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CERE-120: AAV2-NTN · 1 trial · 1 indication
| Name | Type | Description |
|---|---|---|
| CERE-120: AAV2-NTN | GENETIC | - |
Inclusion Criteria: * Diagnosis of bilateral idiopathic Parkinson's Disease of at least 5 years duration since diagnosis with motor fluctuations, despite adequate oral antiparkinsonian therapy. * Diagnosis of moderate to severe Parkinson's Disease based on clinical rating scales. * Males or nonpreg...
CERE-120, also known as AAV2-NTN, is an investigational gene therapy being studied for the treatment of Parkinson's Disease. It is designed to deliver a therapeutic gene to the brain to potentially slow or halt the progression of the disease. The drug is currently in clinical development and has not been approved by regulatory authorities.
CERE-120 is a gene therapy that uses an adeno-associated virus (AAV2) vector to deliver the gene for neurturin (NTN), a neurotrophic factor. Neurturin is intended to support the survival and function of dopamine-producing neurons in the brain, which are progressively lost in Parkinson's Disease. This mechanism aims to address the underlying neurodegeneration.
CERE-120 is being developed by Sangamo Therapeutics, Inc., a biotechnology company traded on the NASDAQ under the ticker symbol SGMO. The company is focused on gene therapy and genome editing. Sangamo is conducting clinical trials to evaluate the safety and potential efficacy of CERE-120 in patients with Parkinson's Disease.
CERE-120 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or any other regulatory agency. The Phase 1 trial has been completed, and the drug is no longer in active clinical trials. Further development status beyond Phase 1 has not been disclosed.
CERE-120 has been studied in one completed Phase 1 clinical trial with the identifier NCT00252850. The trial, titled 'Safety of CERE-120 (AAV2-NTN) in Subjects With Idiopathic Parkinson's Disease,' enrolled 12 participants in the United States. The study was uncontrolled and not blinded, focusing on safety in patients aged 35 years and older.
Yes, CERE-120 is also known as AAV2-NTN. The name AAV2-NTN reflects the drug's composition: an adeno-associated virus serotype 2 (AAV2) vector carrying the gene for neurturin (NTN). Both names refer to the same investigational gene therapy being developed by Sangamo Therapeutics for Parkinson's Disease.