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Mar 21, 2011
 |  BioCentury  |  Product Development

Location, location

Neurologix gene therapy data for Parkinson's; aims to replace brain stimulation

Following the publication of full data from the first successful Phase II trial of a gene therapy candidate for Parkinson's disease, Neurologix Inc. believes it will be able to replace deep brain stimulation as the standard of care for advanced PD. The company says the reason NLX-P101 succeeded is the location within the brain where the therapy is infused.

In its initial readout last June, Neurologix reported NLX-P101 (AAV-GAD) met the primary endpoint of significantly improving off-medication motor scores from baseline to six months vs. a sham infusion of saline. Updated results published in The Lancet last week revealed the mean improvement was 23.1% (8 points) for NLX-P101 (AAV-GAD) vs. 12.7% (4.7 points) for sham control (p=0.04).

The endpoint was measured using the Unified Parkinson's Disease Rating Scale (UPDRS) Part III, which is a composite of multiple symptoms of the disease, including tremor, stiffness, freezing and difficulty walking.

NLX-P101 also showed an improvement over sham on many of the trial's secondary endpoints, although no difference was seen on a few, including activities of daily living and dyskinesia ratings (see B17).

NLX-P101 is a glutamic acid decarboxylase (GAD) 65 and 67 gene therapy delivered using an adeno-associated viral (AAV) vector. The GAD gene is responsible for synthesizing GABA. Decreased GABA in the subthalamic nucleus is one reason for uncontrolled neuron firing in PD, which leads to motor dysfunction.

NLX-P101 is administered to the patient only once, through catheters placed on each side...

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