Frontera Therapeutics Doses First Patient in a Trial of Gene Therapy for the Treatment of X-Linked Retinitis Pigmentosa

Frontera Therapeutics recently announced it has dosed the first patient in a clinical trial of its gene therapy product, FT-002. FT-002 is being studied for the treatment of X-linked retinitis pigmentosa (XLRP) and is Frontera’s third gene therapy product candidate to enter the clinic.

“I am very pleased with our speed of execution and the rapid buildup of our medical and clinical operations capabilities. As a company, we now have three gene therapy clinical studies running in parallel,” said Xinyan Li, PhD, President and Chief Medical Officer of Frontera.

“FT-002 was manufactured entirely in-house and administered to the first patient as a first-in-class product, again validating the maturity and advancement of the company’s AAV R&D platform technology,” said Yong Dai, PhD, founder and CEO of Frontera Therapeutics. “Driven by our mission to develop novel and best-in-class gene therapies, the Frontera team accelerated the R&D process to enable FT-002 to enter the clinic smoothly, and we believe FT-002 could greatly improve the quality of life for XLRP patients for whom there are currently no effective treatments. This year, the company expects to advance additional product candidates into preclinical and clinical studies, which we believe may provide more patients with curative opportunities.”

FT-002 is a recombinant adeno-associated virus gene therapy drug intended to treat patients with X-linked retinitis pigmentosa (XLRP) caused by RPGR gene mutation. There is no treatment for this disease globally. FT-002 is an intraocular injection of recombinant AAV virus carrying the gene that expresses active functional proteins and repairs damaged retinal cell structure and function. One injection can effectively delay disease progression or restore the patient’s visual function. FT-002 is the first AAV gene therapy drug to be tested in XLRP patients in China and is also a potential first-in-class drug.

X-linked retinitis pigmentosa (XLRP) is a form of retinitis pigmentosa (RP) that is mainly transmitted via X-linked recessive inheritance. The clinical symptoms of XLRP are normally severe. Starting around the age of ten, clinical signs 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. XLRP is mainly caused by the RPGR gene mutation, accounting for 70-80% of all cases of XLRP. The RPGR gene encodes the RPGR protein, which is located at the ciliary junction of photoreceptor cells and is involved in protein transport in photoreceptor cells. Studies have shown that the prevalence of XLRP due to RPGR gene variants in European and American men is 3.4-4.4 per 100,000, approximately 20,000 patients. In China, approximately 30,000 to 50,000 people have XLRP.

Frontera Therapeutics is a global, fully integrated, clinical-stage biotechnology company leveraging its novel APEX Technology & Manufacturing platform to develop and manufacture superior and novel gene therapy candidates across multiple disease areas. 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 products. The in-house GMP manufacturing capabilities enable rapid production across the product lifecycle, and efficiently advance AAV therapies from research to clinical development. Frontera’s development pipeline spans not only orphan diseases, but also targets large patient markets – including ophthalmology, hematology, cardiovascular, neurology and metabolic diseases. Three of Frontera’s gene therapy product candidates, FT-001 for LCA-2, FT-002 for XLRP, and FT-003 for wet AMD, have entered clinical development. Frontera Therapeutics has offices in the US and China, allowing it to target global patient populations. For more information, visit www.fronteratherapeutics.com.