RX001

RX001 is a first-in-class, adeno-associated virus (AAV) based gene therapy developed by GeneCraft. The therapy is built on the tumor suppressor gene **RUNX3**, designed to selectively target and eliminate cancer cells, especially in lung cancer, while sparing normal cells. This targeted mechanism is intended to minimize the side effects commonly associated with traditional cancer treatments. RX001 is being investigated as a monotherapy for **advanced KRAS mutant non-small cell lung cancer (NSCLC)**, with drug delivery achieved via direct intratumoral injection, such as through bronchoscopy or CT-guided percutaneous injection. The therapy employs gene transference to introduce RUNX3 to cancer cells, leveraging its role as a tumor suppressor to induce selective cancer cell death and modulate the tumor microenvironment[1][3][4][5][7].

Other names

RX001RX-001RX 001AAV-hRUNX3AAV-hRUNX-3AAV-hRUNX 3