BOSTON, MA., July 1, 2025 — GentiBio, Inc., a biotechnology company pioneering engineered regulatory T cell (EngTreg) therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead investigational product, GNTI-122, for the treatment of recently diagnosed Type 1 Diabetes (T1D). GNTI-122 is a novel, autologous engineered Treg immunotherapy designed to restore immune tolerance and preserve pancreatic islet function in patients with recently diagnosed T1D.
This recognition from the FDA is an important milestone for GentiBio and affirms its mission to address the significant unmet need for innovative disease modifying therapies, with the potential to halt the progression of T1D. “We believe GNTI-122 could fundamentally alter the treatment paradigm for T1D by targeting the root cause of disease, interrupting its progression and preserving natural endogenous insulin production”, said Mark Bach, Chief Medical Officer at GentiBio. “We know that preserving natural insulin production can prevent many of the long-term complications and comorbidities that are associated with T1D treated with insulin therapy.”
Orphan Drug Designation is granted by the FDA to drugs and biologics intended to treat rare diseases or an orphan subset of non-rare conditions affecting fewer than 200,000 people in the United States. The designation provides certain development incentives, including tax credits for qualified clinical trials, exemption from user fees, and potentially seven years of market exclusivity upon regulatory approval. GentiBio plans to initiate the POLARIS Study, a Phase 1 clinical trial of GNTI-122 in adults with recently diagnosed T1D, later this year at centers for diabetes treatment across the US (NCT06919354).
About GentiBio, Inc.
GentiBio, Inc. is a biotherapeutics company co-founded by pioneers in Treg biology and immunology from Seattle Children’s Research Institute and Benaroya Research Institute to develop engineered regulatory T cells programmed to treat autoimmune and inflammatory diseases. GentiBio’s Series A financing was led by Matrix Capital Management with participation by Avidity Partners, JDRF T1D Fund, seed investors OrbiMed, RA Capital Management, Novartis Venture Fund, and Seattle Children’s Research Institute. GentiBio’s autologous and allogeneic engineered Tregs platforms integrate key technologies designed to successfully (re)establish immune tolerance and overcome major limitations in existing Treg therapeutics. GentiBio is at the forefront of leveraging a unique therapeutic modality that has the potential to address the fundamental cause of many diseases that result from overactivity and/or malfunctioning of the immune system. To learn more, visit www.gentibio.com.
Media Contact
Hatem Heikal
Vice President, Finance
Hatem.Heikal@GentiBio.com