AAV2-neurturin / Sangamo Therap, Biogen - LARVOL DELTA

Post-Mortem Studies of Neurturin Gene Therapy for Parkinson's Disease: Two Subjects with 10 Years CERE120 Delivery. (PubMed, Mov Disord) - "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."

Gene therapy • Journal • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease • RET

Therapies on the Horizon for Nonexudative AMD (Retina Today) - "Two C3 inhibitors, NGM621 (NGM Biopharmaceuticals) and pegcetacoplan (Apellis Pharmaceuticals), and one C5 inhibitor, avacincaptad pegol (Zimura, Iveric Bio), are showing promise as potential treatments to reduce the progression of geographic atrophy. Elamipretide (Stealth Biotherapeutics) binds to cardiolipin to stabilize mitochondrial structure and reduce the emission of reactive oxygen species, thereby potentially slowing and reversing the effects of oxidative stress. A recombinant AAV2-based investigational gene therapy, GT005 (Gyroscope Therapeutics), is designed to induce the expression of complement factor I."

Online posting

CERE-120 Prevents Irradiation-Induced Hypofunction and Restores Immune Homeostasis in Porcine Salivary Glands. (PubMed, Mol Ther Methods Clin Dev) - "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."

Journal • Preclinical • Fibrosis • Gene Therapies • Head and Neck Cancer • Immunology • Oncology • Oral Cancer • Solid Tumor

Focal and dose-dependent neuroprotection in ALS mice following AAV2-neurturin delivery. (PubMed, Exp Neurol) - "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."

Journal • Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders • Complement-mediated Rare Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

Long-term post-mortem studies following neurturin gene therapy in patients with advanced Parkinson's disease. (PubMed, Brain) - "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."

Clinical • Journal • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

Long-term post-mortem studies following neurturin gene therapy in advanced Parkinson’s disease (Neuroscience 2019) - "We performed postmortem studies on 2 patients with advanced Parkinson’s disease 8 and 10 years following AAV2-neurturin (CERE120) gene therapy...Therefore, if these cases are representative of other patients their respective cohorts, the dopaminergic changes observed were not likely sufficient to provide significant anti-parkinsonian benefits. This study provides the longest term evidence of persistent transgene expression following gene delivery to the CNS and the first human results when targeting the terminal fields in the putamen as well as in the cells of origin."

Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease (clinicaltrials.gov) - P1/2; N=57; Completed; Sponsor: Sangamo Therapeutics; Active, not recruiting ➔ Completed

Clinical • Trial completion