AAV2-neurturin News - LARVOL Sigma

|||||||||| AAV2-neurturin / Biogen
Journal, Gene therapy: Post-Mortem Studies of Neurturin Gene Therapy for Parkinson's Disease: Two Subjects with 10?Years CERE120 Delivery. (Pubmed Central) - Aug 6, 2023
This study provides evidence of long-term persistent transgene expression and bioactivity following gene delivery to the nigrostriatal system. Therefore, future efforts using gene therapy for neurodegenerative diseases should consider means to enhance remaining dopamine neuron function and stop pathological propagation.

|||||||||| AAV2-neurturin / Biogen
Preclinical, Journal: CERE-120 Prevents Irradiation-Induced Hypofunction and Restores Immune Homeostasis in Porcine Salivary Glands. (Pubmed Central) - Sep 23, 2020
CERE-120 prevented IR-induced hypofunction and restored immune homeostasis, and there was a coordinated contralateral gland response to either damage or treatment. CERE-120 gene therapy is a potential treatment for head and neck cancer patients to influence communication among neuronal, immune, and epithelial cells to prevent IR-induced salivary hypofunction and restore immune homeostasis.

|||||||||| AAV2-neurturin / Biogen
Preclinical, Journal: Focal and dose-dependent neuroprotection in ALS mice following AAV2-neurturin delivery. (Pubmed Central) - Jul 22, 2020
Overall, there were no increases in morbidity, changes in serum chemistries or blood counts and no cases of drug-related mortality. Because there is a broad clinical experience for this compound, these data provide evidence to support further investigation of AAV2-NRTN as a potential ALS therapeutic.

|||||||||| AAV2-neurturin / Biogen
Clinical, Journal: Long-term post-mortem studies following neurturin gene therapy in patients with advanced Parkinson's disease. (Pubmed Central) - Jul 7, 2020
We performed post-mortem studies on two patients with advanced Parkinson's disease 8 and10 years following AAV2-neurturin (CERE120) gene therapy, the longest post-mortem trophic factor gene therapy cases reported to date...These changes were not associated with antiparkinsonian benefits likely due to the limited neurturin expression. This study provides the longest term evidence of persistent transgene expression following gene delivery to the CNS and the first human results when targeting both the terminal fields in the putamen as well as the originating nigral neurons.

||||||||||  AAV2-neurturin / Biogen
Trial completion:  Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease (clinicaltrials.gov) -  Jul 6, 2019
P1/2, N=57, Completed,  Sponsor: Sangamo Therapeutics
This study provides the longest term evidence of persistent transgene expression following gene delivery to the CNS and the first human results when targeting both the terminal fields in the putamen as well as the originating nigral neurons. Active, not recruiting --> Completed

||||||||||  AAV2-neurturin / Biogen
Trial primary completion date:  Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease (clinicaltrials.gov) -  Oct 31, 2016
P1/2, N=60, Active, not recruiting,  Sponsor: Ceregene
Active, not recruiting --> Completed Trial primary completion date: Mar 2013 --> Jan 2018

||||||||||  AAV2-neurturin / Biogen
Enrollment closed:  Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease (clinicaltrials.gov) -  Dec 4, 2011
P1/2, N=60, Active, not recruiting,  Sponsor: Ceregene
Trial primary completion date: Mar 2013 --> Jan 2018 Recruiting --> Active, not recruiting



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