Patient in Gene Therapy Study Dies; Clinical Trial Halted

A patient in a gene therapy clinical trial died after suffering an unspecified serious adverse event (SAE), and the trial has been halted, the sponsor announced.

Targeted Genetics said it decided along with the FDA to shut down the trial of its flagship product after the patient suffered the SAE, but before the death.

The company’s Phase I/II study was designed to assess the safety and efficacy of different doses of tgAAC94, an investigational gene therapy for the treatment of inflammatory arthritis. The investigational product is administered directly into patients’ affected joints using a viral vector that delivers the gene to the cells.

What happened to the deceased patient “has, to our knowledge, never been seen as a consequence of exposure to adeno-associated viral (AAV) vectors or naturally occurring AAV,” Stewart Parker, president and CEO of Targeted Genetics, said. “We continue to work closely and diligently with the FDA and the study’s independent data safety monitoring board to determine the cause of the SAE as quickly as possible.”

The company said that subjects already enrolled in the study will continue to be followed and monitored. Since the trial began in October 2005, 127 adult subjects have received an initial dose of active drug or placebo, 74 out of these patients have received a second dose of active drug and 55 people out of that group have received two doses of the active drug.