Table 2.
Treatment Currently Under Investigation for Stargardt Disease
| Treatment | Class | Mechanism | Company | Phase |
|---|---|---|---|---|
| ACU-4429 (Emixustat) | Visual Cycle Modulator | Inhibition of retinoid isomerohydrolase | Acucela Inc | Phase 3 (SeaSTAR) |
| ALK-001 | Visual Cycle Modulator | Chemically modified Vitamin A preventing Vitamin A dimerization | Alkeus Pharmaceuticals |
Phase 2 (TEASE) |
| VM200 | Visual Cycle Modulator | Primary ammine which reacts with all-trans retinal forming a non-toxic Schiff base | Vision Medicine |
Preclinical Trials |
| Isotretinoin | Visual Cycle Modulator | Inhibition of 11-cis-retinol dehydrogenase | Patent expired | Preclinical Trials |
| Fenretinide | Visual Cycle Modulator | Synthetic retinoid which binds to retinol-binding protein 4 | Sirion Therapeutics |
Preclinical Trials |
| A1120 | Visual Cycle Modulator | Non retinoid RBP4 antagonist | iCura Vision | Preclinical Trials |
| Avacincaptad pegol (Zimura®) | Complement inhibition | Complement C5 inhibitor |
Ophthotech Corporation |
Phase 2b |
| SAR422459 | Gene therapy | Recombinant lentiviral vector containing a functioning ABCA4 | Sanofi, Oxford Biomedica |
Phase I/II |
| Human embryonic stem cell-derived RPE | Stem cells therapy | Replacement of RPE cells restoring the function of overlying retina | Advanced Cell Technology |
Phase I/II |