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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Voyager’s gene therapy shows positive response in Parkinson’s patients

Phase 1 interim data presented at the 2019 American Academy of Neurology meeting in Philadelphia has shown promise in Voyager Therapeutics’ gene therapy for Parkinson’s disease.

12-month data showed that a single infusion of VY-AADC, an investigational gene therapy Voyager is co-developing with Neurocrine Biosciences, improved motor function and reduced the need for antiparkinsonian medications in advanced Parkinson’s disease patients.

The ongoing Phase 1 trial (PD-1102) is designed to evaluate the safety and efficacy of a single dose of VY-AADC injected directly into the striatum of patients using a surgical approach where the gene therapy is delivered through the back of the patient’s head.

VY-AADC is a gene therapy candidate designed to deliver the AADC gene directly into neurons of the putamen where dopamine receptors are located, bypassing the substantia nigra neurons and enabling the neurons of the putamen to produce the AADC enzyme to convert levodopa into dopamine. Voyager reasons that VY-AADC has the potential to durably enhance the conversion of levodopa to dopamine and provide clinically meaningful improvements by restoring motor function in patients and improving symptoms following a single administration.

Administration of a single dose of VY-AADC was able to cover atleast 54% of the putamen brain area in all the eight patients who were enrolled in the trial. 12-momth data showed that the treatment increased AADC enzyme activity in the putamen area by 85%, implying the ability of neurons in this region to convert levodopa to dopamine.

Motor function along with the patients’ quality of life were improved at 12 months and the treatment also lowered patients’ use of antiparkinsonian medications by 28% six and 12 months after infusion. The treatment was well-tolerated with no serious adverse events reported.

Dr Eiry W. Roberts, Neurocrine’s CMO commented: “The results from this Phase I trial in patients with Parkinson’s disease provide further evidence that VY-AADC administration can allow neurons in the brain to convert levodopa to dopamine and improve motor function. These results confirm previous data from a separate, ongoing Phase I study demonstrating that increased coverage of the putamen with VY-AADC leads to an increase in AADC enzyme activity and improvements in motor function and quality of life in patients with Parkinson’s disease — with less need for oral levodopa medication”. Voyager Therapeutics entered into a license agreement with Neurocrine earlier this year to co-develop VY-AADC for Parkinson’s and three other programs for other CNS disease indications.

Source: AANAM — Investigational VY-AADC01 Gene Therapy Provides Benefits for Parkinson’s Patients, Phase 1 Data Show; Press Release

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