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QLT091001 is a synthetic retinoid replacement for 11-cis-retinal. It is an investigational product under development for the treatment of retinal diseases caused by gene mutations that interfere with the availability of 11-cis-retinal.
Preclinical studies have investigated QLT091001 in mouse and dog models of LCA. Oral administration of QLT091001 to mice with mutations in either RPE65 or LRAT genes showed evidence of having corrected the biochemical defect in the retinoid cycle in light-sensing cells (rods) and appeared to restore the ERG responses to light, 2009 ASRS poster. Intravitreal injection of QLT091001 caused a marked improvement in visual function in dogs with LCA-like disease, with no observed adverse effects, 2009 ARVO poster.
A phase I study of oral QLT091001 was completed in healthy volunteers to evaluate safety and estimate an appropriate dose for studies in patients. Results of this study are described in a press release issued, March 12 2009.
An open-label, phase Ib study /proof-of-concept study to evaluate the effects of oral QLT091001 in children with Leber Congenital Amaurosis (LCA) due to inherited defects of retinal pigment epithelium protein 65 (RPE65) or lecithin-retinol acyltransferase (LRAT) is currently underway, see Clinical Trials.
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