Analyst David Nierengarten explained that Voyager Therapeutics has selected various Central Nervous System (CNS) diseases, “with validated, specific gene targets that are amenable to AAV gene therapy.”
The CNS is an “ideal site for gene therapy due to its “immune privilege” status and terminally-differentiated cells that allow long-term transgene expression,” Nierengarten went on to say.
VY-AADC01 And Parkinson's
The company’s lead program, VY-AADC01, is currently being development for the treatment of advanced Parkinson’s disease. The drug aims to restore the expression of an enzyme, AADC, which is responsible for converting L-Dopa to Dopamine, but is progressively lost due to Parkinson’s.
Therefore, by restoring AADC levels, the drug is expected to help with significant improvement in motor skills, as well as L-Dopa responsiveness.
VY-SOD101 And ALS
The second pipeline drug is VY-SOD101, which is gene therapy for the treatment of ALS or monogenic amyotrophic lateral sclerosis. “VYGR’s other preclinical programs target Friedreich’s ataxia, Huntington’s disease and spinal muscular atrophy,” Nierengarten added.
According to the Wedbush report, Voyager Therapeutics’ pipeline products are backed by robust preclinical data.
Partnerships Boost Benefits
In addition, the company’s strategic partnership with Genzyme not only “validates VYGR’s product development platform,” but also “allows leverage of the combined expertise, and provides substantial financial support for VYGR to develop its pipeline,” the report said.
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