Sino-US gene therapy specialist Frontera Therapeutics has announced the first patient dosing in a clinical study for its in-house developed gene therapy FT-002. This marks a significant milestone in the development of treatments for X-linked retinitis pigmentosa (XLRP), a genetic disorder affecting vision.
FT-002: First-in-Class Gene Therapy
FT-002 is a recombinant adeno-associated virus (rAAV) being developed to treat XLRP caused by retinitis pigmentosa GTPase regulator (RPGR) mutation. It is the first of its kind to enter clinical trials in China. The potential first-in-class drug is designed to use rAAV to carry the target gene and enable retinal cells to express active functional protein through intraocular injection. This approach aims to repair the structure and function of damaged retinal cells, with a single injection capable of effectively delaying disease progression or restoring visual function in patients.
Clinical Study Significance
The initiation of the clinical study for FT-002 is a crucial step in bringing this innovative treatment closer to patients. If successful, FT-002 could offer a significant advancement in the treatment of XLRP, potentially improving the quality of life for patients affected by this debilitating condition.-Fineline Info & Tech
