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Tuesday, 10/19/2021 2:59:16 AM

Tuesday, October 19, 2021 2:59:16 AM

Post# of 255
Michael J. Fox on Raising $1 Billion to Help Find Parkinson’s Cure: ‘I Won’t Stop Until It Happens’


https://www.google.com/amp/s/variety.com/2021/scene/news/michael-j-fox-parkinsons-disease-foundation-1235091451/amp/

Gene therapy although not a cure for Parkinson’s disease may seem like one to someone afflicted with Parkinson’s disease.

AXO-Lenti- PD

Parkinson’s Disease: AXO-Lenti-PD
Dopamine DiagramParkinson’s disease is a progressive neurodegenerative disorder that is caused by the loss of dopamine signaling in the brain and results in the continual decline of motor control and quality of life. While there is currently no cure for the disease, through dopaminergic strategies that temporarily increase dopamine levels in the brain, patients see a short-term improvement in symptoms which wanes with disease progression.

AXO-Lenti-PD is the only investigational gene therapy for Parkinson’s disease that delivers the three key genes (TH, CH1, and AADC) required for endogenous dopamine synthesis in a single lentiviral vector. The goal of this one-time infusion is to restore steady, tonic levels of dopamine, potentially reducing the need for daily L-dopa medication while stabilizing the disease to provide long-lasting benefits. Initial clinical data demonstrate that a single dose of AXO-Lenti-PD “turns back the clock” for patients by improving motor function and activities of daily living. AXO-Lenti-PD has been optimized from ProSavin, an earlier gene therapy for the treatment of Parkinson’s disease.


SUNRISE-PD Design
We are currently evaluating AXO-Lenti-PD in a Phase 2 clinical trial (SUNRISE-PD) in patients with moderate to advanced Parkinson’s disease

https://siogtx.com/programs/

Good luck and GOD bless,

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