Name: CERE-110
Synonyms: Nerve Growth Factor gene therapy
Therapy Type: DNA/RNA-based
Target Type: Other (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Discontinued)
Company: Sangamo BioSciences, Inc.
Approved for:


CERE-110 was a gene-therapy approach for neuroprotective treatment of Alzheimer's disease. It employed injection of an AAV2-based vector for expression of the gene encoding the trophic protein nerve growth factor (NGF) into the nucleus basalis of Meynert, a cholinergic brain area that degenerates in the disease. The approach grew out of research showing that local NGF delivery was able to slow age-related neurodegeneration in mouse models and rhesus monkeys, and it has been in small clinical trials since 2000 (Sep 2002 newsSmith et al., 1999Dec 1999 news). CERE-110 was initially developed by by Ceregene, which was bought by Sangamo BioSciences in 2013. 


A pilot study of a previous form of NGF gene therapy—delivered via genetically engineered autologous fibroblasts—reported safety and hints for a potential long-term benefit for locally produced NGF (Apr 2005 newsSep 2006 conference news).

Between 2004 and 2010, a two-year, open-label Phase 1 trial conducted in San Diego and Chicago investigated the safety, tolerability, and biological activity of increasing doses of CERE-110 delivered directly by bilateral stereotactic surgery into 10 patients with mild to moderate Alzheimer's disease. In this trial, CERE-110 was reported to have been safe and well-tolerated for up to two years of observation. Stable expression and biological activity were reported based on postmortem pathology (Nov 2013 conference newsRafii et al., 2014).

From 2009 to 2015, a multicenter, sham-surgery controlled Phase 2 trial run by the ADCS evaluated CERE-110 in 49 people with mild to moderate AD for its ability to slow decline on the ADAS-cog, NPI, and ADCS-ADL batteries and to measure efficacy on cognition, neuropsychiatric well-being, and activities of daily living. In this study, CERE-110 was again safe and well-tolerated, but ineffective. In April 2015, Sangamo announced it was terminating the program (see company press release).

For all trials of CERE-110, see

Last Updated: 09 Oct 2015


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News Citations

  1. Special Delivery: NGF Trial Puts Growth Factor Where It’s Needed
  2. Madrid: Clinical Trials Update—Where Do Things Stand?
  3. Growth Factor Therapy: Safe in Phase 1, Awaiting Efficacy Data
  4. NGF, Galantamine Rescue Anti-NGF Model of Alzheimer's
  5. NGF Gene Therapy Trial Begins

Paper Citations

  1. . A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer's disease. Alzheimers Dement. 2014 Jan 7; PubMed.
  2. . Age-associated neuronal atrophy occurs in the primate brain and is reversible by growth factor gene therapy. Proc Natl Acad Sci U S A. 1999 Sep 14;96(19):10893-8. PubMed.

External Citations

  1. company press release

Further Reading


  1. . Gene transfer provides a practical means for safe, long-term, targeted delivery of biologically active neurotrophic factor proteins for neurodegenerative diseases. Drug Deliv Transl Res. 2011 Oct;1(5):361-82. PubMed.
  2. . CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease. Curr Opin Mol Ther. 2010 Apr;12(2):240-7. PubMed.
  3. . Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology. 2013 Apr 30;80(18):1698-701. PubMed.
  4. . AAV2-neurturin (CERE-120) for Parkinson's disease. Expert Opin Biol Ther. 2013 Jan;13(1):137-45. PubMed.