer offers an effective means of treating diseased and dying neurons with neurotrophic factors in a targeted and prolonged fashion. Ceregene has identified several neurotrophic factors to treat damaged nerve cells in several different diseases. For each disease, the gene encoding the appropriate neurotrophic factor known to provide nourishment for the key neurons that are dying is delivered to those cells in a single dosing procedure. This is accomplished through a gene therapy vector Ceregene created that deliv- ers the therapeutic gene to the target cells. Once the customized vector delivers the therapeutic gene into the cell, these cells are able to continu- ously produce the neurotrophic factor. Elevated levels of the neurotrophic factor enhance the vitality and func- tion of the degenerating neurons while also helping them withstand further damage from the disease.

To carry the neurotrophic factor genes into the targeted neurons in a safe and effective fashion, the company uses a harmless version of a virus common among human exposure. This virus, adeno-associated virus (AAV), appears to be safe since it is not known to be associated with any human disease. In addition, Ceregene modified AAV to block the virus from replicating or reproducing itself, to as- sure that the genes delivered remain exclusively in the targeted tissue.

Ceregene’s AAV-based gene delivery technol- ogy provides a broad platform to develop pioneering treat- ments for many different serious diseases, includ- ing Alzheimer’s disease, ALS, Parkinson’s dis- ease, Hunting- ton’s disease, and several ocular diseases. The company is cur- rently advancing this technology in its human clinical trials for Parkinson’s disease and Alzheimer’s disease.

 
Clinical Programs
The company’s clinical programs in- clude CERE-110, an AAV2 based vec- tor expressing NGF for the treatment of Alzheimer’s disease, which is currently in multi-center, controlled Phase II testing in collaboration with the Alzheimer’s Disease Cooperative Study (ADCS) and Dr. Paul Aisen at UC San Diego.

CERE-120 (AAV2-Neurturin), which completed a Phase II clinical trial in Parkinson’s disease, is currently be- ing tested in a new Phase I/II study. CERE-135 and CERE-140 are in pre- clinical development for amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and ocular diseases, respec-tively.

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Ceregene, Inc.
9381 Judicial Drive Suite 130, San Diego, CA 92121
Tel: (858) 458-8800
Fax: (858) 458-8801
Founded: 2001
Employees: 20

Jeffrey M. Ostrove, PhD – President & CEO
Raymond T. Bartus, PhD – Exec. Vice President & CSO

Financing/Financial Milestones:
Series A: $10.5 million
Series B: $32 million (2004)
Series C: $28.1 million (2007)
Series D: $11.5 million (2010)

Technology Innovator:

Tuszynski

Mark H. Tuszynski, MD, PhD
Professor, Neurosciences
Director, Center for Neural Repair
School of Medicine