Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD)

This study is an open-label dose-escalation investigation of the safety and preliminary
efficacy of unilateral subretinal transplantation of HuCNS-SC cells in subjects with
Geographic Atrophy secondary to Age-Related Macular Degeneration (AMD). Subjects will be
enrolled based on specific inclusion/exclusion criteria and evaluated at regular post
transplant intervals. The investigation will be divided into two sequential cohorts.
Subjects will be enrolled into each cohort based on best-corrected visual acuity (BCVA)
visual acuity as determined by the Electronic Early Treatment Diabetic Retinopathy Study
(E-ETDRS) acuity test. BCVA of less than or equal to 20/400 in the Study Eye will be
enrolled in Cohort I. Subjects with BCVA of 20/320 to 20/100 in the Study Eye will be
enrolled in Cohort II. Cohort I will consist of four subjects who will undergo transplant
with 200,000 cells followed by four subjects who will undergo transplant with 1 million
cells. Cohort II will consist of 7 subjects who will undergo transplant with 1 million
cells. An independent Data Monitoring Committee (DMC) will review accruing safety data for
all subjects.

HuCNS-SC cells will be transplanted by a board-certified vitreoretinal surgeon. The
transplantation will be conducted in the eye with the inferior best-corrected visual acuity
(BCVA) (i.e., the Study Eye). Only the Study Eye will undergo transplantation. The HuCNS-SC
cells will be administered into the subretinal space through a standard surgical approach.

Immunosuppressive agents will be administered orally to all subjects for a period of three
months after surgery.

Subjects will be monitored frequently for a total of one year after HuCNS-SC cell
transplantation. An additional four years of monitoring will be conducted in a separate
follow-up study that will commence with the termination visit of the Phase I/II
investigation. The follow-up study will be conducted as a separate investigation.

Inclusion Criteria:

- Diagnosis of age-related macular degeneration with geographic atrophy (GA)

- Only patients with a specific degree and extent of GA will be eligible

- Subjects with best-corrected visual acuity (BCVA) of less than or equal to 20/400 in
the Study Eye will be enrolled in Cohort I. Subjects with BCVA of 20/320 to 20/100 in
the Study Eye will be enrolled in Cohort II

- No prior or current choroidal neovascularization in either eye

- Must have adequate care-giver support and access to medical care in the local
community

- Able to provide written informed consent prior to any study related procedures

- Agree to comply in good faith with all conditions of the study and to attend all
required study visits

Exclusion Criteria:

- Prior vitreal or retinal surgery

- Glaucoma

- Atrophic macular disease of any other cause

- Diabetic retinopathy or diabetic macular edema in either eye

- Previous organ, tissue or bone marrow transplantation

- Previous participation in a gene transfer or a cell transplant trial

- Autoimmune disease

- Allergy to tacrolimus, mycophenolate mofetil (MMF), scopolamine, Cyclogyl,
Moxifloxacin, or Gatifloxacin

- Current or prior malignancy (or is on chemotherapy)