Table 1.
Non-neovascular age-related macular degeneration trials
| Trial | Disease | Sample size | VA inclusion criteria | Cell derivation and preparation | Transplantation approach | Immuno-suppression | VA outcomes | Adverse events (AE) |
|---|---|---|---|---|---|---|---|---|
|
Palucorcel Phase 1/2a |
NNV-AMD | 35 |
Phase 1: ≤ 20/200 Phase 2a: ≤ 20/80 |
hUTC suspension | Suprachoroidal subretinal delivery | None |
> 10 letter gain in 10/29 > 15 letter gain in 7/29 |
Retinal detachments in 6/35 Retinal perforations in 13/35 Subretinal delivery aborted due to retinal perforations during the surgical procedure in 2/35 AE related to surgery in 25/33 AE related to surgical delivery system in 19/33 AE related to cell suspension in 5/33 |
|
Palucorcel Phase 2b |
NNV-AMD | 21 | 20/80 to 20/800 | hUTC suspension | Suprachoroidal subretinal delivery | None |
Mean VA change: − 5.7 letters (vs. − 3.7 in control group) > 15 letter gain in 0/21 |
No serious AEs, no RDs, no perforations, no significant changes in IOP > 15 letters lost in 3/21 |
|
CPCB-RPE1 Phase 1/2a |
NNV-AMD | 5 | ≤ 20/200 | hESC-RPE polarized monolayer | PPV and subretinal delivery | Tacrolimus |
> 15 letter gain in 1/5 Stable vision in 4/5 |
Subretinal hemorrhage requiring bevacizumab treatment in 1/5 Implantation aborted due to subretinal debris 1/5 |
|
Advanced Cell Technology Phase 1/2a |
NNV-AMD/ SMD |
9 NN-AMD (3 dose escalation cohorts) |
≤ 20/200 | hESC-RPE suspension | PPV and subretinal delivery | Tacrolimus and MMF |
> 15 letter gain in 4/9 10–14 letter gain in 2/9 Stable vision in 3/9 |
Preretinal pigmentation in 2/9 |
|
South Korean Phase 1/2a |
NNV-AMD/ SMD |
2 NN-AMD | ≤ 20/320 | hESC-RPE suspension | PPV and subretinal delivery | Tacrolimus and MMF | Improved VA in 1/2 |
Discontinuation of immunosuppression due to side effects in 1/2 Choroidal neovascular membrane in 1/2 |
h-ESC human embryonic stem cells; HLA human leukocyte antigen; iPSCs induced pluripotent stem cells; MMF mycophenolate mofetil; NV-AMD neovascular age-related macular degeneration; PPV pars plana vitrectomy; RPE retinal pigment epithelium; SMD Stargardt’s macular dystrophy; VA visual acuity