Table 2.
Summary of therapeutic strategies for Leber Congenital Amaurosis.
| University | Drug | Company | Genome | Capsid | Gene Modifications | Promoter | Polyadenylation | Dosage | Administration | |
|---|---|---|---|---|---|---|---|---|---|---|
| Gene Augmentation Therapy | Pennsylvania | Luxturna | Sparks Therapeutics Inc. | 2 | 2 | modified Kozak sequence/hybrid cytomegalovirus enhancer |
β-actin promoter | SV40 | 1.5E10 to1.5E11 vg | Single-dose subretinal injection |
| London | tgAAG76 | Targeted Genetics Corp | 2 | 2 | hRPE65 | BSA | 1E11 | Single-dose subretinal injection |
||
| London | OPTIRPE65 | MeiraGTx UK II Ltd. | 2 | 5 | Condon-optimized/ Kozak sequence |
NA65p Optimized hRPE65 promoter |
SV40 | - | Single-dose subretinal injection |
|
| Florida | rAAV2-CBSB-hRPE65 | National Eye Institute, AGTC |
2 | 2 | CMV enhancer | CBSB Cystathionine-beta-synthase | 5.96E10 to 18E10 | Single dose subretinal injection |
||
| Nantes | rAAV2/4- hRPE65 | AFM, Horama |
2 | 4 | - | hRPE65 | BSA | 1.2E10 to 4.8E10 | Single-dose subretinal injection |
|
| Israel | rAAV2-CB-hRPE65 | Hadassah Medical Organization |
2 | 2 | CMV enhanced | β-actin promoter | 1.19E10 | Single-dose subretinal injection |
||
| Oregon | rAAV2-CB-hRPE65 | Applied Genetic Technology | 2 | 2 | CMV enhanced | β-actin promoter | 1.8E11 to 6E11 | Single-dose subretinal injection |
||
| University | Drug | Company | Component | Dosage | Administration | |||||
| 11-cis Retinal Replacement | Illinois | QLT091001 | QLT Inc. | synthetic 9-cis-retinyl acetate | 40 mg/m2/day | 7-day oral administration | ||||
| University | Drug | Company | Component | Dosage | Administration | |||||
| RNA Therapy | Iowa | QR-110 Sepofarsen | ProQR Therapeutics | 2′ O-methyl-modified RNA oligonucleotide | - | intravitreal injection | ||||
| University | Drug | Company | Capsid | g-RNA/Cas9 | Gene modifications | Promoter | Polyadenylation | Dosage | Administration | |
| Gene Editing Therapy | Florida | EDIT-101 | Allergan | 5 | CEP290-323 & CEP290-64/Staphylococcus aureus Cas9 |
consensus kozak sequence and ATG start codon | human U6 polymerase III | SV40 | - | Single-dose subretinal injection |