Therapeutic Candidate or Device

Autologous stem cell-derived dopaminergic neuron replacement therapy.

Indication

Parkinson’s disease

Therapeutic Mechanism

The investigational cell therapy product is being studied as an autologous neuron replacement for Parkinson’s disease.

Unmet Medical Need

PD is a neurodegenerative disorder, affecting 1+ million people in the US. PD causes loss of dopamine (DA) neurons in the brain, leading to loss of motor and neurological function, and severely affecting quality of life (QoL) and life span. Current therapies have side effects and are not curative.

Project Objective

Complete First in Human Phase 1/2a clinical trial.

Major Proposed Activities

• Manufacture product to supply the proposed trial.
• Conduct trial to evaluate the safety, tolerability, and preliminary efficacy of autologous neuron replacement in patients with moderate to severe PD.
• Patient follow-up and data collection.

In California, ~50,000 people are living with PD. As a potentially effective and durable alternative with low side effects compared to current therapies, an autologous cell replacement therapy has the potential to transform the lives of Californians that have a reduced QoL due to the inability to conduct activities of daily living. This clinical trial will contribute to the continued efforts of the state to advance science to discover cell therapies that can address diseases ailing its citizens.