Regenxbio Reveals Interim Data From Diabetic Retinopathy Gene Therapy Trial

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  • Regenxbio Inc RGNX announced initial data from the Phase 2 ALTITUDE trial of RGX-314 for diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME). 
  • The data were presented at the American Society of Retina Specialists Annual Meeting.
  • RGX-314 is a potential one-time gene therapy under development for wet age-related macular degeneration and DR.
  • Of the 15 patients dosed with RGX-314 in Cohort 1, five patients (33%) demonstrated a two-step or greater improvement from baseline on the disease severity scale at three months, compared to zero in the observational control group. 
  • One patient dosed with RGX-314 had a four-step improvement.
  • In the seven patients who had NPDR (DR severity level 47-53) at baseline, three patients (43%) demonstrated a two-step or greater improvement. 
  • In the eight patients who had PDR (DR severity level over 61) at baseline, two patients (25%) demonstrated a two-step or greater improvement.
  • Related: AbbVie, RegenXBio Ink $1.75B Retinal Gene Therapy Collaboration.
  • Price Action: RGNX shares are up 1.07% at $32.01 during the market session on the last check Monday.
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Posted In: BiotechNewsHealth CareSmall CapGeneralBriefsgene therapyPhase 2 Trial
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