CERE-110 / Sangamo Therap, Biogen - LARVOL DELTA

Long-term persistence and safety of AAV-NGF gene therapy beyond 10 years in human Alzheimer's disease (Neuroscience 2025) - "While microglia were present in regions of NGF expression, they were not associated with neuronal loss. Moreover, NGF gene expression remained detectable for more than 10 years after treatment.We conclude that AAV2 supports safe and long-lasting gene expression beyond 10 years in the degenerating human brain."

Clinical • Gene therapy • Alzheimer's Disease • CNS Disorders • Movement Disorders • Muscular Atrophy • NGF

Long-term nerve regeneration in diabetic keratopathy mediated by a novel NGF delivery system. (PubMed, Diabetes) - "In a mouse DK model, a single injection of AAV-Ngf into the corneal stroma led to marked corneal nerve regeneration for over 5 months. Together, we provide a novel therapeutic paradigm for long-term effective treatment of DK and this therapeutic approach is superior to current DK therapies."

Journal • Metabolic Disorders • Ophthalmology • NGF

Preventing and treating neurotrophic keratopathy by a single intrastromal injection of AAV-mediated gene therapy. (PubMed, Ocul Surf) - "This proof-of-concept study demonstrates that AAV.rh10-Ngf gene therapy is a promising method for preventing and treating of NK. Our results underline the potential for developing clinical trials to further explore the safety and efficacy of such gene therapy."

Gene therapy • Journal • Diabetes • Dry Eye Disease • Gene Therapies • Herpes Simplex • Keratitis • Metabolic Disorders • Ocular Inflammation • Ophthalmology • NGF • TJP1

AAV2/9-mediated gene transfer into murine lacrimal gland leads to a long-term targeted tear film modification. (PubMed, Mol Ther Methods Clin Dev) - "Remarkably, AAV2/9 induces an important and long-lasting secretion of this growth factor in the tear film. Altogether, our findings provide a new clinically applicable approach to tackle corneal blindness."

Journal • Preclinical • Gene Therapies • Ophthalmology • NGF

Nerve growth factor protects salivary glands from irradiation-induced damage. (PubMed, Life Sci) - "The current study findings indicated that the modulation of the NGF pathway might prevent IR-induced salivary hypo-function."

Journal • Gene Therapies • Head and Neck Cancer • Oncology • Solid Tumor • MAPK8 • NGF

[VIRTUAL] 3D neural circuit visualization by neural tracing and tissue clearing for dementia study (AAIC 2020) - " Cholera toxin subunit B (CTB) conjugated with Alexa Fluor-647 (CTB-647) or adeno-associated virus (AAV) expressing nerve growth factor and green fluorescent protein (AAV-NGF-GFP) was injected into MFB by stereotaxic injection... Taken together, nigrostriatal pathway, connecting substantia nigra and striatum, can be traced by using neural tracers together with tissue clearing. With this protocol, the neural circuits related to the dementia processes in PD can be further studied."

Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Dementia • Gene Therapies • Movement Disorders • Parkinson's Disease • NGF

Randomized, Controlled Study Evaluating CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease (clinicaltrials.gov) - P2; N=49; Completed; Sponsor: Sangamo Therapeutics; Active, not recruiting ➔ Completed

Clinical • Trial completion