Date: 2017-05-31
Type of
information: Treatment of the first patient
phase: 1
Announcement: treatment of the first patient
Company: RegenXBio (USA - MD)
Product: RGX-314
Action
mechanism:
- gene therapy. RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein. The expressed protein is designed to neutralize VEGF activity, modifying the pathway for formation of new leaky blood vessels and retinal fluid accumulation. The long-term, stable delivery of this therapeutic protein following a 1 time gene therapy treatment for nAMD could potentially reduce the treatment burden of currently available therapies while maintaining vision with a favorable benefit:risk profile.
- In preclinical animal models with conditions similar to macular degeneration, significant and dose-dependent reduction of blood vessel growth and prevention of disease progression was observed after a single subretinal dose of RGX-314.
- It is being developed under a multi-institutional collaboration with world-renowned gene therapy and ophthalmology experts James M. Wilson, M.D., Ph.D., Jean Bennett, M.D., Ph.D. and Albert Maguire, M.D. from the University of Pennsylvania’s Gene Therapy Program and Center for Advanced Retinal and Ocular Therapeutics (Penn), respectively, and Peter Campochiaro, M.D. at the Johns Hopkins Wilmer Eye Institute (Johns Hopkins).
Disease: wet age-related macular degeneration (wet AMD)
Therapeutic
area: Ophtalmological diseases
Country:
Trial
details:
- RGX-314 will be evaluated in a Phase I, multi-center, open-label, multiple-cohort, dose-escalation study in adult subjects with wet AMD in the United States. The study is expected to include approximately eighteen previously treated wet AMD subjects that are responsive to anti-vascular endothelial growth factor (anti-VEGF) therapy and are 50 years of age or older. The study is designed to evaluate three doses of RGX-314 (3 ×109genome copies (GC)/eye, 1 ×1010GC/eye, and 6 ×1010 GC/eye). Primary endpoints include adverse events, certain laboratory measures (including immunological parameters) and ocular examinations and imaging (including BCVA and SD-OCT). The primary purpose of the clinical study is to evaluate the safety and tolerability of RGX-314 at 24 weeks after a single dose of RGX-314 administered by subretinal delivery. Following completion of the primary study period, it is expected that subjects will enter the follow-up period and will continue to be assessed until week 106 to assess long term safety and durability of effect. (NCT03066258)
Latest
news:
- • On May 31, 2017, RegenXBio announced that the first patient was dosed in a phase I clinical trial evaluating RGX-314 for patients with wet age-related macular degeneration. Six leading retinal surgery centers across the United States are expected to participate in the Phase I trial of RGX-314. The company plans to share an interim trial update by the end of the year.
- • On February 14, 2017, Regenxbio announced the Investigational New Drug application (IND) is active for the planned multi-center, open-label, multiple-cohort, dose-escalation Phase I clinical trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). Six leading retinal surgery centers across the United States, including Penn and Johns Hopkins, are expected to participate in the Phase I trial of RGX-314.
Is
general: Yes
