Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients

This study is a long term, follow up to the ACT MA09-hRPE 001 phase I/II trial, the phase
I/II trial (referred to as the core protocol) was an open-label, non-randomized, dose
escalation, multi-center trial. Thirteen SMD patients were treated in this trial. Ten
patients with profound vision loss (visual acuity <=20/400) received a single subretinal
injection of MA09-hRPE cells, starting at a dose of 50,000 MA09-hRPE cells (three patients),
100,000 MA09-hRPE cells transplanted (three patients), 150,000 MA09-hRPE cells transplanted
(three patients) and increasing to a maximum dose of 200,000 MA09-hRPE cells transplanted
(one patient). Three patients with severe to moderate vision loss (visual acuity <= 20/100)
received a dose of 100,000 MA09-hRPE cells. All patients who participated in the core
protocol are eligible for participation in this follow-up protocol.

The first visit of this long term follow-up protocol will correspond to the last visit of the
core protocol, and will take place at 12 months post cell implantation. Informed consent will
be obtained at this visit.

Patients will be evaluated at 18, 24, 36, 48 and 60 months post-transplant (or more
frequently as clinically indicated). Follow-up will include obtaining information about
ophthalmological findings and events of special interest as defined in the Primary Outcomes.
At the last visit of this follow-up study, whether at 60 months post-transplant or at early
discontinuation, patients will be invited to participate in a Safety Surveillance Study for
an additional 10 years, under a separate protocol, which will continue to monitor the long
term risks of MA09-hRPE cell transplantation.

Inclusion Criteria:

- Must have been treated with hESC-RPE cell transplant in the core protocol.

- Able to understand and willing to sign the informed consent to participate in the
follow-up study.

Exclusion Criteria:

- There are no exclusion criteria