Precigen (PGEN) investor relations material

Company overview and technology platforms

• Focuses on innovative cell and gene therapies using differentiated platforms, including AdenoVerse for repeat dosing and UltraCAR-T for rapid, in-patient cell therapy manufacturing.

• AdenoVerse enables treatment of rare diseases and cancers, with unique adenovirus vectors allowing multiple doses without neutralizing antibodies.

• UltraCAR-T platform completed phase Ib and is preparing for pivotal phase II trials.

Lead product and clinical progress

• PAPZIMEOS, based on AdenoVerse, received full FDA approval for recurrent respiratory papillomatosis (RRP), a rare disease previously treated only with repeated surgeries.

• Achieved 51% complete response and 86% overall response in RRP patients, with durable results exceeding 40 months in some cases.

• Orphan drug exclusivity grants seven years of market protection in the U.S.

• Redosing studies and pediatric trials are underway, with strong FDA support.

Commercial launch and market uptake

• Rapid payer coverage achieved, now exceeding 90% of insured lives, including Medicare and Medicaid.

• Broad FDA label allows treatment of all adult RRP patients, leading to strong uptake in both academic and community centers.

• Permanent J-code obtained, streamlining reimbursement and accelerating adoption.

• Real-world compliance mirrors clinical trials, with high adherence to dosing regimen.