Precigen Inc (Nasdaq: PGEN), biopharmaceutical company that specialises in the development of innovative gene and cell therapies, announced on Monday that it has completed the rolling submission of a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) for PRGN-2012 (zopapogene imadenovec†), an investigational gene therapy for adults with recurrent respiratory papillomatosis (RRP). The submission includes a request for priority review, which, if granted, would shorten the review timeline from 10 to 6 months.

PRGN-2012 is designed to trigger immune responses against cells infected by human papillomavirus (HPV) types 6 and 11, the primary causes of RRP. The therapy has the potential to become the first FDA-approved treatment for this rare and life-threatening condition, which currently relies on repeated surgeries that do not address the underlying viral infection.

The BLA is backed by data from a Phase 1/2 pivotal trial (NCT04724980) involving 38 adult RRP patients. In the study, more than 50% of patients achieved a complete response, defined as no RRP surgeries within 12 months after treatment, while over 85% saw a reduction in surgical interventions compared to the prior year. The therapy was well-tolerated, with no dose-limiting toxicities or treatment-related adverse events exceeding Grade 2.

PRGN-2012 received Breakthrough Therapy Designation and Orphan Drug Designation from the FDA, as well as Orphan Drug Designation from the European Commission. The BLA submission is part of an accelerated approval pathway, reflecting the urgent need for new treatment options for RRP patients.

Precigen's AdenoVerse platform, which underpins PRGN-2012, leverages proprietary adenovectors for efficient gene delivery. The company believes its platform's superior performance characteristics could offer a competitive edge in treating complex diseases.