Ceregene

About Ceregene

Ceregene, Inc. was a clinical-stage biotechnology firm founded in 2001 by Stephen Sherwin, focusing on gene therapies for neurodegenerative diseases. Headquartered in San Diego, the company directed its efforts towards developing treatments for conditions with no effective therapies, such as Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis (ALS), and various ocular diseases. The company's business model was centered on the research, development, and clinical testing of its product pipeline, with a strategy to form partnerships for commercialization, such as its ex-North American licensing deal with Genzyme for CERE-120. Ceregene raised approximately $71.5 million in funding over several rounds from investors including Alta Partners, MPM Capital, and Hamilton BioVentures.

The company’s core technology involved using a modified, harmless adeno-associated virus (AAV) as a vector to deliver genes encoding neurotrophic factors directly to affected cells in the brain or eyes. This approach was designed to enable the patient's own cells to produce therapeutic proteins on a sustained basis after a single administration, aiming to slow or even reverse neuronal degeneration. Ceregene's product pipeline included several candidates at various stages of development. Its lead program was CERE-120 (AAV-Neurturin), which entered Phase 2 trials for Parkinson's disease and was designed to protect and restore dopamine-secreting neurons. Another significant product was CERE-110 (AAV-Nerve Growth Factor), which progressed to Phase 2 trials for Alzheimer's disease, targeting the preservation of cholinergic neurons to improve cognitive function. Other preclinical programs included CERE-130 for ALS and CERE-140 for ocular disorders.

Despite showing a strong safety profile across multiple clinical trials involving over 100 patients, Ceregene faced a major setback when its lead drug, CERE-120, failed to meet its primary efficacy endpoint in a Phase 2b trial for Parkinson's disease in April 2013. Following this result, the company began exploring strategic alternatives. In August 2013, Sangamo BioSciences (now Sangamo Therapeutics) announced its acquisition of Ceregene in an all-stock deal. The acquisition gave Sangamo control over all of Ceregene's assets, including its AAV platform, the ongoing Phase 2 trial for CERE-110 in Alzheimer's, a portfolio of over 120 patents, and a substantial database of manufacturing know-how and clinical safety data. The deal was finalized in September 2013, marking the end of Ceregene's operations as an independent entity.