PGN-503

PGN-503 is an investigational gene therapy drug developed by Periphagen for the prevention and treatment of chemotherapy-induced peripheral neuropathy (CIPN), particularly in patients receiving chemotherapeutic agents such as paclitaxel, oxaliplatin, bortezomib, and cisplatin[1][3][4][6]. The drug utilizes Periphagen's Neuronal Therapeutics (NET) platform to deliver a gene construct encoding neurotrophin-3 (NTF3) directly into sensory neurons via intradermal injection. Once delivered, the vector is taken up by nerve endings and transported to the dorsal root ganglion where it stimulates local production of neurotrophin-3. This promotes nerve survival and regeneration while protecting against or reversing CIPN without affecting chemotherapy efficacy or causing systemic side effects[4][6]. As of March 2025, PGN-503 remains in preclinical development with plans for clinical trials in breast cancer and colorectal cancer patients at risk for CIPN[2][3][6].