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Isabelle S Audo, Richard G Weleber, Tim Stout, Andreas K Lauer, Mark E Pennesi, Saddek Mohand-Said, Pierre-Olivier Barale, Ronald Buggage, David J Wilson, Jose Alain Sahel; Early findings in a Phase I/IIa clinical program for Stargardt disease (STGD1, MIM #248200). Invest. Ophthalmol. Vis. Sci. 2015;56(7 ):3819.
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To present the early findings of a Phase I/IIa clinical program for Stargardt disease (STGD1, MIM #248200) designed to evaluate the safety of ABCA4 replacement therapy with a lentiviral-based vector (StarGen™) administered by subretinal injection.
StarGen™ is ocular gene replacement therapy of ABCA4 using lentiviral-based vector (recombinant Equine Infectious Anemia Virus [EIAV]). StarGen™, a treatment for Stargardt disease, is currently under clinical evaluations in a Phase I/IIa program. The clinical program consists of a 48-week open-label study (NCT01367444) designed as a 4-cohort dose escalation phase followed by an expanded cohort at the highest safe and tolerated dose and a long-term follow-up study (NCT01736592). Treatment evaluation includes best-corrected visual acuity, dilated slit-lamp examination, indirect ophthalmoscopy, VFQ-25, full-field semi-automated kinetic perimetry, full-field GATE static perimetry, microperimetry, electroretinography, fundus photography, fundus autofluorescence, optical coherence tomography, and adaptive optics imaging.
To date, 16 patients with Stargardt disease have been enrolled with follow-up ranging from 9 to 42 months. The dose-escalation phase of the trial has been completed and the expanded cohort is in progress. Subretinal administration of StarGen™ has been well tolerated, causing no ocular inflammation in any patients to date. Immune response to a capsid protein has been observed in two out of 14 patients treated with StarGen™. No significant changes in functional or structural tests were observed after treatment.
StarGen™ has been safe and well tolerated. Neither harmful effect nor biological activity has been observed in the treated patients with advanced Stargardt disease (poor visual acuity and no effective retinal function on ERG). For this reason, a sixth cohort including patients of 6-year old or older will be added to continue to assess safety and better document biological activity.
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