Investigators at the University of Pittsburgh School of Medicine are engaged in administering a new gene therapy from Voyager Therapeutics ($VYGR) in the first small human study aimed at providing some solid proof-of-concept data for Parkinson's disease.
Voyager's lead program, which helped make the case for its recent IPO, is recruiting 20 patients split between Pittsburgh and a site in San Francisco and inserting a gene that makes the enzyme aromatic L-amino acid decarboxylase--which is required to make a supply of dopamine--into their brains. Holes are bored into the patients' skulls and the gene is delivered using an AAV-2 vehicle now commonly used in the field. A catheter is used to concentrate delivery on a region of the brain called the putamen and the holes are later patched.
This particular enzyme is needed to turn levo-dopa into dopamine, a treatment that loses effect as patients' neurons degenerate.
"By inserting the gene for this enzyme into cells in a specific part of the brain, we hope to make levo-dopa treatment more effective for a longer period of time," said Dr. Mark Richardson, an assistant professor of neurological surgery at the Pitt School of Medicine.
Investigators will test various doses of the gene therapy and then follow the patients over the course of three years. Voyager told investors back when they filed for an IPO that the company hoped to provide some clear POC data for this program in the second half of this year.
Voyager, a 2014 Fierce 15 company, is part of a new group of gene therapy pioneers that hopes to ride a wave of enthusiasm for gene therapy to approvals for major therapeutic advances for a variety of diseases. The field has had some sharp ups and downs over the decades, but the recent Phase III success at Spark Therapeutics ($ONCE), a spinoff of the Children's Hospital of Philadelphia, has kept hopes high for this sector.
That enthusiasm, though, hasn't protected Voyager from the sharp downturn on Wall Street for all things biotech. The Cambridge, MA, company's stock is trading at about a third of its former high.
An estimated 7 million to 10 million people around the world suffer from Parkinson's.
- here's the release
Special Report: FierceBiotech's 2014 Fierce 15 - Voyager Therapeutics