Drug intelligence / Profile preview

CERE-110

Development stage
Discontinued
Lead developer
Sangamo Therapeutics
Modality
DNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Stereotactic Injection
01

Overview

CERE-110 is an investigational gene therapy designed for the treatment of Alzheimer's disease. It consists of an adeno-associated virus serotype 2 (AAV2) vector carrying the gene encoding human nerve growth factor (NGF). The therapy is delivered via stereotactic injection into the nucleus basalis of Meynert, a brain region affected by cholinergic neuron degeneration in Alzheimer's disease. By enabling sustained local expression of NGF, CERE-110 aims to provide neuroprotective and function-restoring effects on cholinergic neurons, which are critical for memory and cognitive function. The drug was initially developed by Ceregene and later acquired by Sangamo BioSciences. Clinical trials reached Phase 2 but were discontinued after failing to demonstrate efficacy despite being safe and well-tolerated[1][2][3][5].

Other names
AAV2-NGFAAV-2-NGFAAV 2-NGFNerve Growth Factor gene therapy
02

Targets

NTRK1 (Tropomyosin-related receptor kinase A)

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