Precigen, Inc. announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for the first-in-class investigational PRGN-2012 AdenoVerseâ„¢ immunotherapy for the treatment of recurrent respiratory papillomatosis (RRP).FDA's Breakthrough Therapy Designation expedites the development and review of medicines which are intended to treat serious or life-threatening diseases, and in which preliminary clinical evidence demonstrates substantial improvement on clinically significant endpoints over available therapies. PRGN-2012 incorporates optimized antigen design that uses gorilla adenovector technology, part of Precigen's proprietary AdenoVerse platform, to elicit immune responses directed against cells infected with human papillomavirus type 6 (HPV 6) or HPV type 11 (HPV 11). Gorilla adenovectors have numerous advantages, including the ability for repeat administration, the inability to replicate in vivo, and the ability to deliver a large genetic payload.

PRGN-2012 has previously been granted Orphan Drug Designation in patients with RRP by the FDA. The Breakthrough Therapy Designation was informed by the clinical evidence for PRGN-2012 generated in the Phase 1 study (NCT04724980), which was presented at Precigen's most recent R&D day, and showed strong response at the recommended phase 2 dose (RP2D) in patients who had an average of 5.8 RRP surgeries (range 3 -- 10) in the year prior to PRGN-2012 treatment. Patient follow up is ongoing as are discussions with the FDA regarding potential rapid development paths to enable a future submission of a Biologics License Application (BLA).

Precigen is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases.