Pfizer Presents Initial Clinical Data on Phase 1b Gene Therapy Study

Pfizer (NYSE:PFE) has announced an update on its Phase 1b clinical data for an investigational gene therapy to treat Duchenne muscular dystrophy (DMD).

As quoted in the press release:

The primary endpoint of the ongoing Phase 1b study is to assess the safety and tolerability of this investigational gene therapy. Secondary endpoints of the clinical study include measurement of expression of mini-dystrophin distribution within muscle fibers by immunofluorescence and concentration by liquid chromatography mass spectrometry (LCMS). Pfizer aims to enroll approximately 12 boys with DMD who are ambulatory and aged 5 to 12. To date, 6 study participants ranging in age from 6 to 12 years have received the one-time intravenous dose of PF-06939926 at either 1E14 vector genomes/kilogram (vg/kg) or 3E14 vg/kg, as quantified using an inverted terminal repeat-based quantitative polymerase chain reaction (qPCR) drug product titer assay.

“Gene therapy for single-gene disorders is at a formative stage in its evolution, and the initial data we’ve seen in our study for Duchenne muscular dystrophy may exemplify the potential for this modality to change patients’ lives,” said Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Disease Research Unit. “We are looking forward to building on these initial data and advancing the development of this therapeutic modality.”

Click here to read the full press release.