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Gene therapy developer gets chance at a comeback with Pfizer deal

Voyager’s first generation of gene therapies for ALS, Parkinson’s and Huntington’s disease, all now discontinued, relied on a direct infusion into brain tissue, an invasive procedure. The company claims its new technology, dubbed TRACER, is more cell- and tissue-specific and can penetrate the blood-brain barrier, allowing it to be infused through a less invasive intravenous procedure.

That technology is what interests Pfizer. The big drugmaker has clinical-stage gene therapy programs in Duchenne muscular dystrophy and hemophilia. However, some mutation-driven neurologic diseases have been more challenging to target because the frequently used adeno-associated virus, or AAV, vectors aren’t as tissue-specific as researchers would like.

“We are impressed with Voyager’s results to date and are enthusiastic about the potential to utilize these novel capsids to help accelerate the development of new therapeutic options for patients living with certain neurologic and cardiovascular diseases,” Seng Cheng, chief scientific officer of Pfizer’s rare disease research unit, said in a statement.

For Voyager, TRACER and the Pfizer deal are a bit of a lifeline. The company’s leadership decided to replace on an interim basis the former CEO with board chair Michael Higgins and the former research chief with director Glenn Pierce. The executive reshuffle preceded a strategic shift to increase investment in the TRACER technology and focus on second-generation TRACER-based AAV gene therapies in neurologic and muscular disease.

That decision added years to Voyager’s clinical development timelines, prompting Cantor Fitzgerald analyst Charles Duncan to cut his rating on the company’s stock on Aug. 12. With $149 million in cash and quarterly expenditures of $30 million, Duncan then estimated that Voyager had enough resources to fund itself through early 2023 and the pre-clinical studies necessary to seek regulatory permission to begin human testing.

The Pfizer deal announced Wednesday adds to that cash balance and provides a sign to investors that the new gene therapy technology has promise.