FT-002 / Frontera - LARVOL DELTA

Phase II Preliminary Results of Subretinal Gene Therapy FT-002 in Pediatric Participants with RPGR-associated XLRP (ARVO 2026) - P1/2 | "Conclusions FT-002 is safe in pediatric participants, with no new safety signals identified. A single subretinal injection of FT-002 leads to marked improvements in visual acuity and retinal function, holding great promise to be an effective treatment for patients with RPGR-associated XLRP."

Clinical • Gene therapy • P2 data • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

12-Month Safety and Efficacy Evaluation of Subretinal Gene Therapy (FT-002) for the Treatment of RPGR Gene Mutation Associated X-linked Retinitis Pigmentosa (ASGCT 2025) - P1 | "Subretinal injection of FT-002 at doses of 5×1010 to 20×1010 vg/eye was generally well-tolerated with no RPE changes noted. FT-002 has the potential to provide long-term clinical benefits for XLRP patients with defects in the RPGR gene, including improvements in visual acuity and retinal sensitivity. Disease Focus of Abstract:Ophthalmic Diseases"

Clinical • Gene therapy • Gene Therapies • Genetic Disorders • Inflammation • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa (clinicaltrials.gov) - P1/2 | N=32 | Recruiting | Sponsor: Frontera Therapeutics

Gene therapy • New P1/2 trial • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Efficacy and Safety of a Novel RPGROFR15 Gene Therapy (FT-002) for the Treatment of RPGR-Associated XLRP (ASGCT 2024) - "Together, the comprehensive preclinical assessment demonstrates that subretinal delivery of FT-002 led to durable hRPGRORF15 expression in the retina and provided phenotypic and functional rescues of XLRP in the mouse model. The data supports the initiation of Ph I/II clinical study for the treatment of RPGR-associated XLRP patients with FT-002."

Clinical • Gene therapy • Gene Therapies • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: Frontera Therapeutics

Gene therapy • New P1 trial • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa