Frontera Therapeutics has dosed its first patients in a gene therapy trial for the treatment of X-Linked retinitis pigmentosa (XLRP).
FT-002 is an intraocular injection of recombinant adeno-associated virus (AAV) carrying the gene that expresses active functional proteins and repairs damaged retinal cell structure and function.
The company said one injection could effectively delay disease progression or restore the patient’s visual function.
According to a statement announcing the trial start, FT-002 is intended to treat patients with XLRP caused by RPGR gene mutation. There is no treatment for this disease globally.
It is the first AAV gene therapy drug to be tested in XLRP patients in China and is also a potential first-in-class drug.
In a further announcement, Frontera Therapeutics said the first patient has been dosed in a clinical trial of its innovative gene therapy product, FT-003, at the Ophthalmology Hospital of Tianjin Medical University in Tianjin, China. FT-003 is being studied for the treatment of neovascular (wet) age-related macular degeneration (AMD).
Dr Yong Dai, founder and CEO of Frontera, said FT-003 is designed as a one-time treatment for patients with wet AMD.
Frontera is also completing a Phase 1 clinical trial of its lead gene therapy program, FT001, for the treatment of Leber Congenital Amaurosis-2, a severe inherited retinal disease related to a mutation in the RPE65 gene.
Initial clinical results for both FT-001 and FT-003 are expected later this year.
