Published in Pipeline

Gene therapy trial for x-linked RP doses first patient

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Frontera Therapeutics, Inc. announced the dosing of the first patient in a clinical trial assessing FT-002, its gene therapy formulation for the treatment of x-linked retinitis pigmentosa (XLRP).

Give me a quick rundown on Frontera Therapeutics.

Frontera is a clinical-stage biotechnology company that has designed a novel APEX technology & manufacturing platform to develop and manufacture novel gene therapy candidates for multiple disease states, including wet age-related macular degeneration (AMD) and Leber congenital amaurosis-2 (LCA-2).

Tell me more about this platform.

The platform is an innovative adeno-associated virus (AAV) gene expression system that aims to optimize both new and clinically validated AAV vectors to enhance the safety and efficacy profiles of gene therapy candidates by leveraging vectorology, capsid selection, assay and disease modeling, and GMP (Good Manufacturing Practice).

How is XLRP different from RP?

XLP is a type of RP primarily transmitted via x-linked recessive inheritance and is usually caused by a defect in the retinitis pigmentosa GTPase regulator (RPGR) gene. Clinical symptoms and signs might include night blindness followed by a gradual decrease in visual field and vision acuity with severe vision loss or even blindness that occurs around the age of 40.

Tell me about FT-002.

The recombinant AAV gene therapy drug is Frontera’s third gene therapy candidate to enter clinical testing. It operates as a single intraocular injection that repairs damaged retinal cell structure and function.

How was it developed?

According to Frontera, FT-002 was manufactured entirely in-house and is being investigated as a first-in-class therapy through the company’s novel APEX technology and manufacturing platform.


With no currently approved treatment for XLRP, FT-002 is the first AAV-based gene therapy drug that could potentially enhance the quality of life for patients’ suffering from this genetic condition.